Author Archives: Kirti5

Cyrus Biotechnology Congratulates David Baker, 2024 Nobel Laureate in Chemistry and Cyrus Co-Founder

Baker’s technology overcoming challenges in developing protein drugs

Cyrus Biotechnology today congratulated David Baker from the University of Washington and Demis Hassabis and John Jumper from Google DeepMind for winning and sharing the Nobel Laureate Award in Chemistry based on their extraordinary discoveries in designing and predicting protein structures. Baker, who co-founded Cyrus with Lucas Nivon and Yifan Song, who trained under his leadership, was the first to make high accuracy protein structure predictions, design a novel protein in a computer, and has since achieved many “firsts” in the field. His work includes artificial proteins with new topologies and structures, and hyperstable miniproteins that can act in the challenging environments of the human gut and airways. In addition his work includes new vaccine platforms including for COVID-19, and novel proteins that block viruses or cancer-associated factors. The three laureates’ research also greatly accelerated innovation in the protein AI field and today almost any protein structure can be designed.

“Since being founded in the Baker lab, Cyrus Biotechnology has been proud to be part of this history and is focused on bringing the best of these new technologies to patients through the design of innovative therapeutics,” said Lucas Nivon, Cyrus CEO. “By applying the technology created by Baker’s team, Cyrus scientists are solving some of the biggest challenges of protein drugs, while also developing our own algorithms and new datasets in-house and with the Openfold AI consortium that Cyrus co-founded in 2022,” Lucas added.

Protein drugs often cause strong immune reactions in patients, which can limit drug potency and pose unacceptable safety risks. AI methods are assisting Cyrus researchers in predicting what parts of a protein are immunogenic and how best to modify these without damaging desirable properties. AI methods are also being applied at Cyrus to design therapeutics based on natural proteins, but which have better stability, activity, and potency as well as reduced immunogenicity.

“Cyrus Biotechnology’s engineering of the enzyme IdeS is but one excellent example that connects the tools of the Baker lab to a drug candidate heading to the clinic. IdeS is a highly immunogenic protease from a bacterial pathogen that degrades antibodies, an ideal mechanism for the treatment of antibody-mediated autoimmune diseases such as immune thrombocytopenia, myasthenia gravis, and rheumatoid arthritis, and a total of over 70 known autoimmune diseases with poor current standard of care,” said Yifan Song, Chief Scientific Officer.

Cyrus Biotechnology is expecting to bring a variant of IdeS optimized by AI methods to first-in-human trials in 2026.

 

About Cyrus Biotechnology

Cyrus Biotechnology is a pre-clinical-stage AI-driven therapeutics company with an internal pipeline of novel biologics primarily in autoimmune indications. Cyrus is advancing a next generation IdeS IgG protease with half-life extension and immunogenicity optimization, for treatment of IgG-mediated autoimmune indications, into IND-enabling studies, as well as advancing multiple discovery stage cytokine programs. Cyrus was co-founded with Prof. David Baker, inventor of Rosetta and numerous protein design AI tools, and has worked with dozens of Pharma firms on protein redesign for novel therapeutics, including Genentech, Janssen, and Selecta. In 2021 Cyrus started to develop its own therapeutics. Cyrus’ investors are Orbimed, Agent Capital, Hillhouse, Alexandria, and others.For more information about Cyrus please visit https://cyrusbio.com/

 

NOTICE: The information contained in this document is dated as of Oct 9, 2024. Cyrus Biotechnology, Inc. (the Company) disclaims any obligation to update such information after such date. This document contains forward–looking statements reflecting the Company’s current expectations that necessarily involve risks and uncertainties. Actual results and the timing of events may differ materially from those contained in such forward-looking statements due to a number of factors and the Company undertakes no obligation to revise or update any forward-looking statement to reflect events or circumstances after the issuance of this press release.

 

Contacts:

Lucas Nivon
[email protected]
206-258-6561

 

Source: Business Wire and Cyrus

āshibio Announces Appointment of Dipal Doshi, CEO of Entrada Therapeutics, to its Board of Directors

BRISBANE, Calif. (Sept. 17, 2024) – āshibio, a privately held, clinical-stage biotechnology company developing novel therapeutics for the treatment of bone and connective tissue disorders, today announced the appointment of Dipal Doshi to its Board of Directors. Mr. Doshi currently serves as Chief Executive Officer of Entrada Therapeutics (NASDAQ: TRDA).

“Dipal is a seasoned leader with significant experience setting strategy and driving the operational and commercial growth of biopharmaceutical companies,” said Pankaj Bhargava, M.D., Chief Executive Officer of āshibio. “Under Dipal’s leadership, Entrada has advanced its innovative pipeline of intracellular therapeutics into the clinic, completed multiple private financings, led its successful IPO in 2021, and executed on a transformational business development collaboration. His record of accomplishment is remarkable, and his operating and fundraising experience will be tremendously helpful in the next phase of āshibio’s growth.”

āshibio exited stealth mode in June of 2024 with $40 million in seed and Series A financing. The funding has enabled continued development of investigative therapies targeting bone and connective tissue disorders, including a potential treatment for fibrodysplasia ossificans progressiva (FOP), a rare genetic disease characterized by severe and progressive heterotopic ossification (HO), a pathological condition that causes abnormal bone formation in muscles, tendons, ligaments, and other soft tissues.

“I am honored to join āshibio’s Board as the company continues to advance its novel therapeutic pipeline,” noted Mr. Doshi. “āshibio’s strong focus on bone and connective tissue disorders that have no approved treatments, or where the treatment options are inadequate, fills an important gap in rare disease drug development. Pankaj and his team have the experience and support to deliver for these patients and their families, including those living with FOP, and I am looking forward to helping them succeed.”

“I have had the opportunity to work with Dipal as an investor and former member of Entrada’s Board of Directors and have seen firsthand his dedication and passionate leadership,” said Todd Foley, Chairman of āshibio’s Board. “He has built and led a diverse team focused on a new class of intracellular therapies, and his passion for developing drugs that address the unmet needs of patients living with rare diseases is inspiring. I welcome his voice to āshibio’s Board to contribute his insight and experience to aid in the company’s continued progress.”

