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TwoStep Therapeutics Secures $62.5 Million Series A Funding and FDA Clearance for Cancer Drug

TwoStep Therapeutics Secures $62.5 Million Series A Funding and FDA Clearance for Cancer Drug

TwoStep Therapeutics Secures $62.5 Million Series A Funding and FDA Clearance for Cancer Drug
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Rocket Pharmaceuticals Aligns with FDA on Pivotal Phase 2 Trial for Danon Disease Treatment

Rocket Pharmaceuticals Aligns with FDA on Pivotal Phase 2 Trial for Danon Disease Treatment

Rocket Pharmaceuticals Aligns with FDA on Pivotal Phase 2 Trial for Danon Disease Treatment
ReutersReuters
Experts urge FDA to set clear safety and access standards for psychedelic drugs

Experts urge FDA to set clear safety and access standards for psychedelic drugs

By Puyaan Singh and Kamal Choudhury Sept 14 (Reuters) - Doctors, veterans, pharmacists and therapists on Monday urged the U.S.
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Coya Therapeutics Reaches 100th Participant Enrollment in ALS Trial for COYA 302

Coya Therapeutics Reaches 100th Participant Enrollment in ALS Trial for COYA 302

Coya Therapeutics, Inc., a clinical-stage biotechnology company, has announced the enrollment of its 100th participant in the Phase 2/3 ALSTARS Trial (NCT07161999) for COYA 302. This investigational drug, a combination of proprietary low-dose IL-2 and CTLA-4 Ig, is being evaluated for the treatment of amyotrophic lateral sclerosis (ALS). The company anticipates completing the full enrollment of 120 participants in the coming weeks, with topline data expected in early Q2 2027. COYA 302 has received FDA Fast Track designation for ALS treatment. The ALSTARS Trial is a randomized, multi-center, double-blind, placebo-controlled study designed to assess the efficacy and safety of COYA 302. Participants are randomized to receive one of two dosing regimens or a placebo over a 24-week double-blind treatment phase, followed by a 24-week blinded active-treatment extension phase. The primary endpoint focuses on changes in disease severity as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised...
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Tenaya Therapeutics Receives FDA Regenerative Medicine Advanced Therapy Designation for TN-401

Tenaya Therapeutics Receives FDA Regenerative Medicine Advanced Therapy Designation for TN-401

Tenaya Therapeutics, a clinical-stage biotechnology company, has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) for its gene therapy candidate, TN-401. This designation is for the treatment of PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), a serious heart disease. The RMAT designation is an expedited program designed to facilitate the development and review of regenerative medicine therapies for severe conditions where preliminary clinical evidence suggests the potential to address unmet medical needs. Tenaya's RMAT application included data from the RIDGE-1 clinical trial, which showed improvements in electrical stability measures, such as reduced rates of premature ventricular contractions (PVCs) and non-sustained ventricular tachycardias (NSVTs), common forms of ventricular arrhythmias in ARVC. The company's CEO, Faraz Ali, highlighted that this designation underscores the recognition of the seriousness of PK...
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FDA Accepts Curi Bio's 3D Neuromuscular Junction Assay for ISTAND Program, Advancing Non-Animal Testing for Botulinum Toxin

FDA Accepts Curi Bio's 3D Neuromuscular Junction Assay for ISTAND Program, Advancing Non-Animal Testing for Botulinum Toxin

FDA Accepts Curi Bio's 3D Neuromuscular Junction Assay for ISTAND Program, Advancing Non-Animal Testing for Botulinum Toxin
ReutersReuters
FDA holds public hearing on psychedelic drugs as Trump-driven push gathers pace

FDA holds public hearing on psychedelic drugs as Trump-driven push gathers pace

By Puyaan Singh and Kamal Choudhury Sept 14 (Reuters) - The U.S.
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Thryv Therapeutics Publishes Evidence Supporting THRV-1268 for Heart Failure Treatment

Thryv Therapeutics Publishes Evidence Supporting THRV-1268 for Heart Failure Treatment

