The financial markets were delivered a one-two punch last week – a plunge in oil prices along with fears that the coronavirus continues to spread unabated. As a result, the Dow Jones Industrial Average cratered during the week but did recover some of its losses on Friday to close down almost 3%. Biopharma equities did not escape the carnage, with the BioWorld Biopharmaceutical index trading down. The market meltdown put a damper on what appeared to be the start of a market recovery for biopharma equities, reflecting the fact that investors believed that biopharma blue chip companies offered some safety during these uncertain and turbulent times.
FDA approves treatment for Cushing’s disease
Following the FDA approval of Novartis AG’s Isturisa (osilodrostat), an oral treatment for adults with Cushing’s disease, Recordati SpA is planning its U.S. market launch for the second or third quarter of this year. Recordati, which acquired Isturisa’s worldwide rights from Novartis in October for $390 million, expects sales to peak at $100 million annually.
Coronavirus R&D roadmap released from WHO
The World Health Organization (WHO) has released its COVID-19 R&D roadmap, highlighting the gaps in knowledge about the virus and setting out priorities for research. The organization is now calling on groups around the world to use the document – drawn up by 400 experts – to coordinate their efforts. The research roadmap is backed by a core set of protocols WHO has developed outlining how clinical studies should be designed and how data are collected, to ensure it is possible to compare and pool data from multiple clinical studies.
The Gates Foundation and Wellcome Trust have joined forces with financial services specialist Mastercard in establishing a $125 million seed fund to accelerate development of drugs to treat COVID -19. The COVID-19 Therapeutics Accelerator aims to play a catalytic role by speeding up evaluation of new and repurposed drugs and biologics to treat the novel coronavirus in the near term, and other viral pathogens in the longer term.
In other developments: U.S., China and Harbour Biomed Therapeutics Ltd. teamed up with Mount Sinai Health System, New York's largest academic medical system, to develop novel biotherapies in oncology and immunology. They will also be using Harbour’s H2L2 Harbour Mice platform to develop a monoclonal antibody (MAb) against the coronavirus that has turned into a global epidemic. According to the company, its fully human MAbs have the potential to prevent the spread of the virus by blocking infection of cells. Abbvie Inc. said it is helping with the experimental use of the HIV medicine Kaletra/Aluvia (lopinavir/ritonavir) to determine its efficacy in the treatment of COVID-19. The company is collaborating with select health authorities and institutions globally to determine antiviral activity as well as efficacy and safety of lopinavir/ritonavir against the virus. Algernon Pharmaceuticals Inc. said it will set aside some of its NP-120 (ifenprodil) drug supply from its planned phase II study for idiopathic fibrosis and chronic cough, to make it available on a compassionate use basis, to physicians and their patients with the most serious cases of COVID-19. Beroni Group Ltd., which is developing a medical solution using nanobody technology for COVID-19 with Tianjin University in China, advanced into cytological experiments and expects to complete them by the end of March. Cel-Sci Corp. said it is developing an immunotherapy with the potential to treat the COVID-19 coronavirus using its peptide technology using conserved regions of coronavirus proteins to stimulate protective cell-mediated T-cell responses and reduce viral load. Cocrystal Pharma Inc. said it is developing antiviral compounds to treat COVID-19 infections. The company and the Kansas State University Research Foundation have a new license agreement to develop broad-spectrum antiviral compounds for treating norovirus and coronavirus infections. Cyclica Inc. and Beijing’s Institute of Materia Medica, Chinese Academy of Medical Sciences, are collaborating to discover antiviral drug candidates for COVID-19. GT Biopharma Inc. and Cytovance Biologics LLC are collaborating to develop a treatment for COVID-19 using GT's Trike NK cell engager platform. Emergent Biosolutions Inc. said it initiated development of candidates to treat and prevent COVID-19, using its