Dipal is the Chief Executive Officer of Entrada Therapeutics and a Member of its Board of Directors. He previously served as President and Chief Executive Officer from 2017 to 2023. Dipal has led many critical functions in biotechnology and pharmaceutical companies, including roles focused on business development, corporate strategy, new product planning, commercial planning, and finance. Prior to joining Entrada, Dipal was the Chief Business Officer at Amicus Therapeutics, a global biotechnology company focused on rare diseases. He has also held senior level positions at a healthcare private equity fund and Catalent. Earlier in his career, Dipal worked in Merrill Lynch’s Investment Banking Group and held several roles at Eli Lilly and Company.

Dipal holds an MBA from The Wharton School of the University of Pennsylvania and a BA from Rutgers University. Dipal is a Fellow of the Aspen Institute and on the Board of Advisors of Life Science Cares Boston.

###

About āshibio
āshibio is a privately held, clinical-stage biotechnology company developing a pipeline of novel therapeutics for the treatment of bone and connective tissue disorders. Founded in 2022 by company CEO Pankaj Bhargava, M.D., and the team at MPM BioImpact, āshibio exited stealth mode in June 2024 with $40 million in Seed and Series A financing. The company plans to initiate a Phase 2/3 trial of its lead asset, andecaliximab, in the second half of 2024 in patients with fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder characterized by progressive heterotopic ossification (HO), a pathological condition characterized by abnormal bone formation in muscle and soft tissues. For more information, visit www.ashibio.com and follow āshibio on LinkedIn.

Media Contact:
SmithSolve
Matt Pera
(219) 628-0258

SOURCE: āshibio

Metagenomi Announces Preclinical Data for Lead Hemophilia A Program Demonstrating Durable Factor VIII (FVIII) Activity Levels through Twelve Months

Twelve-month durability data from Metagenomi’s ongoing nonhuman primate (NHP) study
in hemophilia A remains generally consistent with data previously released at 4.5 months

NHPs remain healthy and exhibit normal weight gain;
treatment is generally well tolerated

Program on track for IND filing in 2026

Company to host conference call with management
and Dr. Glenn Pierce, international thought leader in hemophilia A

EMERYVILLE, Calif., Sept. 03, 2024 (GLOBE NEWSWIRE) — Metagenomi, Inc. (Nasdaq: MGX), a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary gene editing toolbox, today announced data from an ongoing preclinical study designed to provide evidence supporting the potential durability and safety of the company’s hemophilia A gene editing investigational therapy, MGX-001.

“We are thrilled to achieve this preclinical milestone supporting our recent decision to declare MGX-001 as our development candidate for hemophilia A,” said Brian C. Thomas, PhD, CEO and founder of Metagenomi. “We conducted this NHP study in response to a competitive landscape where gene therapies have been unable to achieve long term persistence of FVIII activity levels in patients. Establishing proof-of-concept of site specific gene integration and durable activity levels of FVIII in NHPs over twelve months in hemophilia A represents an important validation of our platform. Our goal for MGX-001 is to provide a one-time, curative treatment for adults and children with hemophilia A. Furthermore, we intend to leverage the MGX-001 editing platform to pursue additional therapies for secreted protein disorders.”

The NHP study involved treating three NHPs with a single intravenous dose of an adeno associated virus (AAV) containing a FVIII donor template followed 35 days later by a single intravenous dose of a lipid nanoparticle (LNP) containing a novel Metagenomi nuclease and associated guide RNA targeting the first intron of the albumin gene. Each animal received only a single dose of dexamethasone prior to the AAV and LNP doses. The NHPs were then followed for safety and donor FVIII activity. The NHPs also underwent liver biopsy on Day 7 to evaluate editing and integration efficiency. The study remains ongoing.

All NHPs demonstrated durable FVIII activity levels over the twelve-month period. At the twelve-month time point two of the NHPs had FVIII activity levels within normal/near normal range (82% and 41%) while the third NHP had FVIII activity level in the mild hemophilia range (9%). Comparisons of mean values from the three to six month time points to the nine to twelve month time points were highly consistent, demonstrating no significant decline in donor-derived FVIII activity levels. Liver biopsy data demonstrated gene integration in the forward orientation at a frequency of 0.7% to 2.9%; these values positively correlated with FVIII activity levels. Treatment was generally well tolerated with findings limited to moderate transient elevation of liver transaminases following AAV and LNP administration. There were no notable findings in total bilirubin or albumin levels or adverse clinical observations.

This early NHP study was conducted without the benefit of several subsequent optimizations of the therapeutic candidate designed to enhance safety and efficacy in the clinic. For the development candidate MGX-001, the company selected a bioengineered FVIII construct designed to improve FVIII activity levels, optimized the ratio of different LNP components and timing between AAV and LNP administration, and enhanced aspects of the manufacturing processes.

“The treatment of hemophilia, which has undergone many transformative changes over the past 60 years, is poised for yet another disruptive change: the use of genome editing, with site specific integration of FVIII, to produce functional cures in patients with hemophilia A. I am encouraged by the preclinical progress in the genome editing space to potentially provide a new path to a one-time, curative treatment option for both adults and children in hemophilia A in the future,” said Dr. Glenn Pierce, member of the Metagenomi Scientific Advisory Board.

The company will host a conference call at 8:30am ET, on Wednesday, September 4, 2024. The registration link to the webcast can be found at https://ir.metagenomi.co/.

About Hemophilia A

Hemophilia A is the most common X-linked inherited bleeding disorder, caused by a large variety of mutations in the FVIII gene leading to a loss of functional FVIII protein. Intracranial bleeding is of greatest concern as this can lead to major morbidity and mortality. Bleeding into joints leads to cumulative joint damage and is a major cause of morbidity. Diagnosis typically occurs in infancy due to exaggerated bleeding in response to minor injury or routine medical procedures. Prevalence is estimated to be up to 26,500 patients in the US and more than 500,000 patients globally according to the World Federation of Hemophilia (WFH), with the vast majority of patients being male.

