San Diego-based synthetic biology player Synthorx Inc. has landed $63 million in series C financing to back further advancement of a series of improved cytokine therapies, led by Synthorin IL-2, an interleukin-2 tuned specifically for activity on certain immune cells. Following a pre-investigational new drug (IND) meeting with the FDA, the company expects to file an IND in the first half of 2019, Laura Shawver, the company's president and CEO, told BioWorld.
Solid first-quarter earnings, positive phase III data on Soliris (eculizumab) successor ALXN-1210, and rosy revenue guidance sent shares of Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) climbing Thursday as the company pushed ahead with efforts to rebuild its pipeline. It reported earnings of $1.68 per share, beating analyst expectations of $1.50.
Following an initial FDA rejection in 2013, Otsuka Pharmaceutical Co. Ltd.'s tolvaptan, a drug for autosomal dominant polycystic kidney disease (ADPKD), has now won the agency's approval. The decision will give U.S. adults with the condition — the fourth leading cause of end-stage renal disease — access to a drug that has been available to those with ADPKD in Japan since 2014.
Cedilla Therapeutics Inc., a startup harnessing protein stability mechanisms to broaden the reach of small-molecule drugs in cancer and other diseases, is debuting with $56.2 million in series A funding from Third Rock Ventures. The Cambridge, Mass.-based company, created last summer by academics at Harvard and University of California San Francisco and led by former Editas Medicine Inc. chief operating officer Alexandra Glucksmann, is seeking new points of intervention for diseases caused by protein dysregulation.
Prothena Corp. plc is quitting development of its most advanced candidate, NEOD-001 for AL amyloidosis, after a phase IIb failure triggered a review of the program's phase III study, leading to its discontinuation for futility. The phase IIb study, Pronto, missed both its primary and secondary endpoints, while an independent data monitoring committee (DMC) recommended discontinuation of the phase III trial, called Vital.
Dicerna Pharmaceuticals Inc. has agreed to $25 million worth of cash and stock to settle a long-running legal spat with competitor Alnylam Pharmaceuticals Inc., which had sought damages in excess of $100 million against it. News of the settlement sent Dicerna's shares (NASDAQ:DRNA) briefly more than 40 percent higher Friday, before ending the day at $12.01, up $1.82.
BenevolentAI Ltd., an artificial intelligence (AI)-driven drug discovery firm that sees the pharmaceutical industry as ripe for disruption, has raised $115 million to scale up its drug development activities, broaden its disease focus and hire new talent. Since 2013, it has raised more than $200 million, including the latest inflow, sourced primarily from U.S. investors.
Tetherex Pharmaceuticals Inc., a rare Oklahoma City-based biotech, raised $50 million in a private placement of series B stock led by MPM Capital. The funds will support a phase II trial of the company's lead candidate, SEL-K2, for the potential treatment of venous thromboembolism (VTE) in patients undergoing total knee replacement surgery and a second midstage study in Crohn's disease, another inflammatory indication, or cancer. The financing follows an initial raise of about $4.4 million in convertible debt in 2014.
Ultragenyx Pharmaceutical Inc. and partner Kyowa Hakko Kirin Co. Ltd. (KHK) have won FDA approval for Crysvita (burosumab), making it the first U.S.-approved treatment for X-linked hypophosphatemia (XLH), a rare, inherited form of rickets. The chronic progressive musculoskeletal disorder is estimated to affect about 3,000 children and 9,000 adults in the U.S.
Celldex Therapeutics Inc. is discontinuing its lead program, the antibody-drug conjugate glembatumumab vedotin (glemba), after it failed to help women with tough-to-treat metastatic triple-negative breast cancers (TNBC) stay both alive and progression-free for longer than Roche Holding AG’s Xeloda (capecitabine), missing the primary endpoint of a phase IIb study. The hard blow, which will also bring an end to a phase II combination study in melanoma, precipitated plans for restructuring and re-evaluation at the company as its shares (NASDAQ:CLDX) tumbled 64.6 percent to close at 76 cents on Monday.
