The BioWorld Cancer index, which includes 21 representative companies developing therapies targeting various cancers, entered the new year on a high note, after posting a 22.5% gain for the year. Unfortunately, the group hit a speed bump and the index took a beating in January, dropping almost 9% as a result. However, it didn’t take long for the index to recover those gains and then some as, so far this month, it has pushed 10.6% higher.
Coronavirus latest developments
Multiple China-based clinical trials have been put on hold as the country concentrates on its fight against COVID-19. To curb the spread of the novel coronavirus that has infected more than 72,000 people and killed nearly 2,000, China has imposed travel and transport restrictions, making trips difficult or even impossible for patients and physicians. In Wuhan, the epicenter of the outbreak, which alone has seen around 18,000 people infected, Yue Wang’s site management organization (SMO) is conducting 51 trials, including 39 oncology studies. She said that many visits have been canceled.
Aim Immunotech Inc. said it is re-examining the antiviral properties of Ampligen (rintatolimod) to determine if it could become a vaccine to protect against the Wuhan coronavirus.
Esperion Therapeutics Inc. has won FDA approval for bempedoic acid as an adjunct to diet and maximally tolerated statin therapy. The approval covers the treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or established atherosclerotic cardiovascular disease who require additional lowering of low-density lipoprotein cholesterol (LDL-C). It will be branded as Nexletol, in a variation from the expected European brand name, Nilemdo. An EMA review of the company's MAA is ongoing.
Valrox (valoctocogene roxaparvovec) from Biomarin Pharmaceutical Inc. moved one step closer to entering the U.S. market, with the company reporting that that the FDA had accepted for priority review the BLA for its investigational AAV5 gene therapy for adults with hemophilia A.
Deals of the week
Five Prime Therapeutics Inc. said it's licensing a family of monoclonal antibodies to Seattle Genetics Inc. for inclusion in new early stage cancer-killing antibody-drug conjugate candidates, all directed to a single target. The deal brings $5 million up front for Five Prime, plus up to $525 million in milestone payments for the first two candidates. South San Francisco-based Five Prime's shares (NASDAQ:FPRX) rose about 18% on news of the deal. Nimble Therapeutics Inc. disclosed a multiyear collaboration with Genentech, a member of the Roche Group, to accelerate discovery and development of peptide-based medicines for targets across a range of diseases. Immatics Biotechnologies GmbH received $50 million up front and could earn up to $550 million more in per-product milestones from an adoptive cell therapy deal in solid tumor indications with Glaxosmithkline plc, which initially involves two autologous cell therapies engineered to express T-cell receptors that bind novel cancer targets. Immatics would receive royalties on sales of any approved therapies. In an all-stock transaction, Tocagen Inc. will merge with Forte Biosciences Inc. and be renamed Forte Biosciences. The new company will trade under the ticker FBRX.
Chinese biosimilar maker Bio-Thera Solutions Ltd. launched a pre-revenue listing on Shanghai’s STAR market, a Nasdaq-style tech board, to reap $241 million at a valuation of almost $2 billion. Its share price soared more than 83% during its first trading day. Revolution Medicines Inc. closed its IPO for gross proceeds of approximately $273.7 million. Passage Bio Inc. filed to raise up to $126 million in an IPO. The company is working on treatments for rare monogenic central nervous system diseases. Imara Inc. filed to raise up to $86 million in an IPO. The company is developing and commercializing therapeutics to treat hemoglobinopathies.
Public offerings: Adverum Biotechnologies Inc. raised gross proceeds of approximately $150.2 million. Bioxcel Therapeutics Inc. priced a public offering for gross proceeds of about $64 million. Catalyst Biosciences Inc. raised gross proceeds of approximately $34.5 million. Deciphera Pharmaceuticals Inc. closed its public offering for gross proceeds of about $175 million. Eyepoint Pharmaceuticals Inc. priced an underwritten public offering for gross proceeds of about $21.7 million
Private transactions: Alladapt Immunotherapeutics Inc. completed a $60 million series C financing. Canbridge Pharmaceuticals Inc. completed a $98 million series D financing. Omass Therapeutics, which is harnessing mass spectrometry and other biophysical technologies to drive drug discovery, said it completed an extended series A financing of £27.5 million (US$35.757 million). Neotx Therapeutics Ltd. raised $45 million in a series C round. Spruce Biosciences Inc. raised $88 million in a series B financing.
The FDA is releasing a draft guidance, “Nonclinical safety evaluation of the immunotoxic potential of drugs and biologics,” to provide consistent recommendations for the nonclinical assessments of immune endpoints and to supplement recommendations provided in other guidances. The draft replaces the withdrawn 2002 guidance, “Immunotoxicology evaluation of investigational new drugs.” The new guidance addresses multiple aspects of immune suppression, modulation and stimulation, including carcinogenicity assessment, dermal sensitization, adjuvanted vaccine development, and developmental and juvenile animal studies, according to a notice in the Feb. 20 Federal Register. Comments on the draft should be submitted by April 20.
The Institute for Clinical and Economic Review (ICER) said it plans to assess the comparative clinical effectiveness and value of Ferring Pharmaceuticals SA’s nadofaragene firadenovec (rAd-IFN/Syn3, also known as Instiladrin) for the treatment of bladder cancer. An FDA review decision on the candidate is expected in mid-2020, ICER said.
Word on the Street: BioWorld's favorite quotes of the week
“We now know all the 20,000 different genes and how they are expressed, so we can then dig in and ask what the exact differences are between these two different [tissue samples]. That gave us a list of genes that are highly expressed in asthma. Actually, we find that a lot of these genes have to do with how cells talk to and recruit immune cells such as interleukins and cytokines. That is highly relevant to the disease.”
Kerstin Meyer, principle scientist leading Human Cell Atlas projects at the Sanger Institute in Cambridge, U.K., speaking to attendees of the AAAS meeting. The initiative has mapped 100 million of 100 billion cells, and by comparing gene expression profiles of normal and aberrant cells and building networks of cellular interactions, is leading to breakthroughs in understanding of disease at a molecular level. Meyer is involved in a project that combines the two approaches to look at bronchial cells from healthy individuals and asthma patients. That “cellular census” has identified novel cell states in asthma.
“The parachute was invented 500 years before there was an airplane. Until the checkpoint inhibitor era, this was a drug that struggled a little bit.”
Asher Nathan, co-founder and CEO of Neotx Therapeutics Ltd., which raised $45 million in a series C round to continue clinical development of naptumomab estafenatox, a fusion protein comprising an antibody fragment that recognizes the oncofetal antigen 5T4 and a bacterial “super-antigen” comprising a modified version of the Staphylococcal enterotoxin A
“From the beginning, there was an understanding that there were probably lots of reasons not to go into this space from a market perspective. I only saw it as an opportunity to do well. You only have to look at Melinta and Achaogen to understand that single-product portfolios are challenged.”
Armand Balboni, CEO, Appili Therapeutics Inc., which closed a public offering of CA$10.25 million (US$7.7 million) to ready its new asset, ATI-2307, an antifungal agent for a phase II trial