Skyhawk’s SKY-0515 demonstrated dose-dependent huntingtin (HTT) mRNA reduction in healthy volunteers, with 72% reduction at the highest dose tested in the multiple ascending dose study. SKY-0515 was generally well tolerated at all doses tested. Given these positive topline results, we anticipate dosing in the patient arm of the study in Q3 2024 and initiation of a Phase 2 study early next year
BOSTON, MA., July 10, 2024 – Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company developing novel small molecule therapies designed to modulate critical RNA targets, today announced positive results from Parts A and B of its Phase 1 clinical trial of SKY-0515, which is being developed as a potential treatment for Huntington’s disease (HD). SKY-0515 demonstrated an average HTT mRNA reduction of 72% at a daily oral dose of 9mg and was generally well tolerated at all doses tested.
SKY-0515 is Skyhawk’s investigational small molecule RNA splicing modifier developed through the company’s novel RNA-splicing platform. SKY-0515 is designed to reduce both HTT protein and PMS1 protein, an additional key driver of somatic CAG repeat expansion and HD pathology.
“Huntingtin-lowering and somatic expansion have been two of the hottest targets in HD research in the past decade. Reducing both HTT and PMS1 could have greater therapeutic benefit than lowering HTT alone,” said Ed Wild, Professor of Neurology at University College London. “Huntington’s disease is a rare hereditary neurodegenerative disease affecting over 40,000 patients in the United States. There are no approved treatments that can reverse or slow its course of progression. SKY-0515’s HTT reduction has the highest dynamic range I’ve seen from any therapeutic modality and gives me great hope for SKY-0515’s potential to one day help those patients in need.”
“We believe that, with these impressive HTT mRNA lowering results and the drug’s predicted suppression of the PMS1 protein, SKY-0515, if approved, could make a meaningful difference in Huntington’s patients’ lives,” said Douglas V. Faller, M.D., Ph.D., Chief Medical Officer, Skyhawk Therapeutics. “The Safety Review Committee has determined that SKY-0515 has been generally well tolerated at all tested doses with a dose proportional increase in systemic exposure and, given these favorable safety results, approved this study to move into the patient arm. Recruitment has begun, and topline data from this part of the trial are expected to report in Q2 2025.”
“After initiating this Phase 1 clinical trial in late 2023, we’re delighted with the speed at which we’ve conducted this study and thrilled to report such compelling results for SKY-0515,” said Clint Musil, Chief Executive Officer, Skyhawk Therapeutics. “These topline data represent a crucial step forward for SKY-0515 and demonstrate the immense potential of the Skyhawk platform to target indications for which there are no approved disease modifying therapies.”
HTT mRNA levels in the blood from pre-dose are described in the charts below.
About SKY-0515 Phase 1 Clinical Study
SKY-0515 is currently being evaluated in a Phase 1 clinical trial. The Phase 1 clinical trial is a first-in-human trial designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics, specifically blood biomarker modulation activity, of SKY-0515 in healthy volunteers and individuals with early-stage Huntington’s disease (HD). The trial is separated into three parts. Parts A and B evaluated SKY-0515 in healthy volunteers.
Part A was a double-blind placebo-controlled single ascending dose study in healthy adult volunteers. In Part A, five cohorts of participants were dosed with escalating single doses of SKY-0515 ranging from 1mg to 16mg or placebo. Additionally, the influence of food on the pharmacokinetics of SKY-0515 was examined in a dedicated cohort.
Part B was a double-blind placebo-controlled multiple ascending dose study in healthy adult volunteers. In Part B, three cohorts of participants were randomized to receive multiple ascending doses of SKY-0515 ranging from 1mg to 9mg or placebo administered daily from days 1 to 14 (inclusive). Dose levels of SKY-0515, identified in Parts A and B, will be evaluated in Part C.
Part C is a double-blind placebo-controlled parallel design study of two dose levels of SKY-0515 and placebo of individuals with early-stage HD (HD-ISS Stage 1, 2, or mild Stage 3) preceded by an observational period lasting a minimum of 28 days, which aims to evaluate pharmacodynamic parameters such as mutant HTT protein and mRNA. Recruitment for Part C has begun, and topline data are expected in Q2 2025.
About Skyhawk Therapeutics
Skyhawk Therapeutics is a clinical-stage biotechnology company focused on the discovery and development of novel small molecule therapies designed to modulate critical RNA targets and revolutionize patient treatment for some of the world’s most intractable diseases. Skyhawk’s discovery expertise is rooted in its proprietary drug discovery platform, which assesses, identifies, and tests RNA splicing targets and small molecules across a broad range of therapeutic areas and disease states. Skyhawk has built collaborations with multiple pharma partners that leverage Skyhawk’s novel platform across disease areas including neurodegenerative disease, autoimmune disease, and oncology. For more information visit www.skyhawktx.com.
