As the nation started to reopen this week following two months of closed businesses and stay-at-home orders, public health officials and economists cautioned that society won’t truly return to anything resembling a pre-pandemic normal without a COVID-19 vaccine.
Moderna (NASDAQ: [[ticker:MRNA]]) moved one step closer to filling that need with the release of preliminary Phase 1 data for its messenger RNA vaccine candidate. No major safety problems were reported and there were some indications that the injections elicited antibodies. But Phase 1 clinical trials are small, and it’s unknown whether those early results can be replicated in larger studies. Furthermore, neutralizing antibody data were available for only only eight patients, and the company provided little detail to contextualize those findings.
Nevertheless, Morderna plans to move quickly into a Phase 2 test, followed by a Phase 3 study in July while the second stage of testing is still underway. It’s rare for a company to talk about Phase 3 even before full Phase 1 data are available but the rapid plan reflects the urgency of these times.
COVID stories dominated the headlines but there was other life science news this week. Several cancer drugs won regulatory approvals, a pharma giant struck a deal to gain a place in a crowded field, and a stealthy biotech emerged from the shadows with $145 million. Let’s recap those stories and more in this week’s roundup.
COVID-19 NEWS
—Moderna reported preliminary Phase 1 data for its experimental COVID-19 vaccine, mRNA-1273, and the Cambridge, MA-based company is preparing to advance the drug into Phases 2 and 3, which will evaluate the drug in larger numbers of patients.
—Mammoth Biosciences is collaborating with the consumer health division of GlaxoSmithKline (NYSE: [[ticker:GSK]]) to develop a disposable, handheld test that uses the startup’s CRISPR-based technology to detect the novel coronavirus.
—The US promised AstraZeneca (NYSE: [[ticker:AZN]]) up to $1.2 billion to more rapidly advance a COVID-19 vaccine candidate the company has licensed from the University of Oxford. The money will go toward late-stage development and manufacturing of the investigational vaccine slated to enter late-stage clinical testing this summer. It’s the fourth vaccine partnership struck by the Biomedical Advanced Research and Development Authority.
DOLLARS & DEALS
—Rallybio closed a $145 million Series B round of funding to advance a pipeline of rare disease drugs including a Phase 1 ready antibody drug for fetal and neonatal alloimmune thrombocytopenia, a blood disorder.
—Emeryville, CA-based startup Octant raised $30 million to develop technology it is using to determine how small molecules interact with a family of membrane proteins known as G-protein coupled receptors.
—Amplyx Pharmaceuticals, a San Diego biotech advancing a new kind of antifungal treatment for hospitalized patients who are especially vulnerable to such infections, brought aboard new investors including Pfizer (NYSE: [[ticker:PFE]]) to extend its Series C financing to more than $90 million.
—Vividion Therapeutics and Roche inked an alliance to work on discovering and developing new drugs that leverage cells’ protein degradation process—how damaged or unwanted proteins are discarded—to treat disease. The Swiss pharma giant paid the San Diego biotech $135 million up front.
—HotSpot Therapeutics landed $65 million in Series B financing to advance two allosteric molecules to tests in humans.
—South San Francisco-based Applied Molecular Transport filed plans for an IPO intended to finance clinical tests of biologic drugs that use the company’s technology to reach disease targets in the gut.
—Clinical-stage cell therapy developer SQZ Biotech announced a $65 million Series D funding round to support its oncology pipeline and expand into infectious diseases.
—Nautilus Biotechnology raised a $76 million Series B round of funding to develop its technology for analyzing and quantifying the human proteome, the entire set of proteins produced by a cell.
DRUG DEVELOPMENTS
—Apellis Pharmaceuticals (NASDAQ: [[ticker:APLS]]) plans to submit its investigational drug pegcetacoplan to the FDA for review as a treatment for paroxysmal nocturnal hemoglobinuria (PNH). The announcement comes four months after the company reported Phase 3 data showing that pegcetacoplan beat an Alexion Pharmaceuticals (NASDAQ: [[ticker:ALXN]]) PNH drug head-to-head in a Phase 3 study.