About Metagenomi

Metagenomi is a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary, comprehensive metagenomics-derived toolbox. Metagenomi is harnessing the power of metagenomics, the study of genetic material recovered from the natural environment, to unlock four billion years of microbial evolution to discover and develop a suite of novel editing tools capable of correcting any type of genetic mutation found anywhere in the genome. Its comprehensive genome editing toolbox includes programmable nucleases, base editors, and RNA and DNA-mediated integration systems (including prime editing systems and clustered regularly interspaced short palindromic repeat associated transposases). Metagenomi believes its diverse and modular toolbox positions the company to access the entire genome and select the optimal tool to unlock the full potential of genome editing for patients. For more information, please visit https://​metageno​mi​.co.

Cautionary Note Regarding Forward‐​Looking Statements

This press release contains ​“forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements, which are often indicated by terms such as ​“anticipate,” ​“believe,” ​“could,” ​“estimate,” ​“expect,” ​“goal,” ​“intend,” ​“look forward to,” ​“may,” ​“plan,” ​“potential,” ​“predict,” ​“project,” ​“should,” ​“will,” ​“would” and similar expressions, include, but are not limited to, any statements relating to our growth strategy and product development programs, including the timing of and our ability to conduct IND-enabling studies, make regulatory filings such as INDs, statements concerning the potential of therapies and product candidates, including our development candidate, MGX-001, statements concerning the timing of data presentations and publications, and any other statements that are not historical facts. Forward looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition, and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in ​“Risk Factors,” in our most recent Form 10-K and our most recent 10-Qs on file with the Securities and Exchange Commission. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.

Investor Contact:
Simon Harnest – CIO, SVP Investor Relations
simon@​metagenomi.​co

Media Contact:
Ashlye Hodge – Communications Manager
ashlye@​metagenomi.​co

Metagenomi Reports Business Updates and Second Quarter 2024 Financial Results

Declared development candidate MGX-001 for the treatment of hemophilia A;
plans to present 12-month NHP durability study data in September 2024

All Wave 1 Ionis collaboration programs advancing in lead optimization, unlocking potential for multiple development candidate nominations in 2025

Achieved milestone from partner Affini-T related to Metagenomi licensed technology

Well capitalized with $299.9M in cash, cash equivalents and available-for-sale marketable securities at the end of Q2 2024; cash runway anticipated to support operating plans into 2027

EMERYVILLE, Calif., Aug. 14, 2024 (GLOBE NEWSWIRE) — Metagenomi, Inc. (Nasdaq: MGX) (Metagenomi), a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary, comprehensive metagenomics-derived gene editing toolbox, today provided a business update and reported second quarter 2024 financial results.

“We are progressing our pipeline while remaining laser focused on our vision to create curative genetic medicines by harnessing the power of our AI-driven metagenomics platform,” said Brian C. Thomas, PhD, CEO and Founder. “We have significantly advanced our wholly-owned program in hemophilia A, including recent positive regulatory interactions with Food and Drug Administration (FDA) and the declaration of MGX-001 as our lead development candidate. Our team has worked diligently to allow us to accelerate the data release from our 12-month nonhuman primate (NHP) durability study from the end of 2024 into September 2024. This study is an important step towards advancing MGX-001 as a potentially life-long curative treatment for both adults and children with hemophilia A, and demonstrating our ability to potentially achieve large targeted gene integration in patients using our metagenomics toolbox.”

Dr. Thomas continued, “We are also progressing our partnered programs and advancing our metagenomics-based toolbox. The foundation of our Ionis collaboration was strengthened by the addition of two high-value targets associated with cardiometabolic disease, and all collaboration programs have progressed into the lead optimization phase of development. We achieved a development milestone with Affini-T, where our team provided support for Affini-T’s regulatory filing related to Metagenomi licensed technology and current good manufacturing practices (cGMP) material supply. We continue to advance a broad range of technologies to support our goal of being able to address any disease by enabling any therapeutic genome edit in the human genome. Going forward, we remain well capitalized to execute our strategic priorities, with a cash runway into 2027.”

Second Quarter 2024 Business Updates and Key 2024-2025 Anticipated Milestones

Therapeutic Pipeline Updates

  • Metagenomi continues to advance its wholly-owned lead program in hemophilia A, designed as a one-time curative treatment for both adults and children.
    • The company engaged with FDA in the second quarter to discuss key aspects of the planned Investigational New Drug (IND) submission.
    • The company declared a lead development candidate (DC), MGX-001, meeting previously announced guidance for the timing of a DC nomination.
    • The company plans to accelerate the release of data from the 12-month NHP durability study for the lead hemophilia A program from late 2024 into September 2024. The 12-month NHP durability study was designed to show therapeutically relevant, durable expression of Factor VIII in NHPs, addressing a key concern which has impacted other genetic medicines programs in hemophilia A that have experienced loss of Factor VIII expression over time.
    • The company plans to initiate cGMP manufacturing and related IND enabling activities in 2024 and continue IND enabling activities in 2025.
    • As previously disclosed, the company remains on track to file an IND in hemophilia A in 2026.
  • Building on the recent success of the hemophilia A program, Metagenomi plans to advance additional wholly-owned therapeutic candidates targeting secreted protein disorders, leveraging the MGX-001 editing system with the goal of achieving targeted and durable gene expression.
    • This editing system consists of two components: a highly efficient and specific nuclease creates a precise cut at the albumin safe harbor gene locus after delivery by LNP; and an AAV vector delivered FVIII DNA template is inserted into the nuclease cut site by a naturally occurring DNA repair process.
  • Following selection of the remaining Wave 1 targets in the Ionis collaboration in the first quarter of 2024, all four therapeutic programs successfully advanced into the lead optimization phase in the second quarter of 2024.
    • All four therapeutic targets in Wave 1 collaboration focus on cardiometabolic diseases; the company announced Refractory Hypertension as the initial disease target leveraging the angiotensinogen pathway.
    • The company aims to demonstrate in vivo proof-of-concept in 2024, and advance NHP studies supporting DC nominations thereafter.
    • The company plans to nominate one to two DCs for cardiometabolic programs in 2025.
  • The company achieved a milestone in connection with its Affini-T collaboration, establishing cGMP gene editing
    reagents for cell therapy and filing related Drug Master Files with FDA to support an IND for Affini-T’s T-cell receptor-based therapy.

    • Metagenomi received 933,650 shares of Affini-T common stock upon achievement of this regulatory milestone.