Thryv Therapeutics Inc. has announced the publication of a new study in JACC: Heart Failure, presenting human genetic, preclinical, and clinical evidence that supports the use of its lead SGK1 inhibitor, THRV-1268, for treating heart failure with reduced ejection fraction (HFrEF). The study highlights SGK1 as a genetically validated cardiometabolic therapeutic target. Key findings indicate that THRV-1268 maintained cardiac function and outperformed empagliflozin monotherapy in an animal model of heart failure. Additionally, Phase 1 studies demonstrated a favorable safety profile and statistically significant QTcF shortening in individuals with obesity, consistent with cardiac target engagement. These findings provide a strong rationale for advancing THRV-1268 into Phase 2a clinical development through the ASPIRE-HF study, which is expected to begin enrollment in Q4 2026.
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Diasome Pharmaceuticals Strengthens Leadership Team Ahead of Phase 3 Development for HDV-Insulin

Diasome Pharmaceuticals Strengthens Leadership Team Ahead of Phase 3 Development for HDV-Insulin

Diasome Pharmaceuticals Strengthens Leadership Team Ahead of Phase 3 Development for HDV-Insulin
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Representatives Introduce Bipartisan Bill to Permanently Authorize Rare Pediatric Disease Priority Review Voucher Program

Representatives Introduce Bipartisan Bill to Permanently Authorize Rare Pediatric Disease Priority Review Voucher Program

U.S. Representatives Gus Bilirakis (R-FL), Nanette Barragán (D-CA), Kat Cammack (R-FL), Jake Auchincloss (D-MA), Tom Kean, Jr. (R-NJ), and Kevin Mullin (D-CA) have introduced bipartisan legislation to permanently authorize the U.S. Food and Drug Administration’s (FDA) Rare Pediatric Disease Priority Review Voucher (PRV) Program. This program, established in 2012, incentivizes the development of treatments for children suffering from rare and devastating diseases. The PRV program awards a transferable priority review voucher to companies that successfully develop and receive FDA approval for qualifying treatments. Since its inception, the program has facilitated the market entry of treatments for 47 rare pediatric diseases, a significant increase from the four FDA-approved treatments that existed before the program. The current authorization for the program is set to expire in 2029. The proposed permanent authorization aims to provide long-term certainty for researchers and investors, encouraging continued ...
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Vyriad Initiates Phase 1 Clinical Trial for In Vivo CAR T Cell Therapy VV169 in Multiple Myeloma

Vyriad Initiates Phase 1 Clinical Trial for In Vivo CAR T Cell Therapy VV169 in Multiple Myeloma

Vyriad Initiates Phase 1 Clinical Trial for In Vivo CAR T Cell Therapy VV169 in Multiple Myeloma
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Opus Genetics Advances Gene Therapies for Inherited Retinal Diseases, Secures FDA Alignment for Phase 3 Trial

Opus Genetics Advances Gene Therapies for Inherited Retinal Diseases, Secures FDA Alignment for Phase 3 Trial

Opus Genetics is making significant strides in developing gene therapies for inherited retinal diseases (IRDs), specifically OPGx-LCA5 for LCA5-associated IRD and OPGx-BEST1 for BEST1-associated IRD. According to Dr. George Magrath, CEO of Opus Genetics, the OPGx-LCA5 program, targeting an ultra-rare disease affecting approximately 200 U.S. children, has shown promising results. All six participants treated to date (three pediatric and three adult) demonstrated improvement across various measures, including full-field stimulus threshold (FST) testing, best-corrected visual acuity (BCVA), virtual-reality maze tests, and microperimetry. Retinal sensitivity improved by about 1.5 log units at both blue and red wavelengths. The company has also secured FDA alignment for an eight-participant Phase 3 registrational trial for OPGx-LCA5, utilizing a 6-month pretreatment period where each participant serves as their own control. The OPGx-BEST1 program aims to restore bestrophin-1 channel function in the retinal pigm...