hyperimmune platforms. Humanigen Inc. is exploring clinical-stage strategies and partnerships to evaluate whether lenzilumab, its anti-human-GM-CSF monoclonal antibody, can reduce or prevent the cytokine storm resulting from COVID-19 infection. Medicago Inc. said it has produced a virus-like particle (VLP) of the coronavirus just 20 days after obtaining the SARS-CoV-2 gene. Production of the VLP is the first step in developing a vaccine for COVID-19, which will now undergo preclinical testing for safety and efficacy. Pluristem Therapeutics Inc. signed a collaborative agreement with the BIH Center for Regenerative Therapy and the Berlin Center for Advanced Therapies at Charite’ University of Medicine Berlin to expand its existing framework and research agreement and conduct a joint project evaluating the therapeutic effects of Pluristem’s patented PLX cell product candidates for potential treatment of the respiratory and inflammatory complications associated with the COVID-19 coronavirus. Vir Biotechnology Inc. is collaborating with the National Institute of Allergy and Infectious Diseases, Vaccine Research Center to develop monoclonal antibodies against coronaviruses, including SARS, MERS and SARS-CoV-2. It also plans to collaborate with Biogen Inc. to develop and manufacture human monoclonal antibodies to treat COVID-19 infected patients.
Sector ramps ups its R&D investments
According to an analysis conducted by BioWorld of the fourth-quarter and year-end 2019 financial reports filed by the top 100 public biopharmaceutical companies ranked by market cap, and excluding big pharma companies, the amount that was invested in research and development during the year increased 35% compared to the same period in 2018. The elevated spending reflects the continuing supportive financial environment and the relatively easy access to cash to support product development.
Deals of the week
Bridge Biotherapeutics Inc. has enlisted San Francisco-based AI drug discovery shop Atomwise Inc. to help it identify up to 13 small-molecule inhibitors of Pellino proteins and other targets in an effort to develop new therapies for inflammatory diseases. Atomwise stands to receive an up-front payment of undisclosed value, as well as milestone and royalty payments under terms of the deal. Presuming success, it estimates the partnership's payoff could reach up to $1.08 billion.
Bristol Myers Squibb Co. has partnered with digital therapeutics company Voluntis SA and plans to develop digital products to support cancer patients by enabling the reporting of and feedback on treatment-related symptoms, as well as remote monitoring by health care providers. Voluntis has previously structured similar pharma partnerships and aims to cultivate additional ones. Cancer patients often have adverse reactions to treatment; proactively and quickly managing these could help to reduce related hospitalizations, as well as improve the patient experience thereby supporting uninterrupted treatment as prescribed.
Forty Seven Inc. and Rocket Pharmaceuticals Inc. have agreed to collaborate on pursuit of clinical proof of concept for Forty Seven's antibody-based conditioning regimen, FSI-174 plus magrolimab, with Rocket’s ex vivo lentiviral vector hematopoietic stem cell gene therapy, RP-L102.
Public offerings: Ayala Pharmaceuticals Inc. has filed to raise up to $50 million in an IPO on Nasdaq intended to support clinical development of AL-101 and AL-102. AL-101, previously known as BMS-906024, is the company's lead candidate and is under study as a potential therapy for recurrent metastatic adenoid cystic carcinoma and triple-negative breast cancer. Compugen Ltd. priced its public offering grossing approximately $75 million. Imara Inc. sold 4.7 million shares at $16 in its IPO, grossing the company $75.2 million. Kala Pharmaceuticals Inc. priced an underwritten public offering for gross proceeds of about $126.2 million. Lyra Therapeutics Inc. has filed to raise up to $57 million in a yet-to-price Nasdaq IPO. Zentalis Pharmaceuticals LLC filed to raise up to $100 million in an IPO. The proceeds will support advancement of lead candidate, ZN-c5, an oral selective estrogen receptor degrader, currently in a phase I/II trial for the treatment of ER-positive/HER2-negative, advanced or metastatic breast cancer. Zogenix Inc. closed its underwritten public offering for gross proceeds of about $230.3 million.