Technology Platform Updates

  • Metagenomi achieved in vitro proof-of-concept for an undisclosed neuromuscular target with its ultra small editing system, a key milestone, opening up therapeutic potential for extrahepatic disease targets.
  • Metagenomi achieved in vitro proof-of-concept using its RNA-mediated integration system (RIGS) for undisclosed liver targets.
  • Metagenomi achieved multiplex base editing proof-of-concept and plans to present this data at a scientific conference in the second half of 2024.
  • Metagenomi achieved in vitro proof-of-concept in human cells for large gene integration using its potentially industry leading, compact CRISPR-associated transposase (CAST) technology, with a publication planned.
  • As previously disclosed, the company remains on track to demonstrate in vivo proof-of-concept for a large gene integration in 2026.

Other Business Updates

  • The company continues to exercise fiscal responsibility in prioritizing certain internal development programs while looking for collaboration partners to leverage other development opportunities.
    • Metagenomi is looking for a partner or licensee for further development of its program for primary hyperoxaluria type 1 (PH1). Previously disclosed data demonstrated preclinical proof-of-concept in an accepted disease model of PH1.
    • The company is continuing to focus its internal efforts on in vivo gene editing therapeutic approaches and pursue technology out-licensing for ex vivo cell therapy, where next generation gene editing systems are an important enabler of novel therapies.
    • Going forward, the company is not pursuing amyotrophic lateral sclerosis (ALS) based upon recent peer company clinical data regarding the lack of efficacy of Ataxin-2 as a therapeutic target for ALS.
    • The company is revising its pipeline design to consolidate earlier stage programs and make it easier to identify our near term pipeline priorities focused on in vivo gene editing and liver indications.
  • As a result of the acceleration of all four Wave 1 Ionis targets into lead optimization, as well as the company’s decision to deprioritize PH1, Metagenomi is revising to provide pipeline guidance only through year-end 2025, including the next one to two DC nominations in 2025. The company is maintaining its guidance for the planned IND filing for hemophilia A in 2026. The updated milestones are available in the corporate presentation on the company website.

Upcoming Events
Metagenomi plans to participate in the following events during the third quarter of 2024:

  • Wells Fargo Healthcare Conference, September 4-6, 2024, Everett, Massachusetts
  • H.C. Wainwright 26th Annual Global Investment Conference, September 9-11, 2024, New York City
  • Chardan 8th Annual Genetics Medicines Conference, September 30-October 1, 2024, New York City

Second Quarter 2024 Financial Results

  • Cash Position: Cash, cash equivalents, and available-for-sale marketable securities were $299.9 million as of June 30, 2024.
  • R&D Expenses: Research and development (R&D) expenses were $28.3 million for the three months ended June 30, 2024, compared to $22.7 million for the three months ended June 30, 2023.
  • G&A Expenses: General and administrative (G&A) expenses were $8.6 million for the three months ended June 30, 2024, compared to $6.6 million for the three months ended June 30, 2023.

About Metagenomi

Metagenomi is a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary, comprehensive metagenomics-derived toolbox. Metagenomi is harnessing the power of metagenomics, the study of genetic material recovered from the natural environment, to unlock four billion years of microbial evolution to discover and develop a suite of novel editing tools capable of correcting any type of genetic mutation found anywhere in the genome. Its comprehensive genome editing toolbox includes programmable nucleases, base editors, and RNA and DNA-mediated integration systems (including prime editing systems and clustered regularly interspaced short palindromic repeat associated transposases). Metagenomi believes its diverse and modular toolbox positions the company to access the entire genome and select the optimal tool to unlock the full potential of genome editing for patients. For more information, please visit https://​metageno​mi​.co.

Cautionary Note Regarding Forward‐​Looking Statements

This press release contains ​“forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements, which are often indicated by terms such as ​“anticipate,” ​“believe,” ​“could,” ​“estimate,” ​“expect,” ​“goal,” ​“intend,” ​“look forward to,” ​“may,” ​“plan,” ​“potential,” ​“predict,” ​“project,” ​“should,” ​“will,” ​“would” and similar expressions, include, but are not limited to, any statements relating to our growth strategy and product development programs, including the timing of and our ability to conduct IND-enabling studies, make regulatory filings such as INDs, statements concerning the potential of therapies and product candidates, statements concerning the timing of data presentations and publications, and any other statements that are not historical facts. Forward looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition, and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in ​“Risk Factors,” in our most recent Form 10-K and our most recent 10-Qs on file with the Securities and Exchange Commission. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.

Investor Contact:
Simon Harnest – CIO, SVP Investor Relations
simon@​metagenomi.​co

Media Contact:
Ashlye Hodge – Communications Manager
ashlye@​metagenomi.​co

SOURCE: Metagenomi

Primrose Bio

Synthetic biology and manufacturing company developing next-generation solutions for new therapeutics and vaccines.

Q32 Bio

Developing targeted biotherapeutics that rebalance the immune system.

Cerevance

Developing treatments derived from proprietary discovery platform for CNS disorders and neurodegenerative diseases.

AshiBio

Developing novel therapies for patients with rare bone and connective tissue disorders.

Palvella Therapeutics and Pieris Pharmaceuticals Announce Definitive Merger Agreement

Proposed merger to create a Nasdaq-listed, clinical-stage biopharmaceutical company focused on developing and commercializing novel treatments for serious, rare genetic skin diseases for which there are no FDA-approved therapies

Combined company is expected to have approximately $80.5 million of cash and cash equivalents at closing, including approximately $78.9 million from an oversubscribed private financing with participation from a syndicate of leading healthcare-dedicated investors, which is expected to provide cash runway into the second half of 2027

Anticipated cash and cash equivalents are expected to fund combined company through multiple clinical trial milestones, including pivotal Phase 3 clinical trial results from a single-arm, baseline-controlled clinical trial of QTORIN™3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of microcystic lymphatic malformations currently being conducted under FDA’s Breakthrough Therapy Designation and Fast Track Designation programs

QTORIN™ rapamycin has the potential to be the first approved therapy and standard of care in the U.S. for microcystic lymphatic malformations and cutaneous venous malformations, if approved

Companies to host joint webcast on Wednesday, July 24 at 8:30am ET

WAYNE, PA AND BOSTON, MA / ACCESSWIRE / July 24, 2024 / Palvella Therapeutics, Inc. (Palvella), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, and Pieris Pharmaceuticals, Inc. (Nasdaq: PIRS) (Pieris) today announced they have entered into a definitive merger agreement to combine the companies in an all-stock transaction. The combined company will focus on developing and commercializing Palvella’s lead clinical product candidate, QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), for the treatment of microcystic lymphatic malformations (microcystic LMs), cutaneous venous malformations, and other serious, functionally debilitating skin diseases driven by the overactivation of the mammalian target of rapamycin (mTOR) pathway. Upon completion of the proposed merger, the combined company will operate under the name Palvella Therapeutics, Inc., will be headquartered in Wayne, PA, and is expected to trade on The Nasdaq Capital Market (Nasdaq).

