Although the product pipeline for vaccines and therapeutics targeting COVID-19 is top of mind right now, investors are also keeping a close eye on companies involved in the development of medicines targeting cancer and the central nervous system. According to financings tracked by BioWorld and deals and grants logged in Cortellis, the therapeutic areas of cancer, neurology and psychiatry attracted the highest amounts of investments last year with a collective $101.9 billion and $27.5 billion raised, respectively. Public biopharma companies in those areas have also seen their share prices increase despite the uncertain environment pervading the general markets.
New product approvals
Immunomedics Inc. gained accelerated FDA clearance for Trodelvy (sacituzumab govitecan-hziy) to treat patients with metastatic triple-negative breast cancer (TNBC) who have undergone at least two prior therapies. It’s the first antibody-drug conjugate given the go-ahead specifically in relapsed/refractory TNBC and the first anti-Trop-2 ADC bound for the market. The FDA granted accelerated approval to Incyte Corp.'s Pemazyre (pemigatinib), the first treatment to gain the green light for adults with certain types of previously treated, advanced cholangiocarcinoma. Action on Pemazyre had been expected towards the end of May.
Deals of the week
Applied Biomath LLC has formed a collaboration with Northern Biologics Inc. to conduct pharmacokinetic and pharmacodynamic modeling of Northern Biologics' lead preclinical cancer antibody to help inform selection of a clinical starting dose. Asklepios Biopharmaceutical Inc. has acquired Brainvectis SAS, which has expertise and intellectual property related to treating neurodegenerative disorders. Brainvectis is focused on therapies that restore brain cholesterol metabolism to treat Huntington’s disease and related disorders. Cue Biopharma Inc. will collaborate with Merck & Co Inc., through a subsidiary, to evaluate the combination of CUE-101 with Merck’s anti-PD-1 therapy Keytruda (pembrolizumab) in first-line human papillomavirus-positive advanced head and neck cancer. Gilead Sciences Inc., Kite, a Gilead Co., and Onko-innate, have established a three-year cancer immunotherapy research collaboration to support discovery and development of next-generation drugs and engineered cell therapies focused on natural killer cells. Mogrify Ltd. and Sangamo Therapeutics Inc. have entered a collaboration and exclusive license agreement for Sangamo to develop allogeneic cell therapies from Mogrify’s induced pluripotent stem cells and embryonic stem cells and Sangamo’s zinc finger protein gene-engineered chimeric antigen receptor regulatory T-cell (CAR-Treg) technology. The deal provides Sangamo with access to Mogrify’s cell conversion technology, which will diversify options as the firms develop off-the-shelf allogeneic CAR-Treg cell therapies. Prothera Biologics Inc. and Takeda Pharmaceutical Co. Ltd. have entered a global licensing agreement to develop a plasma derived IAIP (Inter-alpha Inhibitor Proteins) therapy for the treatment of acute inflammatory conditions. Supernus Pharmaceuticals Inc. has struck an agreement with Navitor Pharmaceuticals Inc. to run a joint phase II program for Navitor's mTORC1 activator, NV-5138, in treatment-resistant depression. The company stands to earn up to $475 million if Supernus ultimately licenses or acquires the drug and the work hits certain milestones. Tracon Pharmaceuticals Inc. retained global rights to and has sole responsibility for development and commercialization of TRC-253 based on the decision by Johnson & Johnson not to exercise its option to reacquire global rights following a review of the phase II data in prostate cancer patients with acquired resistance to Xtandi (enzalutamide, Astellas Pharma Inc./Pfizer Inc.) or Erleada (apalutamide, Johnson & Johnson).