Skyhawk Company Contact
Maura McCarthy, SVP Market Development
Investor Contact
AnneMarie Fields, Stern Investor Relations
332-213-1956
SOURCE Skyhawk Therapeutics, Inc.
Will initiate a first-in-human Phase 1 clinical trial for INT2104, its first-in-class in vivo targeted gene therapy, creating CAR cells for the treatment of B cell malignancies in 4Q24
PHILADELPHIA, July 9, 2024 /PRNewswire/ — Interius BioTherapeutics, a leading developer of in vivo cell-specific gene medicines, today announced that it has been granted Human Research Ethics Committee (HREC) approval and Clinical Trial Notification (CTN) clearance by the Australian Therapeutic Goods Administration (TGA) to commence a first-in-human Phase 1 clinical trial of INT2104, its lead in vivo CAR candidate for treatment of B-cell malignancies.
“Receiving HREC approval and CTN clearance for our first clinical trial is a significant milestone for Interius. We are very pleased that the regulators have approved the start of our first-in-human clinical trial for INT2104. The approval allows us to enroll patients in our first clinical study and recognizes the potential of our novel in vivo targeted gene therapy candidate, INT2104, to address an unmet medical need for patients with B cell malignancies,” said Interius President and Chief Executive Officer Phil Johnson, M.D. “We look forward to continuing to work closely with the TGA and other regulators in the future to bring this innovative therapy to patients as quickly as possible.”
The CTN clearance was granted based on HREC approval in Australia which included extensive review of Interius’s preclinical data and study protocol. Interius intends to begin the trial in the fourth quarter of 2024 and is well positioned to deliver key program milestones as early as the first quarter of 2025.
About the INT2104 Clinical Program
Interius’s Phase 1 trial (INVISE, Injectable Vectors for In Situ Engineering) will evaluate the safety of a single INT2104 infusion in adults with refractory/relapsing B cell malignancies. The Phase 1 study is a global, two-part, multicenter, open-label, single dose design with a dose escalation portion designed to inform the dose of INT2104 to be used in the dose confirmation part of the trial and future studies.
About INT2104
INT2104 is a wholly-owned investigational gene therapy candidate, which specifically targets CD7-positive T and NK cells and delivers a CAR transgene to create effector CAR-T and CAR-NK cells in vivo. The CAR cells target CD20-positive B cells for the treatment of B cell malignancies. Unlike ex vivo CAR-T therapies, INT2104 is an off-the-shelf, single dose treatment, administered systemically through intravenous infusion without the need for lymphodepletion or for any special equipment or training.
About Interius BioTherapeutics
Interius BioTherapeutics, a leading biotechnology company, is developing novel off-the-shelf gene therapies enabling the generation of autologous chimeric antigen receptor (CAR) cells in vivo using targeted lentiviral vector technology. The company’s lead program is an intravenous in vivo CAR therapy to treat B cell lymphomas, in which proprietary engineering delivers exquisite specificity for target tissues. The company is developing a second program to treat autoimmune diseases. Interius has created a differentiated new therapeutic modality for precision delivery of gene medicines, which could be available to patients without delays, without preconditioning chemotherapy, and in expanded care settings. For more information, visit www.interiusbio.com.
SOURCE Interius BioTherapeutics, Inc.
« Back Carisma Therapeutics Announces Nomination of First In Vivo CAR-M Development Candidate for Hepatocellular Carcinoma Under Collaboration with ModernaDevelopment Candidate targets Glypican-3 for the treatment of solid tumors, including hepatocellular carcinoma
Nomination triggers a $2 million milestone payment to Carisma
PHILADELPHIA, June 27, 2024 /PRNewswire/ — Carisma Therapeutics Inc. (Nasdaq: CARM) (“Carisma” or the “Company”), a clinical stage biopharmaceutical company focused on discovering and developing innovative immunotherapies, today announced the nomination of the first development candidate (“Development Candidate”), under its collaboration with Moderna, Inc. The Development Candidate is an in vivo CAR-M targeting Glypican-3 (“GPC3”) and is designed to treat solid tumors, including hepatocellular carcinoma (“HCC”), the most prevalent type of liver cancer and the fastest-rising cause of cancer-related death in the U.S. This nomination triggers a $2 million milestone payment to Carisma.
The nomination of this Development Candidate leverages Carisma’s expertise in engineering chimeric antigen receptor monocytes and macrophages (“CAR-M”) with Moderna’s mRNA and lipid nanoparticle platform to create a novel in vivocell therapy for oncology. Pre-clinical data demonstrated that the Development Candidate can successfully create CAR-M directly in vivo, redirecting endogenous myeloid cells to attack cancer cells.