In connection with the proposed merger, Palvella has secured commitments from a syndicate of leading healthcare-dedicated investors in an oversubscribed $78.9 million concurrent private financing co-led by BVF Partners, L.P., an existing investor, and Frazier Life Sciences, a new investor. Additional new investors include Blue Owl Healthcare Opportunities, Nantahala Capital, DAFNA Capital Management, ADAR1 Capital Management, and a healthcare dedicated fund. Existing investors Samsara BioCapital, Petrichor, CAM Capital, Ligand Pharmaceuticals (Nasdaq: LGND), Integrated Finance Group (an AscellaHealth partner company), BioAdvance, and Gore Range Capital also committed to participate in the financing. The concurrent private financing includes approximately $18.9 million in principal and interest from Palvella convertible notes that will be funded prior to the close and convert into shares of common stock of the combined company. The concurrent private financing is expected to close immediately following the completion of the proposed merger.

The combined company is expected to have approximately $80.5 million of cash and cash equivalents at closing of the proposed merger and concurrent private financing, inclusive of the net proceeds expected to be received in the concurrent private financing and after deducting operating expenses incurred prior to closing and estimated transaction expenses. These cash resources are expected to be used to advance Palvella’s product candidate QTORIN™ rapamycin through multiple clinical data milestones and are expected to fund the combined company’s operations into the second half of 2027. The proposed merger and concurrent private financing are expected to close in the fourth quarter of 2024, subject to stockholder approval of both companies, the effectiveness of a registration statement to be filed with the U.S. Securities and Exchange Commission (SEC) to register the shares of Pieris common stock to be issued in connection with the merger, and the satisfaction of customary closing conditions. Pieris pre-merger stockholders will also be issued a contingent value right (CVR) representing the right to receive payments from proceeds received by the combined company, if any, under Pieris’ existing partnership agreements with Pfizer and Boston Pharmaceuticals.

“We are pleased to announce our merger with Pieris, allowing Palvella to become a publicly traded company and pursue our vision of becoming the leading rare disease company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases,” said Wes Kaupinen, Founder and Chief Executive Officer (CEO) of Palvella. “The expected proceeds from the merger and concurrent private financing are expected to fund us through multiple clinical trial milestones, including generating results from the single-arm, baseline-controlled Phase 3 clinical trial of QTORIN™ rapamycin for the treatment of microcystic lymphatic malformations, a serious, rare and chronically debilitating genetic disease for which there are currently no FDA-approved therapies.”

“This transaction represents Pieris’ deep commitment to delivering value to its stockholders by preserving the future potential milestone and royalty streams from our partnered immuno-oncology bispecifics franchise for Pieris legacy stockholders through the CVRs, while also providing the opportunity for upside in an attractive, late-stage, rare disease company,” said Stephen S. Yoder, President and CEO of Pieris. “With the anticipated funding and an accomplished management team, we believe Palvella is well-positioned to advance a Phase 3 clinical program with the FDA’s Breakthrough, Fast Track, and Orphan Drug Therapy Designations. This transaction is the culmination of a comprehensive review of strategic alternatives, and our board of directors believes that the merger with Palvella is in the best interests of our stockholders.”

Palvella’s QTORIN™ Platform and QTORIN™ rapamycin

Palvella’s research team developed QTORIN, a patented and versatile platform designed to generate novel topical therapies that penetrate the deep layers of the skin to locally treat a broad spectrum of serious, rare genetic skin diseases.

QTORIN™ rapamycin is the lead product candidate from Palvella’s QTORIN platform. QTORIN™ rapamycin is a novel, patented 3.9% rapamycin anhydrous gel, which aims to harness the potential therapeutic benefits of rapamycin, an mTOR inhibitor, while minimizing systemic exposure of rapamycin and potential adverse reactions associated with systemic therapy. QTORIN rapamycin is currently under development for the treatment of microcystic LMs, cutaneous venous malformations, and other serious, functionally debilitating skin diseases driven by the overactivation of the mammalian target of rapamycin (mTOR) pathway. QTORIN™ rapamycin is protected by multiple issued composition patents in the U.S. and Japan and pending patent applications broadly covering anhydrous gel formulations of rapamycin in the U.S., Europe, and Japan.

QTORIN™ Rapamycin for the Treatment of Microcystic LMs

Palvella initiated research on QTORIN™ rapamycin as a targeted therapy for microcystic LMs in 2017 based on scientific insights implicating abnormal activation of the mTOR pathway in this disease. Microcystic LMs is a rare, chronically debilitating genetic disease caused by dysregulation of the phosphatidylinositol 3-kinase (PI3K)/mTOR pathway. The disease is characterized by malformed lymphatic vessels that protrude through the skin and persistently leak lymph fluid (lymphorrhea) and bleed, often leading to recurrent serious infections and cellulitis. The natural history of microcystic LMs is progressive, with symptoms generally worsening during life, including increases in the number and size of malformed vessels that lead to complications and lifetime morbidity. There are currently no FDA-approved treatments for the estimated more than 30,000 diagnosed patients with microcystic LMs in the United States.

