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New medicines #50 - April 24, 2021 - April 30, 2021
A weekly overview of the development of new medicines.
- ADC Therapeutics announced the FDA approved Zynlonta (loncastuximab tesirine) for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more previous treatments:
Continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial
- Adverum Biotechnologies announced a Suspected Unexpected Serious Adverse Reaction (SUSAR) of hypotony (decrease in ocular pressure) in its Phase 2 Infinity trial evaluating ADVM-022 gene therapy for the treatment of diabetic macular edema (DME).
- Aldeyra Therapeutics released top-line data from a phase 3 trial of reproxalap in patients with allergic conjunctivitis:
The trial achieved statistical significance for the primary endpoint and all secondary endpoints
- Cara Therapeutics announced its phase 2 trial of Oral Korsuva for the treatment of moderate-to-severe pruritus in mild-to-severe atopic dermatitis (AD) patients did not meet primary endpoint of the change from baseline in the weekly mean of the daily 24-hour Itch-Numeric Rating Scale (I-NRS) score at Week 12.
- Coherus BioSciences announced that its partner Junshi Biosciences’ phase 3 trial of toripalimab, in combination with paclitaxel/cisplatin as first-line treatment for patients with advanced esophageal squamous cell carcinoma (ESCC), met the primary endpoints of progression free survival (PFS) and overall survival (OS) at the interim analysis.
- Protalix BioTherapeutics announced that the FDA issued a Complete Response Letter (CRL) regarding the Biologics License Application (BLA) seeking approval of pegunigalsidase alfa (PRX‑102) for the proposed treatment of adult patients with Fabry disease:
FDA needs to inspect its manufacturing facility in Israel prior to its decision but was unable to do so due to travel restrictions
Issues were also noted regarding a third-party facility in Europe
The FDA noted that the recent full approval of Fabrazyme by Sanofi must be addressed in the context of any potential resubmission seeking accelerated approval of PRX‑102
- Reata Pharmaceuticals announced that the FDA accepted its New Drug Application (NDA) for bardoxolone for the treatment of patients with chronic kidney disease caused by Alport syndrome and assigned a PDUFA date of February 25, 2022.
- uniQure announced that the FDA has removed the clinical hold on its hemophilia B gene therapy program with 52-week data from their Phase 3 HOPE-B trial are due later this quarter.
- Voyager Therapeutics announced the FDA has removed its clinical hold on its Investigational New Drug (IND) application for VY-HTT01, a gene therapy candidate for the treatment of Huntington’s disease (HD):
Voyager plans to initiate VYTAL, a Phase 1/2 clinical trial of VY-HTT01, this year
Source: BPC (reformatted), SY, Bioworld