—SutroVax, a vaccine developer spun out of Sutro Biopharma (NASDAQ: [[ticker:STRO]]), is changing its name to Vaxcyte. The company’s lead product candidate, a pneumococcal conjugate vaccine, is in preclinical development.
—Gilead Sciences (NASDAQ: [[ticker:GILD]]) and partner Galapagos reported that the high dose of their experimental drug, filgotinib, met the main goals of a pivotal study testing it in ulcerative colitis. But the low dose failed to beat a placebo.
—Cancer drug developer Zentalis Pharmaceuticals (NASDAQ: [[ticker:ZNTL]]) established a China-based joint venture called Zentera Therapeutics. Zentera, which will develop and commercialize Zentalis drugs in China, is backed by $20 million in Series A financing.
—AMAG Pharmaceuticals (NASDAQ: [[ticker:AMAG]]), which is exiting the women’s health business, reached a deal to sell intercourse pain drug prasterone (Intrarosa) to Millicent Pharma for $20 million up front.
DRUG DECISIONS
—The FDA approved Sunovion Pharmaceuticals sublingual film formulation of apomorphine hydrochloride (Kynmobi) as a treatment for “off” episodes experienced by Parkinson’s disease patients.
—Deciphera Pharmaceuticals (NASDSAQ: [[ticker:DCPH]]) drug, ripretinib (Qinlock), was approved by the FDA to treat patients with a gastrointestinal stromal tumor (GIST) who have failed at least three previous treatments. The green light for the Boston company—its first—came three months earlier than anticipated, one of a number of recent early FDA rulings for cancer drugs.
—But the FDA rejected an application from Blueprint Medicines (NASDAQ: [[ticker:BPMC]]) seeking approval of its drug, avapritinib, as a fourth-line treatment for GIST.
—The European Commission granted conditional approval to Zolgensma, a Novartis (NYSE: [[ticker:NVS]]) gene therapy for the rare disease spinal muscular atrophy. The decision follows FDA approval of the therapy nearly a year ago.
— Clovis Oncology (NASDAQ: [[ticker:CLVS]]) cancer drug rucaparib (Rubraca), a so-called PARP inhibitor that addresses cancers with a specific genetic mutation, was approved for prostate cancer. It’s the third approved indication for rucaparib and the first for a PARP drug in prostate cancer.
—Just days after Clovis’s PARP inhibitor was approved, a similar drug developed by AstraZeneca and Merck (NYSE: [[ticker:MRK]]), also received an FDA nod. Their PARP drug, olaparib (Lynparza), was also approved for advanced prostate cancers carrying a certain genetic mutation.
—A dual immunotherapy from Bristol Myers Squibb (NYSE: [[ticker:BMY]]), nivolumab (Opdivo) plus ipilimumab (Yervoy) was approved by the FDA as a first-line treatment of adults with metastatic non-small cell lung cancer (NSCLC) whose tumors express the protein PD-L1.
—The FDA OK’d Roche cancer drug atezolizumab (Tecentriq) as an initial treatment option for adults with certain types of non-small cell lung cancer. The approval was the drug’s fifth in lung cancer.
PEOPLE ON THE MOVE
Merck spinoff Organon added Matthew Walsh as its chief financial officer and Rachel Stahler as chief information officer… Tushar Shah was appointed chief medical officer of Liquidia Technologies (NASDAQ: [[ticker:LQDA]])… Ardelyx (NASDAQ: [[ticker:ARDX]]) named Susan Rodriguez its chief commercial officer… Sonya Montgomery was appointed chief medical officer of Evox Therapeutics… William Erhardt joined Oligomerix as chief medical officer… Mirati Therapeutics (NASDAQ: [[ticker:MRTX]]) named Joseph Leveque its chief medical officer… and Tara Heitner was appointed CEO of Cyxone.
Sarah de Crescenzo contributed to this report.
Image: iStock/MarianVejcik