In November 2023, based on Phase 2 clinical trial results, Palvella received FDA Breakthrough Therapy Designation for QTORIN™ rapamycin for microcystic LMs. Palvella previously announced positive topline Phase 2 clinical trial results from the multi-center, open-label study of 12 subjects receiving QTORIN™ rapamycin once-daily for 12-weeks. The Phase 2 clinical trial featured multiple pre-specified efficacy assessments, including clinician and patient global impression assessments as well as assessments of individual clinical manifestations that are important disease burdens for individuals living with microcystic LMs. All participants in the Phase 2 clinical trial demonstrated improvements on the Clinician Global Impression of Change scale, with all participants in the study rated as either “Much Improved” (n=7, 58%) or “Very Much Improved” (n=5, 42%) after 12-weeks of treatment compared to the pre-treatment baseline period. In addition to Breakthrough Therapy Designation, the FDA has granted both Fast Track Designation and Orphan Drug Designation to QTORIN™ rapamycin for the treatment of microcystic LMs.

In February 2023, Palvella had an End of Phase 2 meeting and, in April 2024, a Type B Breakthrough Therapy Designation meeting with FDA regarding the clinical trial program. Palvella considered FDA feedback on study ethics and other considerations related to selection of key study design features, site feedback on study ethics and feasibility, and input from expert regulatory advisors, and Palvella initiated SELVA, a 24-week, pivotal Phase 3, single-arm, baseline-controlled clinical trial of QTORIN™ rapamycin for the treatment of microcystic LMs, in the third quarter of 2024. The study’s primary and key secondary endpoints are clinician-reported outcomes and will enroll 40 subjects at leading vascular anomaly centers across the U.S.

QTORIN™ Rapamycin for the Treatment of Cutaneous Venous Malformations

Palvella is also developing QTORIN™ rapamycin for the treatment of cutaneous venous malformations. Cutaneous venous malformations are a rare genetic disease that results from overactivation of the PI3K/mTOR signaling pathway in the development of the venous network, leading to dilated and dysfunctional veins within the skin. Cutaneous venous malformations cause functional impairment, significantly impact quality of life, and are associated with severe long-term complications. In April 2024, the FDA granted Fast Track Designation to QTORIN™ rapamycin for the treatment of venous malformations. Palvella plans to initiate a Phase 2 baseline-controlled clinical trial of QTORIN™ rapamycin for the treatment of cutaneous venous malformations in the second half of 2024.

About the Proposed Merger

Under the terms of the merger agreement, Pieris will issue shares of Pieris common stock to pre-merger Palvella stockholders as merger consideration in exchange for the cancellation of shares of capital stock of Palvella, and Palvella will become a wholly-owned subsidiary of Pieris.

Pre-merger Pieris stockholders are expected to own approximately 18% of the combined company and pre-merger Palvella stockholders are expected to own approximately 82% of the combined company, in each case, prior to the issuance of the shares under the concurrent private financing. The percentage of the combined company that pre-merger Palvella stockholders and pre-merger Pieris stockholders will own upon the closing of the merger is subject to further adjustment based on the amount of Pieris’ net cash at the time of closing. In connection with the closing of the proposed transactions under the merger agreement, Pieris pre-merger stockholders will also be issued a contingent value right (CVR) representing the right to receive payments from proceeds received by the combined company, if any, under Pieris’ existing partnership agreements with Pfizer and Boston Pharmaceuticals, in addition to other potential licensing agreements involving certain of Pieris’ legacy assets, as well as certain potential payments related to historical research and development tax credits, which may or may not be realized.

The transactions contemplated by the merger agreement have been unanimously approved by the boards of directors of both companies and are expected to close in the fourth quarter of 2024, subject to approvals by the stockholders of each company, the effectiveness of a registration statement to be filed with the SEC to register the shares of Pieris common stock to be issued in connection with the merger, and other customary closing conditions. Additional information about the transaction will be provided in a Current Report on Form 8-K that will be filed by Pieris with the SEC and will be available at www.sec.gov.

Management and Organization

Following the merger, the combined company will be led by Wes Kaupinen, Founder and CEO of Palvella, and other members of the Palvella management team. The combined company’s board of directors will be comprised of four of the current directors of Palvella’s board of directors, and one director designated from Pieris’ current board of directors, who is expected to be Christopher Kiritsy, the chair of Pieris’ audit committee. Pieris will be renamed “Palvella Therapeutics, Inc.”

Conference Call Information

The companies will host a webcast call and presentation to discuss the proposed transactions, as well as Palvella’s pipeline assets on Wednesday, July 24 at 8:30 am ET. The live webcast can be accessed here and on the Pieris website at www.pieris.com/investors in the ‘Investors’ section or by calling 877-407-8920 or +1 412-902-1010. A replay of the webcast will be archived and available following the event.

Advisors

TD Cowen is serving as lead placement agent and Cantor is serving as a placement agent for Palvella’s planned concurrent financing. Troutman Pepper Hamilton Sanders LLP is serving as legal counsel to Palvella. Cooley LLP is serving as legal counsel to the placement agents. Stifel is serving as the exclusive financial advisor to Pieris and Mintz, Levin, Cohn, Ferris, Glovsky, and Popeo, P.C. is serving as legal counsel to Pieris.

About Palvella Therapeutics

Founded and led by rare drug disease drug development veterans, Palvella Therapeutics is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no FDA-approved therapies. Palvella is developing a broad pipeline of product candidates based on its patented QTORIN™ platform, with an initial focus on serious, rare genetic skin diseases, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), is currently in clinical development for microcystic lymphatic malformations (microcystic LMs) and cutaneous venous malformations. QTORIN™ rapamycin has received FDA Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation for microcystic LMs, and Fast Track Designation for venous malformations. QTORIN™ rapamycin is for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency.

About Pieris Pharmaceuticals

Pieris is a biotechnology company based in Boston, Massachusetts that historically developed inhalable Anticalin proteins to treat respiratory diseases and locally-activated Mabcalin® (antibody-Anticalin®) bispecific proteins for immuno-oncology. Pieris’ pipeline consists of clinical stage 4-1BB bispecific proteins that are currently being developed by Pfizer (formerly Seagen) and Boston Pharmaceuticals, along with other pre-clinical programs under development with Pfizer. Pieris could potentially be entitled to receive development, regulatory, and sales-based milestones from its partnered 4-1BB bispecific immuno-oncology assets.

