1204 Quick Takes
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Product Development

Dec. 4 Quick Takes: Zogenix in Dravet deal with start-up; plus updates from BioCryst, Takeda, AB Science, Sutro, Lilly, Cedars-Sinai

Dec 5, 2020 | 1:42 AM GMT

Aiming to reverse Dravet, Tevard partners with Zogenix
Zogenix Inc. (NASDAQ:ZGNX) will collaborate with Tevard Biosciences to develop transfer RNA-based gene therapies to treat Dravet syndrome and other genetic epilepsies. The Cambridge, Mass.-based start-up, which has already received $5 million of the $10 million it is due up front in the deal, has two platforms for mRNA stabilization and nonsense codon suppression. Tevard is eligible for $70-$100 million in milestones for each program. FDA has approved Zogenix's Fintepla fenfluramine to treat Dravet.

FDA approves oral, non-steroidal product for HAE
FDA approved oral, once-daily Orladeyo berotralstat from BioCryst Pharmaceuticals Inc. (NASDAQ:BCRX) to prevent attacks of hereditary angioedema in patients 12 years and older. It is the first orally administered non-steroidal option for preventing HAE attacks, according to the U.S. Hereditary Angioedema Association.

Takeda’s maribavir meets in Phase III for CMV
Maribavir (TAK-620) from Takeda Pharmaceutical Co. Ltd. (Tokyo:4502; NYSE:TAK) met the primary endpoint in a Phase III trial to treat transplant recipients with resistant cytomegalovirus (CMV) infection.

Masitinib meets in pancreatic cancer Phase III 
Masitinib from AB Science S.A. (Euronext:AB) met the primary endpoint of a statistically significant increase in overall survival in first-line pancreatic cancer with pain. The company said the difference in median overall survival was 1.8 months (p=0.007) favoring masitinib (13.0 months  vs. 11.2 months for control), with a 0.46 hazard ratio of death, representing a reduction in risk of death of 54%.

Latest ovarian cancer data cut for ADC boosts Sutro’s shares
Sutro Biopharma Inc. (NASDAQ:STRO) gained 33% Friday, lifting its market cap to about $875 million, after updated data from a Phase I trial of antibody-drug conjugate STRO-002 showed that among 31 evaluable patients, the treatment led to one complete response, three confirmed partial responses and six unconfirmed PRs for an objective response rate of 32%. A readout in September had revealed eight PRs among 33 evaluable patients for a 24% ORR following treatment with the ADC targeting FOLR1.

Lilly, UnitedHealth readying pragmatic study of bamlanivimab
Eli Lilly and Co. (NYSE:LLY) partnered with UnitedHealth Group (NYSE:UNH) to conduct a pragmatic study of bamlanivimab in high-risk, COVID-19 infected individuals that comprises symptom tracking, in-home testing and in-home infusions to detect, intercept and treat COVID-19 early. FDA granted the mAb, which is partnered with AbCellera Biologics Inc., emergency use authorization last month. 

Cedars-Sinai opens cell therapy manufacturing center in Southern California
Cedars-Sinai Medical Center opened a more than 28,000-square foot cell therapy-focused biomanufacturing center in the Los Angeles area. The Cedars-Sinai Biomanufacturing Center’s core technology is induced pluripotent stem (IPS) cells; among its first clients are the Department of Defense, which has contracted with the facility to manufacture banks of stem cells that could be used to repair vascular injuries in military personnel.

TARGETS
FOLR1 (FR-α; FOLR) - Folate receptor 1

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