Strategic Signals Weekly

Capricor Therapeutics announced that the FDA has extended the PDUFA target action date for its Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD), from August 22, 2026, to November 22, 2026. This three-month extension is due to a major amendment submitted by Capricor, which included 24-month open-label extension data from the pivotal Phase 3 HOPE-3 study and additional analyses supporting a refined proposed indication focused on upper limb function.

Drug: Deramiocel · Indication: Duchenne muscular dystrophy

PTC Therapeutics, Inc. has entered into an asset purchase agreement to acquire ST-920, a one-time administered AAV gene therapy product candidate for Fabry disease, from Sangamo Therapeutics, Inc. for an upfront payment of $111 million. The agreement also includes up to $100 million in contingent milestone payments tied to accelerated and full FDA approval of a BLA for ST-920 based on Sangamo's Phase 1/2 STAAR study. This acquisition follows PTC being selected as the winning bidder in Sangamo's bankruptcy auction.

Drug: ST-920 · Indication: Fabry disease · Partner: Sangamo Therapeutics, Inc.

Biohaven Ltd.'s subsidiary, Biohaven Bioscience Ireland Limited (BBIL), entered into a global licensing agreement with SK Biopharmaceuticals Co., Ltd. (SKBP). Under the agreement, SKBP gains exclusive worldwide rights to Biohaven's Kv7 ion channel platform, including the lead candidate opakalim (BHV-7000) for focal epilepsy. Biohaven will receive an upfront payment of $400 million, with potential for up to $150 million in development and regulatory milestones, plus tiered royalties on net sales.

Drug: opakali · Indication: focal epilepsy · Partner: SK Biopharmaceuticals Co., Ltd.

Zymeworks' partner, Jazz Pharmaceuticals, received FDA approval for two Ziihera® (zanidatamab-hrii)-containing regimens for the first-line treatment of adults with unresectable locally advanced or metastatic HER2-positive gastroesophageal adenocarcinoma (GEA). This approval triggered a $250 million milestone payment to Zymeworks. Zymeworks is also eligible for up to $1.3 billion in additional regulatory and commercial milestones, plus tiered royalties of 10% to 20% on net sales.

Drug: Ziihera (zanidatamab-hrii) · Indication: unresectable locally advanced or metastatic HER2-positive (HER2+) gastroesophageal adenocarcinoma (GEA) · Partner: Jazz Pharmaceuticals Ireland Limited

Quoin Pharmaceuticals Ltd. entered into a private placement agreement with institutional and accredited investors to raise gross proceeds of up to $50.0 million. The company expects to receive approximately $30.8 million upfront, with an additional $19.2 million potentially from the cash exercise of ordinary warrants. Quoin intends to use the net proceeds for general corporate purposes, including the completion of clinical development for QRX003 for Netherton Syndrome, and expects the funds to last into the second half of 2029.

Coherus Oncology, Inc. entered into an at-the-market (ATM) sales agreement with Leerink Partners LLC, allowing the company to sell up to $50.0 million of its common stock. The company is not obligated to sell any shares, and any net proceeds will be used for general corporate purposes, including working capital.

Serina Therapeutics, Inc. entered into a Common Stock Purchase Agreement with Roth Principal Investments, LLC, establishing an at-the-market (ATM) offering. The company has the discretion to sell up to $25,000,000 of its common stock to Roth Principal Investments over a 36-month period, following the effectiveness of a registration statement. Sales will occur at a discount to the volume-weighted average price (VWAP), with the discount varying based on the cumulative amount sold and the type of purchase.

Gossamer Bio, Inc. announced the closing of its previously disclosed private placement on August 24, 2026. In connection with this financing, the company amended its certificate of incorporation by filing a Certificate of Designation for Series A-1 Convertible Preferred Stock with the State of Delaware. This filing details the rights and preferences of the Series A-1 Preferred Stock.

Skye Bioscience, Inc. entered into a Securities Purchase Agreement with Redmile Biopharma Investments III, L.P. for an equity line of credit (ELOC) of up to $22 million. This ELOC is contingent on the gross equity proceeds from a concurrent PIPE financing; if the PIPE exceeds $103 million, the ELOC amount will be reduced, and if it reaches $125 million, the ELOC will not be utilized. Additionally, the company agreed to issue a warrant to Redmile for the purchase of up to $5 million in shares.