Other public offerings: CTI Biopharma Corp. closed a rights offering in which it raised gross proceeds of about $60 million.
Venture funding: Life Sciences Partners BV (LSP), said its LSP 6 fund achieved a final close at a hard cap of $600 million, surpassing the fund’s $450 million target and giving LSP $1.1 billion to invest across LSP 6, the LSP Health Economics Fund and LSP's public fund and mandates. Investors comprised pension funds. Biosight Ltd. raised $19 million in a series C financing. Exuma Biotech Corp., which changed its name from F1 Oncology, raised $19 million in a series B round and will use the proceeds to develop its logic gated CAR T therapies. Harbour Biomed Therapeutics Ltd. raised $75 million in a series B+ round and will use the funds to continue development of its biotherapeutics for treating cancer and immunological diseases. Kymera Therapeutics Inc. closed a $102 million series C round. Numab Therapeutics AG closed a CHF22 million (US$23.8 million) series B financing to support a further broadening of its pipeline. Nurix Therapeutics Inc. closed a $120 million financing. Silverback Therapeutics Inc. completed an oversubscribed series B financing of $78.5 million to support its lead candidate, an anti-HER2 antibody conjugated to a TLR8 agonist for treating moderate or high HER2-expressing solid tumors.
The FDA and the FTC issued warning letters to seven companies for selling fraudulent COVID-19 products. These are unapproved drugs that pose significant risks to patient health and violate federal law, the agencies said. The warning letters are the first to be issued by the FDA for unapproved products intended to prevent or treat COVID-19. The jointly issued letters were sent to Vital Silver, Quinessence Aromatherapy Ltd., Xephyr LLC doing business as N-ergetics, Gurunanda LLC, Vivify Holistic Clinic, Herbal Amy LLC and The Jim Bakker Show.
The Institute for Clinical and Economic Review released an evidence report assessing the comparative clinical effectiveness and value of treatments for sickle cell disease, including Adakveo (crizanlizumab, Novartis AG), Oxbryta (voxelotor, Global Blood Therapeutics Inc.) and Endari (L-glutamine, Emmaus Life Sciences Inc.). The report details key clinical findings as well as cost-benefit analyses. According to ICER, the aggregate benchmark range for crizanlizumab pricing is $16,900 to $52,000 and would require a 61% to 89% discount off the treatment’s $132,000 annual list price, while the aggregate benchmark range for voxelotor, $8,300 to $36,500, would require a 71% to 93% discount off the $127,000 annual list price. The aggregate benchmark range for L-glutamine of $9,900 to $33,400 would require a discount of 18% to 76% off the $40,540 annual list price.
Word on the Street: Industry responses to the leading issues of the week
“We’re very encouraged by the level of interest around the world in accelerating research as part of the response. It is vital to coordinate research so that different groups around the world complement each other, so WHO can give better advice, and countries can take evidence-based decisions that save lives.”
Tedros Adhanom Ghebreyesus, WHO director general, announcing publication of the organization’s R&D roadmap
“Up-to-date, significant knowledge has been gained on how COVID-19 infects human cells. And with the experience we gained in the past on other viruses, we are confident in being able to generate antibodies to neutralize the function of COVID-19 and, thus, treat patients infected by the virus and prevent the infection in high-risk populations such as those with close contact with the patients, including health care workers.”
Jingsong Wang, the founder, chairman and CEO of Harbour Biomed Therapeutics Ltd., which has teamed up with Mount Sinai Health System to develop novel biotherapies in oncology and immunology
“Our sector should be preparing for significant global disruption to business through at least the first half of 2020. Members are making me aware of the impact on their businesses of the virtualization of Bio-Spring Europe, the cancellation of many business meetings by partners and disruption to their own supply chains.”
Steve Bates, chief executive of the U.K. Bioindustry Association