Adaptvac ApS said it is partnering with AGC Biologics, a contract development and manufacturing organization, to develop and produce a COVID-19 vaccine using its virus-like particle technology. Anixa Biosciences Inc. has formed a strategic collaboration with Ontochem GmbH to discover and develop antiviral drug candidates to treat COVID-19. The program will focus on two proteins of the coronavirus, including the main protease, Mpro, an enzyme that severs a large polypeptide into functional proteins that enable the virus to replicate in the human host, and an endoribonuclease that plays a role in breaking up the ribonucleic acid of the virus. Bayer AG said that Bayer Inc. is partnering with the Population Health Research Institute in launching a clinical research program to identify potential treatments for COVID-19. The studies will evaluate the safety and efficacy of different combination therapies, including Bayer’s chloroquine and interferon beta-1b. Cyclacel Pharmaceuticals Inc. has inked an agreement with the University of Edinburgh to study fadraciclib (CYC-065) and seliciclib (CYC-202 or R-roscovitine), its cyclin-dependent kinase-2/9 inhibitor candidates, to treat the inflammatory response observed in individuals with COVID-19. The partners will assess the candidates for suitability in safety and efficacy studies in COVID-19 patients as part of the university’s broader STOPCOVID project studying the inflammatory pathways that lead directly to COVID-19 lung injury. Inmune Bio Inc. will enroll patients in a new therapeutic program to treat patients with pulmonary complications from COVID-19 infection. The program will use the company’s dominant-negative TNF inhibitor platform to target hospitalized patients with hypoxia who do not yet require intensive respiratory support. Innocan Pharma Corp. entered a sponsored research agreement with Ramot at Tel Aviv University to develop a COVID-19 treatment using cannabidiol-loaded exosomes. Innocan will fund the research based on agreed milestones, for about $450,000 in the first stage. The technology targets both CNS indications and COVID-19. Innovation Pharmaceuticals Inc. said that, based on molecular screening of 11,552 compounds comprising already FDA-approved drugs and those in clinical testing, brilacidin, the company’s defensin-mimetic drug candidate targeting COVID-19, was identified as one of the most promising potential inhibitors of SARS-CoV-2. Mesoblast Ltd.’s shares were up nearly 39% last week on the news that its allogeneic cell therapy showed an 83% survival rate in ventilator-dependent COVID-19 patients with moderate to severe acute respiratory distress syndrome (ARDS) treated at New York’s Mount Sinai Hospital. Nine of the 12 treated patients (75%) have successfully come off ventilator support within 10 days following two infusions of remestemcel-L. Novartis AG has reached an agreement with the FDA to run a phase III trial evaluating hydroxychloroquine for the treatment of hospitalized patients with COVID-19, with enrollment of 400 patients beginning within weeks and results to be reported as soon as possible. The study will join a multitude of others, some of ever larger scale, that are testing the drug against the pandemic infection. Shenzhen Hepalink Pharmaceutical Group Co. Ltd. said its subsidiary, Cytovance Biologics, signed a collaboration agreement with Akshaya Bio Inc. to provide rapid access to clinical materials using Akshaya’s Chimigen platform technology for vaccine development for COVID-19 and hepatitis B virus. Valneva SE and Dynavax Technologies Corp. are collaborating to initiate a vaccine program targeting COVID-19 leveraging Valneva’s Ixiaro, a cell-derived vaccine for Japanese encephalitis, and Dynavax’s Toll-like receptor 9 agonist adjuvant, CpG 1018, which is used in hepatitis B vaccine Heplisav-B.
The Australian Government is providing AU$13 million (US$8 million) to fast-track research into treatments for COVID-19, and several promising candidates are about to enter the clinic. The funding from the Medical Research Future Fund will support rapid development of treatment options for COVID-19, with AU$8 million to identify and develop antiviral therapies for people infected with COVID-19, and AU$5 million for clinical trials to better treat and manage COVID-19 patients with severe acute respiratory distress.
The World Health Organization launched a global collaboration to accelerate development and production of COVID-19 vaccines, therapeutics and diagnostics, with leaders of countries around the world appearing live to pledge their support.
Allterum Therapeutics Inc. closed a $2.9 million product development award by the Cancer Research and Prevention Institute of Texas to support preclinical development of its 4A10 antibody therapy for the treatment of pediatric patients suffering from T-cell acute lymphoblastic leukemia. The technology was licensed from the National Cancer Institute.
VBL Therapeutics said it received a nondilutive grant of up to ILS 3.175 million (US$900,000) from the Israel Innovation Authority. The funds will support the continued development of VBL's lead product candidate, VB-111, a targeted cancer gene therapy agent that is being evaluated in a phase III potential registration study to treat platinum-resistant ovarian cancer.
Public offerings: Actinium Pharmaceuticals Inc. priced an underwritten public offering for expected gross proceeds of approximately $27.5 million. Arcturus Therapeutics Holdings Inc. raised gross proceeds of $80.5 million from an underwritten public offering. Akeso Inc. launched an IPO on April 24 in Hong Kong to reap HK$2.4 billion (US$314 million), even though the economy is taking a hard hit from the COVID-19 pandemic. The proceeds will help advance its PD-1/CTLA4 bispecific antibody for cervical cancer, aiming to be on the market by late 2021. Crinetics Pharmaceuticals Inc. closed its underwritten public offering for gross proceeds of approximately $115.1 million. Cyclacel Pharmaceuticals Inc. priced a public offering with expected total gross proceeds of approximately $20 million. Oric Pharmaceuticals Inc. priced its IPO of 7.5 million shares of common stock at $16 each. VBI Vaccines Inc. priced an underwritten public offering for expected gross proceeds of approximately $50 million.
