When he first stepped into the field of glycoproteomics, particularly the potential of truncated O-glycans as cancer targets, Constantine Theodoropulos, CEO of Go Therapeutics Inc., said the reaction from peers was less than encouraging.
Shares of Aeglea Biotherapeutics Inc. tumbled 51% June 2 on news that it received a refusal to file (RTF) letter from the U.S. FDA regarding the BLA for pegzilarginase for the treatment of arginase 1 deficiency, with the agency requesting additional efficacy data.
When he first stepped into the field of glycoproteomics, particularly the potential of truncated O-glycans as cancer targets, Constantine Theodoropulos, CEO of Go Therapeutics Inc., said the reaction from peers was less than encouraging. “They said, ‘You’re nuts, it’s too complicated.’” Now, however, the company he co-founded has landed a deal with Astellas Pharma Inc.’s immuno-oncology-focused subsidiary, Xyphos Biosciences Inc., that brings $20.5 million up front, with the potential for up to $763 million in milestone and contingency payments down the road.
The U.S. FDA has rejected Verrica Pharmaceuticals Inc.’s drug-device combination to treat the viral skin disease molluscum for a third time, losing more ground to a potential rival from Novan Inc., because of continued manufacturing issues. There are no FDA-approved treatments for molluscum contagiosum, which leads to skin-colored or pink lesions and affects around 6 million people in the U.S. annually.
Dermavant Sciences Inc. has received its first FDA approval with the agency’s blessing of Vtama (tapinarof) for treating plaque psoriasis in adults. The treatment is also the first FDA-approved, steroid-free topical medication in its class in addition to being the first psoriasis novel topical chemical entity introduced to the market in the past 25 years.
Unveiling further positive data for SER-109 in preventing recurrent Clostridium difficile infection at Digestive Disease Week (DDW) over the weekend, Seres Therapeutics Inc. reiterated plans to file a BLA with the U.S. FDA in mid-2022. Should the FDA accept the application and grant priority review – SER-109 has breakthrough and orphan status – the medicine could be the first microbiome-based therapy to reach the market.
A move by Chimerix Inc. to strengthen its balance sheet by $225 million through the sale of smallpox drug Tembexa (brincidofovir) to biodefense specialist Emergent Biosolutions Inc. and extend its cash runway into 2026, should have proved a big win. Instead, shares (NASDAQ:CMRX) plunged nearly 61% May 16 on worry that Chimerix might be handing off a likely profitable program to fund a riskier oncology pipeline, a concern heightened by recent U.S. FDA feedback indicating lead oncology program ONC-201 might not be eligible for accelerated approval as previously expected.
Newly appointed President and CEO Jackie Shea looks to have her work cut out for her, as Inovio Pharmaceuticals Inc. decided to ditch its phase II/III COVID-19 vaccination trial in favor of pursuing a booster strategy with INO-4800. That update, disclosed during Inovio’s first-quarter earnings late May 10 alongside a likely delay in filing for approval of HPV immunotherapy candidate VGX-3100, sent the stock (NASDAQ:INO) falling 27% May 11. Over the past year, shares have fallen more than 70%.
Askgene Pharma Inc., which less than two weeks ago reported positive initial data from an ongoing phase I/II trial testing its claudin 18.2-targeting candidate, ASKB-589, added $20 million in a series A round, intended to advance the company’s clinical pipeline and support further development of its Smartkine cytokine drug platform.
Connect Biopharma Holdings Ltd. pointed to positive secondary endpoint data and a numerical trend on the primary endpoint in favor of its S1P receptor modulator, CBP-307, in ulcerative colitis, but those results weren’t enough to keep the stock (NASDAQ:CNTB) from falling by more than 57% to close at 82 cents, as investors focused on the primary endpoint miss and the company’s decision to partner the program going forward.