Forward-Looking Statements

This press release contains forward-looking statements (including within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended (Securities Act)) concerning Palvella, Pieris, the proposed transactions, and other matters. These statements may discuss goals, intentions, and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the management of Palvella and Pieris, as well as assumptions made by, and information currently available to, management of Palvella and Pieris. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Statements that are not historical facts are forward-looking statements. Forward-looking statements include, but are not limited to, expectations regarding the proposed merger transaction and concurrent private financing; the potential benefits and results of such transactions, including any potential benefits of the CVRs; the sufficiency of the combined company’s capital resources; the combined company’s cash runway; the expected timing of the closing of the proposed transactions; statements regarding the potential of, and expectations regarding, Palvella’s programs, including QTORIN™ rapamycin, and its research-stage opportunities, including its expected therapeutic potential and market opportunity; the expected timing of initiating, as well as the design of Palvella’s Phase 2 clinical trial of QTORIN™ rapamycin in cutaneous venous malformations; Pieris’ eligibility and potential to receive milestones from its partnered assets in connection with its contingent value rights; statements by Pieris’ President and CEO; and statements by Palvella’s Founder and CEO. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the limited operating history of each company; the significant net losses incurred since inception; the ability to raise additional capital to finance operations; the ability to advance product candidates through preclinical and clinical development; the ability to obtain regulatory approval for, and ultimately commercialize, Palvella’s product candidates, including QTORIN™ rapamycin; the outcome of early clinical trials for Palvella’s product candidates, including the ability of those trials to satisfy relevant governmental or regulatory requirements; the fact that data and results from clinical studies may not necessarily be indicative of future results; Palvella’s limited experience in designing clinical trials and lack of experience in conducting clinical trials; the ability to identify and pivot to other programs, product candidates, or indications that may be more profitable or successful than Palvella’s current product candidates; the substantial competition Palvella faces in discovering, developing, or commercializing products; the negative impacts of the global events on operations, including ongoing and planned clinical trials and ongoing and planned preclinical studies; the ability to attract, hire, and retain skilled executive officers and employees; the ability of Palvella and Pieris to protect their respective intellectual property and proprietary technologies; reliance on third parties, contract manufacturers, and contract research organizations; the risk that the conditions to the closing of the proposed transactions are not satisfied, including the failure to obtain stockholder approval for the proposed merger transaction from both Palvella’s and Pieris’ stockholders or to complete the proposed merger and concurrent private financing in a timely manner or at all; uncertainties as to the timing of the consummation of the proposed merger transaction and concurrent private financing transaction and the ability of each of the parties to consummate the proposed transactions; risks related to Pieris’ continued listing on Nasdaq until closing of the proposed transaction; risks related to Palvella’s and Pieris’ ability to correctly estimate their respective operating expenses and expenses associated with the proposed transaction, as well as uncertainties regarding the impact any delay in the closing would have on the anticipated cash resources of the combined company upon closing and other events and unanticipated spending and costs that could reduce the combined company’s cash resources; the occurrence of any event, change or other circumstance or condition that could give rise to the termination of the merger agreement or the concurrent private financing transaction; competitive responses to the proposed transactions; risks related to the likelihood that the holders of CVRs will be entitled to any future payments; unexpected costs, charges or expenses resulting from the proposed transactions; the outcome of any legal proceedings that may be instituted against Palvella, Pieris, or any of their respective directors or officers related to the merger agreement, the concurrent private financing, or the proposed transactions contemplated thereby; the effect of the announcement or pendency of the proposed transactions on Palvella’s and Pieris’ business relationships, operating results and business generally; and legislative, regulatory, political and economic developments and general market conditions. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in Pieris’ most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and Current Reports on Form 8-K filed with the SEC, as well as the registration statement on Form S-4 to be filed with the SEC by Pieris in connection with the merger. Palvella and Pieris can give no assurance that the conditions to the proposed transactions will be satisfied. Except as required by applicable law, Palvella and Pieris undertake no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.

This press release contains hyperlinks to information that is not deemed to be incorporated by reference into this press release.

No Offer or Solicitation

This press release is not intended to and does not constitute an offer to sell or the solicitation of an offer to subscribe for or buy or an invitation to purchase or subscribe for any securities or the solicitation of any vote in any jurisdiction pursuant to the proposed transaction or otherwise, nor shall there be any sale, issuance or transfer of securities in any jurisdiction in contravention of applicable law. No offer of securities shall be made except by means of a prospectus meeting the requirements of the Securities Act. Subject to certain exceptions to be approved by the relevant regulators or certain facts to be ascertained, the public offer will not be made directly or indirectly, in or into any jurisdiction where to do so would constitute a violation of the laws of such jurisdiction, or by use of the mails or by any means or instrumentality (including without limitation, telephone and the internet) of interstate or foreign commerce, or any facility of a national securities exchange, of any such jurisdiction.

NEITHER THE SEC NOR ANY STATE SECURITIES COMMISSION HAS APPROVED OR DISAPPROVED OF THE SECURITIES OR DETERMINED IF THIS PRESS RELEASE IS TRUTHFUL OR COMPLETE.

Important Additional Information About the Proposed Transactions Will be Filed with the SEC

In connection with the proposed transaction between Pieris and Palvella, Pieris intends to file relevant materials with the SEC, including a registration statement on Form S-4 that will contain a proxy statement and prospectus of Pieris and an information statement of Palvella. PIERIS URGES INVESTORS AND STOCKHOLDERS TO READ THESE MATERIALS CAREFULLY AND IN THEIR ENTIRETY WHEN THEY BECOME AVAILABLE, AS WELL AS ANY AMENDMENTS OR SUPPLEMENTS TO THESE MATERIALS, BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION ABOUT PIERIS, PALVELLA, THE PROPOSED TRANSACTION AND RELATED MATTERS. Investors and stockholders will be able to obtain free copies of the proxy statement/prospectus/information statement and other documents filed by Pieris with the SEC (when they become available) through the website maintained by the SEC atwww.sec.gov. In addition, investors and stockholders will be able to obtain free copies of the proxy statement/prospectus/information statement and other documents filed by Pieris with the SEC free of charge on Pieris’ website at www.pieris.com, or by contacting Investor Relations by email at [email protected]. Investors and stockholders are urged to read the proxy statement/prospectus/information statement and the other relevant materials when they become available before making any voting or investment decision with respect to the proposed transaction.