Other public offerings: Immunic Inc. said it sold 1.76 million shares at $8.50 each in a registered direct offering.
Venture funding: Accent Therapeutics Inc. completed a $63 million series B financing. With completion of a $120 million series B financing, Affinivax Inc. said it's poised to advance several new vaccine candidates for hospital-associated infections into the clinic. Genespire Srl, a Milan-based startup led by gene therapy pioneer Luigi Naldini, is the third company to receive funding from Sofinnova Partners’ Italian €108 million (US$117.5 million) gene therapy fund, the Sofinnova Telethon Fund. Swanbio Therapeutics Inc. completed a $52 million expanded series A financing designed to boost the company’s R&D of adeno-associated virus (AAV)-based gene therapies to treat adrenomyeloneuropathy (AMN) and other rare, monogenic neurological diseases.
The U.K. treasury announced a £500 million (US$622.5 million) COVID-19 bailout plan for research-based startups, which could see the government holding equity stakes in venture capital-backed firms. The aid will come in the form of a government loan of between £125,000 and £5 million, which must be matched by an equivalent private investment. Those loans will automatically convert into equity on a company’s next qualifying funding round, at a 20% discount to the valuation, or at the end of the loan if they are not repaid. To be eligible, a business must be an unlisted U.K. registered company that has previously raised at least £250,000 in equity investment from third-party investors in the last five years.
The Swiss biotech sector raised about CHF1.2 billion (US$1.2 billion) in equity investment in 2019, a significant downward dip on the last two years, but well ahead of its average annual raise of CHF800 million over the past decade, according to the newly published Swiss Biotech Report 2020. Privately held firms took in CHF577 million while publicly listed firms raised another CHF614 million. The comparable totals for 2018 and 2017 were CHF1.5 billion and CHF1.7 billion, respectively.
The FDA created the Coronavirus Treatment Acceleration Program (CTAP) to evaluate proposals for possible therapies. Under CTAP, the agency said it’s using “every available method to move new treatments to patients as quickly as possible, while at the same time finding out whether they are helpful or harmful.”
The FDA issued a guidance on a temporary policy allowing state-licensed pharmacies and federal facilities that are not registered outsourcing facilities to compound certain drugs for hospitalized patients during the COVID-19 public health emergency. “In light of unprecedented disruptions to, and demands on, the global pharmaceutical supply chain as a result of the COVID-19 pandemic, and in order to respond to evolving regional conditions, additional flexibility is temporarily needed to help ensure that treatment options are available when hospitals are unable to obtain FDA-approved drugs used for hospitalized patients with COVID-19,” according to the guidance. Under the temporary policy, the compounded drug must be provided directly to a hospital that is treating COVID-19 patients and that is unable to get adequate supplies of certain FDA-approved drugs or drugs compounded at a registered outsourcing facility. The compounded drugs must be labeled with a default by-use-date. State-licensed pharmacies must have state permission before compounding the drugs.
Word on the Street: Industry responses to the issues of the week
“By definition, we’re probably not going to be able to vaccinate everyone simultaneously. If you had a situation where you do still have virus circulating, one could imagine that, as vaccine runs off the fill-and-finish line, you might be able to set up a trial whereby essentially what you’re doing is using real-world evidence.”
Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, commenting on vaccine studies that might not be done in clinics but in the streets
“BIA, along with representative bodies across the science and tech community, have been calling for this, after it became clear that existing government support measures would not be accessible to our sector. The chancellor’s statement will enable U.K. biotech companies to emerge from the COVID-19 disruption in a strong position to continue their growth.”
Steve Bates, chief executive of the Bioindustry Association (BIA), commenting on a U.K. £500 million (US$622.5 million) COVID-19 bailout plan for research-based startups
“What’s exciting is that our patients in the same epicenter of this disease with the same treatment everyone else is getting, suddenly 75% are coming off ventilators within 10 days, and we’ve got 83% survival. At the same time, the kind of drugs that are being touted by big pharma are gone. You have hydroxychloroquine that every man and his dog were talking about, and that came out in a randomized controlled trial and it failed, and maybe it’s killing people. We’re tackling the hardest of the hard, which these pharma companies have got nothing for, and we’re getting these survival outcomes.”
Silviu Itescu, CEO, Mesoblast Ltd., whose allogeneic cell therapy, remestemcel-L., showed an 83% survival rate in ventilator-dependent COVID-19 patients with moderate to severe acute respiratory distress syndrome