“The nomination of the first Development Candidate underscores our productive collaboration with Moderna to develop mRNA-based in vivo CAR-M cell therapies,” said Michael Klichinsky, PharmD, PhD, Co-Founder and Chief Scientific Officer at Carisma. “The Development Candidate targets GPC3, a tumor antigen that is highly expressed in HCC. This milestone represents a significant step forward in the development of immunotherapies for solid tumors, and an advance for the in vivo cell therapy field.”
Lin Guey, PhD, Chief Scientific Officer of Therapeutics Research Ventures and Biotherapeutics at Moderna, stated, “We are thrilled with the significant progress we’ve made in advancing in vivo CAR-M therapies alongside Carisma. We eagerly anticipate further development of the nominated candidate for patients with solid tumors, and we look forward to continued success as we develop additional in vivo CAR-M together to bring new therapies to patients with HCC and other cancers.”
The in vivo CAR-M program targeting GPC3 is being developed under the 2022 strategic collaboration agreement between Carisma and Moderna to discover, develop, and commercialize in vivo engineered CAR-M therapeutics for the treatment of cancer. In addition to this program, Moderna has nominated four undisclosed oncology research targets under the collaboration. Carisma is responsible for the discovery and optimization of development candidates, while Moderna will lead the development and commercialization of resulting therapeutics.
About Carisma Therapeutics
Carisma Therapeutics Inc. is a clinical stage biopharmaceutical company focused on utilizing our proprietary macrophage and monocyte cell engineering platform to develop transformative immunotherapies to treat cancer and other serious diseases. We have created a comprehensive, differentiated proprietary cell therapy platform focused on engineered macrophages and monocytes, cells that play a crucial role in both the innate and adaptive immune response. Carisma is headquartered in Philadelphia, PA. For more information, please visit www.carismatx.com.
Cautionary Note on Forward-Looking Statements
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to Carisma’s business, strategy, future operations, cash runway, the advancement of Carisma’s product candidates and product pipeline, and clinical development of Carisma’s product candidates, including expectations regarding timing of initiation and results of clinical trials. The words “anticipate,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “goals,” “intend,” “may,” “might,” “outlook,” “plan,” “project,” “potential,” “predict,” “target,” “possible,” “will,” “would,” “could,” “should,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.
Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, (i) Carisma’s ability to realize the anticipated benefits of its pipeline reprioritization and corporate restructuring, (ii) Carisma’s ability to obtain, maintain and protect its intellectual property rights related to its product candidates; (iii) Carisma’s ability to advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials and with its current financial and human resources; (iv) Carisma’s ability to replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; (v) Carisma’sability to realize the anticipated benefits of its research and development programs, strategic partnerships, research and licensing programs and academic and other collaborations; (vi) regulatory requirements or developments and Carisma’sability to obtain and maintain necessary approvals from the U.S. Food and Drug Administration and other regulatory authorities related to its product candidates; (vii) changes to clinical trial designs and regulatory pathways; (viii) risks associated with Carisma’s ability to manage expenses; (ix) changes in capital resource requirements; (x) risks related to the inability of Carisma to obtain sufficient additional capital to continue to advance its product candidates and its preclinical programs; and (xi) legislative, regulatory, political and economic developments.
For a discussion of these risks and uncertainties, and other important factors, any of which could cause Carisma’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” set forth in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, its Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, as well as discussions of potential risks, uncertainties, and other important factors in Carisma’s other recent filings with the Securities and Exchange Commission. Any forward-looking statements that are made in this press release speak as of the date of this press release. Carisma undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.
Investors:
Shveta Dighe
Head of Investor Relations
[email protected]
Media Contact:
Julia Stern
(763) 350-5223
[email protected]
Source: Carisma Therapeutics Inc.
āshibio Raises $40M in Seed and Series A Financing to Advance Therapies for Bone and Connective Tissue Disorders– First clinical program focuses on fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder causing extra-skeletal bone formation –
– Experienced leadership team poised to accelerate pipeline development with lead asset based on novel research –
BRISBANE, Calif. (June 20, 2024) – āshibio, a privately held biotechnology company developing novel therapeutics for the treatment of bone and connective tissue disorders, exited stealth mode today with $40 million in seed and Series A financing. The company was founded in 2022 by Chief Executive Officer Pankaj Bhargava, M.D., and the team at MPM BioImpact, where Dr. Bhargava is also an entrepreneur partner. MPM BioImpact led the Series A round, with contributing funds from Agent Capital, YK Bioventures, and Mirae Asset Venture Investment. In conjunction with the financing, Agent Capital Partner and Co-Founder Preston Noon has joined the Board of Directors.
The funding will allow āshibio to continue to advance development of several investigative therapies 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. FOP typically starts in early childhood, is characterized by episodic and painful flare-ups, and leads to severe debilitation and shortened life span.
