Despite prior marketing approvals in Europe and Canada, an effort to win FDA support for the bifunctional alkylating agent treosulfan has drawn a complete response letter (CRL) from the agency, the company's U.S. rights holder, Medexus Pharmaceuticals Inc., said.
Fresh off licensing a potential Parkinson’s disease therapy last month, Ipsen SA is again looking to build out its neurodegenerative disease portfolio, this time in Huntington’s disease and Angelman syndrome. An exclusive options deal with Exicure Inc. could bring it two new spherical nucleic acid (SNA) programs for the indications while delivering up to $1 billion for Exicure, plus potential royalties, on top of a $20 million up-front payment. The deal, Exicure's second major SNA collaboration after a hair-loss disorders deal with Abbvie Inc.’s Allergan, sent Exicure shares (NASDAQ:XCUR) up 34.1% to $1.81 on Aug. 2.
Peptidream Inc., fresh off expanding a multibillion-dollar research and license agreement with Takeda Pharmaceutical Co. Ltd. earlier this week, has inked a brand new deal with Alnylam Pharmaceuticals Inc. Valued at up to $2.2 billion, including milestones, the new deal is aimed at the discovery and development of peptide-siRNA conjugates for the delivery of therapies to a wide range of cell types and tissues beyond the liver, the central target of Alnylam's marketed products to date.
Eli Lilly and Co. has established a multiyear collaboration with Kumquat Biosciences Inc. for the discovery, development and commercialization of new small molecules for stimulating tumor-specific immune responses. Kumquat will receive $70 million up front, including an equity investment, and is eligible for more than $2 billion in potential milestone payments plus royalties on any marketed products.
New data on the addition of Infinity Pharmaceuticals Inc.'s oral PI3K-gamma inhibitor, eganelisib, to PD-(L)1 in both urothelial and triple-negative breast cancer patients appeared to evidence benefits for the combination. Benefit for company shares (NASDAQ:INFI), however, was nowhere to be seen, as they fell 31.5% to $1.52 on July 27. In a same-day earnings report, the Cambridge, Mass.-based company said it had a cash balance of $97.3 million to fund further development of the drug and filed to raise the maximum amount of capital it may raise in future sales of its shares.
Amid a mix of controversy and clinical progress, the Alzheimer’s Association International Conference 2021 kicked off July 26 in Denver and online, delivering both new clinical data and insights into a key form of dementia affecting as many as 35 million people worldwide and growing. While interest in Biogen Inc.'s recently approved Aduhelm (aducanumab) remained high, new data on investigational therapies from Synaptogenix Inc. and Inmune Bio Inc. also drew attention, weighing on shares of both companies. New epidemiological research also revealed the first evidence that reducing air pollution may improve cognitive function and reduce dementia risk.
In search of new therapies for amyotrophic lateral sclerosis (ALS), Eli Lilly and Co. has agreed to pay Verge Genomics $25 million in up-front, equity and potential near-term payments, plus as much as $694 million in milestones, to discover and validate up to four new targets for treating the disease over the course of a three-year collaboration. Verge retained rights to its internal lead ALS program, poised to enter the clinic next year.
In a new chapter for the ongoing story of sodium glucose transporter-2 inhibitor (SGLT2) inhibitors, Boehringer Ingelheim International GmbH and Eli Lilly and Co.'s Jardiance (empagliflozin) has become the first therapy of the class to significantly reduce the risk of cardiovascular death or hospitalization vs. placebo for heart failure patients with preserved ejection fraction (HFpEF). The finding comes from the phase III Emperor-Preserved study, which tested once-daily Jardiance 10 mg vs. placebo in nearly 6,000 adults living with HFpEF, with and without diabetes.
An ongoing FDA review of Iterum Therapeutics plc's NDA for a bilayer tablet containing sulopenem etzadroxil and probenecid for uncomplicated urinary tract infections (uUTI) has uncovered "deficiencies that preclude the continuation" of talks on labeling and postmarketing requirements, the company said. The revelation, less than four weeks before the NDA's July 25 PDUFA date, "throws an on-time approval into serious doubt," making Iterum's receipt of a complete response letter "a reasonably likely scenario," H.C. Wainwright analyst Ed Arce wrote.
Just months after Jazz Pharmaceuticals plc's inventory of a therapy key to treating certain acute lymphoblastic leukemia and lymphoblastic lymphoma patients appears to have run out, FDA approval has arrived for an alternative: a recombinant version of the medicine the company developed, Rylaze (asparaginase Erwinia chrysanthemi (recombinant)).