Participants in the Solicitation

Palvella, Pieris and their respective directors and executive officers may be considered participants in the solicitation of proxies in connection with the proposed transaction. Information about Pieris’ directors and executive officers is included in Pieris’ most recent Annual Report on Form10-K, as amended, including any information incorporated therein by reference, as filed with the SEC on March 29, 2024, and amended on April 29, 2024. Additional information regarding the persons who may be deemed participants in the solicitation of proxies will be included in the proxy statement/prospectus/information statement relating to the proposed transaction when it is filed with the SEC. These documents can be obtained free of charge from the sources indicated above.

Palvella Therapeutics Contact Information

Investors
Wesley H. Kaupinen
Founder and CEO, Palvella Therapeutics
[email protected]

Media
Marcy Nanus
Managing Partner, Trilon Advisors, LLC
[email protected]

Pieris Pharmaceuticals Contact Information
Investors
[email protected]

SOURCE: Pieris Pharmaceuticals, Inc.

Vittoria Biotherapeutics Announces Peer-Reviewed Publication in Science Immunology

PHILADELPHIA, July 22, 2024 (GLOBE NEWSWIRE) — Vittoria Biotherapeutics,  a clinical-stage cell therapy company, announces a recent publication by investigators at the University of Pennsylvania in the peer-reviewed journal, Science Immunology. The publication details the critical influence of CD5, a key immunomodulatory protein, on engineered T-cell therapies, which is the foundational basis of the company’s proprietary Senza5TM platform. This technology, developed by the University of Pennsylvania and exclusively licensed to Vittoria, leverages the therapeutic potential of CD5 modulation to enhance the efficacy of CAR T-cell therapies and underscores its utility across multiple hematological and solid tumor animal models.

“We are incredibly excited about the findings shared in Science Immunology, which support the clinical translation of our Senza5TM platform and exemplify Vittoria’s commitment to pioneering potent and innovative solutions to advance the field of enabled, autologous cell therapies,” said Nicholas Siciliano, Ph.D., Chief Executive Officer of Vittoria Biotherapeutics. “Despite significant advances in cell therapy across various diseases, considerable unmet needs remain for certain cancers and chronic diseases. This publication comes at a pivotal time as we initiate the clinical trial for our lead program, VIPER-101 for the treatment of T-cell lymphoma.”

The research was conducted in the laboratory of Marco Ruella, M.D., a Physician-Scientist in the Perelman School of Medicine at the University of Pennsylvania, who is also the scientific founder of Vittoria Biotherapeutics. Dr. Ruella, the senior author of the study, added, “I am honored to see our seminal study on the role of CD5 in engineered cell therapies published in Science Immunology. The study highlights the ongoing innovation and continued commitment to advance the field of cell therapy and cancer research at the University of Pennsylvania. Recognizing the transformative potential of this technology, we founded Vittoria to accelerate its translation into the clinic. The first clinical candidate developed from this research is now being evaluated in a first-in-human clinical trial at Penn Medicine as a potential new treatment for T-cell lymphoma, a disease with significant unmet therapeutic need.”

The preclinical study, titled “CD5 Deletion Enhances the Anti-Tumor Activity of Adoptive T Cell Therapies,” describes the benefits of the approach of modulating the CD5 signaling pathway and utilizing a proprietary five-day manufacturing process to enhance CAR T potency, durability, and accessibility. These features may address existing challenges with current CAR T-cell treatments including CAR T exhaustion, suboptimal efficacy, and manufacturing inefficiencies. To learn more in-depth about these approaches, which underline Vittoria’s Senza5TM platform, the full publication is available here: https://www.science.org/doi/10.1126/sciimmunol.adn6509.

Ruchi Patel, Ph.D., the lead author of the study and former graduate student in the Ruella laboratory at the University of Pennsylvania who recently joined Vittoria’s scientific team, added, “I am thrilled to see our work published in such a prestigious journal, and excited to contribute to the clinical translation of this groundbreaking technology in my new role at Vittoria. This brings us one step closer to enabling the next generation of transformative cell therapies and potentially improving patient outcomes.”

Vittoria’s VIPER-101 clinical trial is now open for enrollment for the treatment of T-cell lymphoma. Information about the trial can be found at: https://clinicaltrials.gov/study/NCT06420089?a=1. Initial clinical data is expected in early 2025 and will provide valuable insights into the Senza5 platform technology, paving the way for more effective cell therapies that can transform the treatment landscape for patients with cancer and autoimmune diseases.

Editor’s Note: Ruella is the scientific founder of, a paid consultant for, and an equity holder in, Vittoria Biotherapeutics. The University of Pennsylvania holds equity in Vittoria Biotherapeutics, has received sponsored research funding from Vittoria, has licensed certain intellectual property to Vittoria and may receive future research funding and financial consideration based on development and commercialization of certain products by Vittoria.

About Senza5TM

Senza5TM is a proprietary cell therapy engineering and manufacturing platform that combines the power of genetic engineering and a proprietary five-day manufacturing process to maximize stemness, durability, and efficacy of its produced cell therapies by disabling the CD5 signaling pathway on engineered CAR T cells and bypassing CD5’s immunosuppressive effects to amplify the therapy’s antitumor activity. The expedited five-day manufacturing process further enhances stemness, which promotes greater in vivo expansion and durability and the potential for longer-lasting responses. Senza5 can be widely utilized to improve the efficacy of engineered T-cell therapies by acting on the fundamental biology of T cells.

About Vittoria Biotherapeutics

Vittoria Biotherapeutics, Inc., a clinical-stage cell therapy company, is developing novel CAR T-cell therapies that transcend the limitations of current cell therapies. Based on technology exclusively licensed from the University of Pennsylvania, the Company’s proprietary Senza5TM platform unlocks the cytotoxic potential of engineered T cells and utilizes a five-day manufacturing process to maximize stemness, durability, and potency. By acting on the fundamental biology of T cells, Senza5 can be used to improve the efficacy of engineered T-cell therapies with pipeline applications in oncology and autoimmune diseases. To learn more, visit vittoriabio.com and follow us on LinkedIn.

Investor Contact

Vittoria Biotherapeutics, Inc.
Nicholas A. Siciliano, Ph.D.
Chief Executive Officer
+1 215-600-1380

Media Contact

LifeSci Communications
Michael Tattory
Associate Director
LifeSci Communications
+1 609-802-6265

SOURCE: Vittoria Biotherapeutics