Clinical Development

Groton, Conn. – September 30, 2026 -- Astrocyte Pharmaceuticals Inc. has closed a $9.5 million Series B financing round to advance Phase 2 development of AST-004, its cerebroprotective therapy for concussion, traumatic brain injury (TBI) and stroke. The round brings total capital raised by the clinical-stage biotech, including grants, to $40 million.

DeepWork Capital leads financing with four new investors joining the round

The Series B was led by DeepWork Capital, with continued backing from existing investors and new participation from MintPharma Capital, Waterstar Capital, Dynagrow Capital and Angel Star Ventures. Jackson Streeter, M.D., Venture Partner at DeepWork Capital, said the firm has been impressed by the consistency of Astrocyte's science and the progress of its clinical programs.

Calgary – September 29, 2026 -- Providence Therapeutics Holdings, Inc. has formed its first Clinical Advisory Board to guide development of its therapeutic mRNA cancer vaccine programs, including a personalized vaccine for pediatric brain cancer and off-the-shelf precision therapies for solid tumors.

Chief Medical Officer Leon Rozen chairs new four-member advisory panel

Dr. Rozen leads the board, which brings in Ezra Cohen, MD; Melinda Merchant, MD, PhD; and John Powderly, MD, to steer clinical strategy for the company's cancer vaccine pipeline.

Tempus AI oncology chief joins board with immunotherapy track record

Ezra Cohen currently serves as Chief Medical Officer for Oncology at Tempus AI. He previously held roles as Professor of Medicine and Division Chief of Hematology/Oncology at the Moores Cancer Center, University of California, San Diego.

Valencia – – September 29, 2026 -- Arthex Biotech has appointed Glenn Sblendorio, former CEO of IVERIC bio, as Chairman of its Board of Directors as the clinical-stage RNA therapeutics company advances its lead program ATX-01 through Phase I/IIa trials for myotonic dystrophy type 1 (DM1).

Sblendorio brings three decades of biotech exit experience to Arthex's board

Sblendorio most recently led IVERIC bio, Inc. as CEO until its acquisition by Astellas Pharma, Inc. He previously served as President and Chief Financial Officer of The Medicines Company through its acquisition by Novartis, and as Executive Vice President and CFO of Eyetech Pharmaceuticals, Inc. until it was acquired by OSI Pharmaceuticals, Inc. Earlier in his career he was a Managing Director at MPM Capital Advisors.

Chengdu, China – September 29, 2026 -- iRegene Therapeutics has secured clinical trial clearances in both China and the United States for NouvSight001, an off-the-shelf cell therapy targeting retinitis pigmentosa (RP), a retinal disease affecting roughly 1.5 million people worldwide.

China's NMPA clears Phase I/III trial while FDA grants Phase I/II clearance with special exemption

China's National Medical Products Administration authorized a Phase I/III trial to assess safety, tolerability and preliminary efficacy of NouvSight001 in RP patients. The U.S. FDA separately cleared a Phase I/II trial for the same indication and granted a special exemption, building on the Orphan Drug Designation the agency awarded the program in 2024.

Cambridge, Mass. – September 26, 2026 -- NeuroSense Therapeutics (NASDAQ: NRSN) cut operating expenses to $3.4 million in the first half of 2026, down from $4.7 million a year earlier, as the biotech pushes its ALS candidate PrimeC toward a Canadian regulatory filing targeted for December 2026.

NeuroSense trims R&D and G&A spending by double digits in H1 2026

Research and development expenses fell 16% to $2,102 thousand from $2,503 thousand, driven by lower subcontractor and consultant costs, partly offset by higher share-based payment expense. General and administrative expenses dropped 38.6% to $1,345 thousand from $2,189 thousand on reduced professional services spending.

San Mateo, Calif. – September 25, 2026 -- Kali Therapeutics has been named one of the 11 most promising private biotechnology companies in the 2026 Endpoints 11, a list curated annually by Endpoints News editors from hundreds of private candidates.

Kali advanced two clinical candidates and closed a Sanofi licensing deal in just over two years

Founded in July 2024, the clinical-stage biotech moved from formation to two molecules in the clinic and a worldwide partnership with Sanofi inside roughly two years. The honorees were announced Thursday, September 24, 2026, at an awards gala at The State Room in Boston.

Rockville, Md. – September 25, 2026 -- REGENXBIO Inc. (Nasdaq: RGNX) reported that 60% of Dose Level 3 participants with three-year follow-up data in its Phase II ALTITUDE study achieved a greater-than-2-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) after a single in-office treatment with investigational gene therapy sura-vec, with no vision-threatening events recorded among responders.

Three-year data confirm durable DRSS improvement without repeat injections

In long-term follow-up as of September 25, 2026, six of ten Dose Level 3 participants with non-proliferative diabetic retinopathy (NPDR) who reached the three-year visit sustained a greater-than-2-step DRSS improvement without additional diabetic retinopathy treatment. The suprachoroidal delivery used Dose Level 3 (1.0x10^12 GC/eye) combined with short-course prophylactic topical steroids.

New Taipei City – September 24, 2026 -- Caliway Biopharmaceuticals (TWSE: 6919) said on September 21 that its Phase 2 CBL-0202DD trial of CBL-514 met its primary efficacy endpoint, delivering statistically significant pain reduction in patients with Dercum's Disease compared with placebo, in both the Full Analysis Set (FAS) and Per-Protocol (PP) populations.

CBL-514 Cuts Pain Scores by 3.1 Points at Week 20 Versus Placebo

In the FAS population, patients treated with CBL-514 achieved a mean pain-score reduction of 3.1 points at Week 20, versus 1.9 points for placebo (p=0.005). In the PP population, the same 3.1-point reduction was measured against a 1.4-point placebo response (p=0.0002).

Bridgewater, N.J. – September 21, 2026 -- The U.S. Food and Drug Administration has accepted Insmed Incorporated's supplemental New Drug Application (sNDA) for full approval of ARIKAYCE (amikacin liposome inhalation suspension) to treat Mycobacterium avium complex (MAC) lung disease, granting Priority Review with a PDUFA target action date of January 28, 2027.

FDA sets January 28, 2027 decision date under Priority Review

The Priority Review designation applies to therapies that could offer significant improvement in safety or efficacy for serious conditions. Insmed said full approval would allow ARIKAYCE, administered via PARI Pharma's Lamira Nebulizer System, to reach patients earlier in their disease course, including newly diagnosed cases and those with recurrent infections.

Germantown, Md. – September 21, 2026 -- The U.S. Food and Drug Administration has granted platform technology designation to Precigen's AdenoVerse immunotherapeutic platform, the technology underlying the company's first commercial product, PAPZIMEOS, approved in August 2025 for recurrent respiratory papillomatosis (RRP) in adults.

FDA designation opens accelerated pathway for future AdenoVerse cancer programs

The designation allows Precigen to leverage manufacturing and preclinical data generated for PAPZIMEOS to support future marketing applications for other AdenoVerse-based candidates, reducing duplicative development work. It also grants the company earlier and more frequent FDA engagement and allows regulators to consider prior manufacturing inspections when reviewing future applications.