Biotech Daily: Merck’s Two Combo Cancer Trials Miss Main Goal, Approval For Sinovac’s COVID-19 Shot For Youngest Kids, Alnylam Aces Protein Disorder Trial

Here’s a roundup of top developments in the biotech space over the last 24 hours:

Alnylam Reports Upbeat Data From Amyloidosis Study

Alnylam Pharmaceuticals Inc’s ALNY APOLLO-B Phase 3 study of patisiran for transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy met the primary endpoint of change from baseline in the 6-Minute Walk Test (6-MWT) at 12 months compared to placebo. 

The study also met the first secondary endpoint of change from baseline in quality of life compared to placebo.

Patisiran also demonstrated an encouraging safety and tolerability profile, with deaths numerically favoring the patisiran arm.

The company plans to file a supplemental marketing application with the FDA in late 2022.

Shares are up 45.8% at $207.00 during the premarket session.

Sinovac’s COVID-19 Shot Approved For Kids Below 3 Years 

The regulatory authority in Hong Kong has approved Sinovac Biotech Ltd’s SVA COVID-19 vaccine (CoronaVac), intended for children aged six months to 3 years.

Three doses of vaccines will be used, with the first two doses given 28 days apart, followed by a third dose at least three months after the second dose.

FDA Approves Coherus’ Lucentis Referenced Biosimilar

The FDA has approved Coherus BioSciences Inc’s CHRS Cimerli (ranibizumab-eqrn) as a biosimilar product interchangeable with Roche Holding AG’s RHHBY Lucentis for five retina indications.

Cimerli belongs to the anti-VEGF therapy class of biologics.

Commercial availability of Cimerli, in both 0.3 and 0.5 mg dosages, is planned for early October 2022.

Rigel Pharma Expanded Hematology-Oncology Portfolio With License Pact

Rigel Pharmaceuticals Inc RIGL and Forma Therapeutics Inc FMTX entered into an exclusive, worldwide license agreement to develop, manufacture and commercialize olutasidenib.

Forma will receive an upfront payment of $2.0 million and is eligible to receive an additional $17.5 million as near-term regulatory, approval, and first commercial sale milestones. 

In addition, Forma is eligible to receive up to an additional $215.5 million in development and commercial milestones. 

Merck’s Two Keytruda Combo Cancer Trials Miss Primary Goal

Merck & Co Inc’s MRK Phase 3 KEYNOTE-921 trial of Keytruda combined with chemotherapy (docetaxel) did not meet its dual primary endpoints of overall survival (OS) and radiographic progression-free survival (rPFS) for metastatic castration-resistant prostate cancer.

Separately, Merck and Eisai Co Ltd’s ESALY Phase 3 LEAP-002 trial of Keytruda plus Lenvima did not meet its dual primary endpoints of OS and PFS in unresectable hepatocellular carcinoma.

Regenxbio Plans To File Application For Hunter Syndrome Gene Therapy In 2024

Regenxbio Inc RGNX intends to file a marketing application in 2024 using the FDA’s accelerated approval pathway for RGX-121 for Mucopolysaccharidosis Type II, known as Hunter Syndrome. 

The company also announced that a pivotal program for RGX-121 is active and enrolling patients. RGX-121 is an investigational, one-time AAV Therapeutic using the NAV AAV9 vector to deliver the gene that encodes the iduronate-2-sulfatase enzyme.

Also Read: Top Questions Tesla Shareholder Want Musk, Company Leaders To Address At Thursday’s Annual Shareholder Meeting

Poseida Shares Rally As Roche Takes Next Big Step Into Allogeneic CAR-T Field

Poseida Therapeutics Inc PSTX has entered into a strategic collaboration and license agreement with Roche Holdings AG RHHBY, focused on developing allogeneic CAR-T therapies directed to hematologic malignancies. 

Under the agreement, Roche will receive from Poseida either exclusive rights or options to develop and commercialize several allogeneic CAR-T programs in Poseida’s portfolio.

Poseida will receive $110 million upfront and could receive up to $110 million in near-term milestones.

In addition, Poseida is eligible to receive up to $6 billion in milestone payments.

PSTX shares are up 73.7% at $4.22 during the premarket session.

Offering

Arcutis Biotherapeutics Inc ARQT priced its underwritten public offering of 7.5 million at $20 per share, with gross proceeds of $150.

Kintara Therapeutics Inc KTRA entered into an equity purchase agreement for up to $20 million with Lincoln Park Capital Fund, LLC

Shares surged 119% to 39 cents during the premarket session.

On The Radar

PDUFA Dates

Sanofi SA SNY and Regeneron Pharmaceuticals Inc REGN: Supplemental application for Dupixent (dupilumab) for eosinophilic esophagitis (EoE) in patients 12 years and above.

Earnings

United Therapeutics Corporation UTHR: Before the market open.

Eyepoint Pharmaceuticals Inc EYPT: Before the market open.

Seres Therapeutics Inc MCRB: Before the market open.

Intercept Pharmaceuticals Inc ICPT: Before the market open.

Editas Medicine Inc EDIT: Before the market open.

Horizon Therapeutics PLC HZNP: Before the market open.

Amarin Corporation plc AMRN: Before the market open.

Regeneron Pharmaceuticals Inc REGN: Before the market open.

Moderna Inc MRNA: Before the market open.

Jazz Pharmaceuticals PLC JAZZ: After market close.

Arcus Biosciences Inc RCUS: After market close.

Mirati Therapeutics Inc MRTX: After market close.

Regenxbio Inc RGNX: After market close.

Fate Therapeutics Inc FATE: After market close.

Vanda Pharmaceuticals Inc VNDA: After market close.

Biomarin Pharmaceutical Inc BMRN: After market close.