“We are building a pipeline of innovative therapeutics that leverage emerging biology and novel mechanisms to impact bone and connective tissue disorders that have no approved treatments or where current treatment options are inadequate,” said Dr. Bhargava. “We are grateful to our investors for recognizing the promise of our approach and for supporting our development programs.”
The company’s lead program is based on the discovery that the matrix metalloproteinase-9 (MMP-9) enzyme could serve as a novel target for FOP, as published in Journal of Bone and Mineral Research in February 2024.
The research described the case of a unique 35-year-old patient who carries the classic genetic mutation of FOP (ACVR1 R206H, present in over 95% of patients), yet has extreme lack of heterotopic ossification (HO) and near normal mobility. The authors found that the patient has an MMP-9 mutation in addition to the FOP mutation that appears to protect him from flare-ups and the abnormal bone formation typically seen in individuals with FOP. Further studies with MMP-9 gene knockout and pharmacological experiments confirmed that MMP-9 may serve as a novel target in FOP and other more common forms of HO.
āshibio licensed andecaliximab, a humanized antibody that specifically inhibits MMP-9, from Gilead Sciences, Inc. Andecaliximab has been administered to approximately 1,000 patients in previous clinical trials.
āshibio plans to initiate a Phase 2/3 trial of andecaliximab in the second half of 2024 in patients with FOP. The U.S. Food and Drug Administration (FDA) designated andecaliximab an Orphan Drug for the treatment of FOP in March 2024, one month after the European Medicines Agency (EMA) issued its own Orphan Drug Designation for the therapy. The FDA also cleared āshibio’s Investigational New Drug (IND) application for andecaliximab in March 2024.
Experienced senior leadership team announced
Prior to joining MPM BioImpact, Dr. Bhargava was the Oncology Therapeutic Area Head at Gilead Sciences. He has also held executive positions at multiple biotech companies during his career, including Sanofi and Dicerna Pharmaceuticals, where he led development programs for oncology and rare genetic diseases.
In addition to completion of the Series A funding, āshibio also announced the following appointments to its senior leadership team:
- Victoria Smith, Ph.D., joins āshibio as Chief Scientific Officer. She has worked across several therapeutic areas including oncology, and autoimmune and inflammatory diseases. She has previously held executive or senior positions at Amphivena Therapeutics, Gilead Sciences, and Arresto Biosciences, where she was a co-inventor of andecaliximab.
- Deborah Wenkert, M.D., joins āshibio as Chief Medical Officer. She is an experienced pediatric rheumatologist, specializing in rare bone and connective tissue disorders, with extensive experience in both academic and industry settings. She has previously held executive or clinical development positions at Inozyme Pharma, PreciThera, and Amgen.
“At āshibio, we are rapidly advancing novel therapeutics for severe and debilitating bone and connective tissue disorders, an area of great clinical need,” commented Dr. Smith. “It is extremely gratifying to be a part of such an impressive team of seasoned industry executives, clinicians, and researchers who are devoted to improving the lives of patients who have not been adequately served by currently available treatment options.”
“āshibio is clearly at the forefront of drug development in heterotopic ossification and other bone and connective tissue pathologies,” added Todd Foley, managing partner at MPM BioImpact and Chair of the āshibio Board of Directors. “The company is now on a clinical path for its lead asset and is poised to advance a pipeline of programs that address unmet needs.”
āshibio researchers will present an update on the andecaliximab FOP program in an oral and poster presentation at the FOP Drug Development Forumin Stockholm, Sweden on June 27-28, 2024. Company leadership will also present a poster at the International Conference on Children’s Bone Healthin Salzburg, Austria from June 22-25, 2024.
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About āshibio
āshibio is a privately held 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.
About MPM BioImpact
MPM BioImpact is a world-leading biotechnology investment firm with over 30 years of expertise creating and investing in innovative companies to deliver transformative therapies to patients. Its experienced and dedicated team of investment professionals, entrepreneurs, advisors and leading scientists translate scientific discoveries into breakthrough medicines and potential cures. For more information, visit www.mpmbioimpact.com.
About Agent Capital
Agent Capital is an international life sciences investment firm that supports disruptive healthcare companies focusing on novel, differentiated therapeutics and treatments that address unmet patient needs. Agent Capital aligns with scientists, entrepreneurs, and other investors to develop the next generation of healthcare innovations, leverages their industry expertise and successful track record to source premier deals, accelerate value, and drive successful exits. Since 2017, Agent Capital has invested in over 25 companies, the majority of which have executed collaborations with major pharmaceutical companies or successfully raised additional capital in the private or public markets. For more information, visit www.agentcapital.com.
Media Contact:
SmithSolve
Matt Pera
(219) 628-0258
Source: āshibio