According to an analysis conducted by BioWorld of the first quarter 2020 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 (R&D) during the period increased by 13% compared to the same period last year. The elevated investment is reflective of the cash resources available to companies. For example, in the first quarter, follow-on offerings generated approximately $8.5 billion compared to $6.9 billion raised in the same period last year. The analysis found that the 100 biopharmaceutical companies collectively invested a total of $9.7 billion on R&D compared to $8.6 billion invested during Q1 2019.

COVID-19 developments

EMA Director General Guido Rasi has hit out at the lack of coordination in COVID-19 clinical trials, questioning if the huge number of small studies that are running will support regulatory decision-making.

Aetion Inc. signed a research collaboration agreement with the FDA to use real-world data to advance the understanding of and response to COVID-19. Astrazeneca plc is to get up to $1.2 billion from the new U.S. COVID-19 vaccines program, Operation Warp Speed, to support further development and manufacturing of a vaccine developed at Oxford University’s Jenner Institute. The company said it will begin to ship the product in September, with the U.K. and U.S. first in line for deliveries. Be The Match Biotherapies LLC is providing donor material for Nantkwest Inc.’s phase Ib trial to address acute respiratory distress syndrome. Cellect Biotechnology Ltd. said it signed a development agreement with an international consortium to examine the therapeutic effects of Apograft-treated stem cells on the reduction of pulmonary manifestations caused by COVID-19. The COVID-19 Research Outcomes Worldwide Network (CROWN) Collaborative, is testing whether the antimalaria drug chloroquine can prevent COVID-19 infection or decrease its severity in front-line health care workers, according to the Washington University School of Medicine in St. Louis, which is coordinating the study. An estimated 30,000 participants from across the globe will participate in the five-month trial. E-therapeutics plc started testing on the first set of compounds for the treatment of COVID-19 identified using its Network-driven Drug Discovery platform. The company will work with Wuxi Apptec Co. Ltd. to perform the studies. Kancera AB is the latest contender to reposition a clinical-stage small molecule with an immunomodulatory mechanism as a drug candidate for COVID-19. The Stockholm-based firm is planning a phase II trial of its fractalkine receptor inhibitor, KAND-567, to prevent hyperinflammation in COVID-19 patients before they develop full-blown acute respiratory distress syndrome. Mercaptor Discoveries Inc. said it is pivoting its neurotherapeutic development plan toward COVID-19. Mercaptor's lead Capton product inhibits COX2, an upstream component of the inflammatory response, thereby restricting the cytokine storm that leads neuroinflammation, protecting the patient from pulmonary and cardiac collapse. Vir Biotechnology Inc. identified and characterized S309, an antibody isolated from a patient who recovered from severe acute respiratory syndrome in 2003. The antibody has been shown to prevent SARS-CoV-2 live virus infection of cells. Vir is advancing two clinical development candidates based on the S309 antibody as potential therapeutics for COVID-19, VIR-7831 and VIR-7832, in collaboration with London-based Glaxosmithkline plc.

Clinical trials

Women, black and Hispanic/Latinx participants were underrepresented in pivotal clinical trials for drugs approved from 2007 to 2017, according to a new report by the Tufts Center for the Study of Drug Development.

Drug approvals

Evofem Biosciences Inc. won FDA approval for pregnancy prevention candidate, Phexxi, the vaginal pH regulator. It's the first non-hormonal contraceptive product in decades, according to the company. Combining lactic acid, citric acid and potassium bitartrate, it inhibits the motility of sperm.

Deals of the week

Amag Pharmaceuticals Inc. completed sale of its rights to Intrarosa (prasterone) to Millicent Pharma Ltd. for up to $125 million, including up-front fixed consideration of $20 million and contingent, sales-based milestone payments of up to $105 million. Assertio Therapeutics Inc. completed its merger with Zyla Life Sciences Inc. and will operate under the Assertio name. Beigene Ltd. signed an exclusive distribution agreement for Medison Pharma Ltd. to commercialize its BTK inhibitor, Brukinsa (zanubrutinib), in Israel. The University of Texas MD Anderson Cancer Center and Innovent Biologics Inc. formed a strategic collaboration agreement to co-develop Tyvyt (sintilimab), Innovent’s anti-PD-1 monoclonal antibody, in rare cancers in the U.S. Kleo Pharmaceuticals Inc. and Celularity Inc., a developer of allogeneic natural killer cell therapies, have entered a preclinical research collaboration to advance synergistic combinations of each company’s technology platform as potential treatments for COVID-19 and multiple myeloma. Qingdao Primedicine Pharmaceutical Co. Ltd. and Primary Peptides Inc. entered an exclusive license to develop and commercialize an innovative product for the treatment of heart attack. Roche Holding AG said it acquired Stratos Genomics Inc., an early stage sequencing technology company, to advance the development of its nanopore sequencer. Roche gains access to Stratos’ chemistry, sequencing by expansion (SBX). The Roche nanopore sequencer, once developed, will combine electronic and biological components to sequence DNA for clinical diagnostic testing. Surface Oncology Inc. signed a clinical trial collaboration with Merck & Co. Inc. to evaluate the safety and efficacy of combining Surface’s SRF-617, an antibody therapy targeting CD39, with Merck’s Keytruda (pembrolizumab), an anti-PD-1 therapy. Vividion Therapeutics Inc. has signed a multitarget collaboration deal with Roche Holding AG, which will use its platform and small-molecule library to target E3 ligases and a range of oncology and immunology targets. Vividion gets $135 million cash up front and could receive, according to the company, “several billion” in payments. Xenetic Biosciences Inc. entered a research funding and option agreement with Scripps Research to advance the development of the XCART CAR T technology for B-cell malignancies.

Financial transactions

Public offerings: ADC Therapeutics SA closed its IPO for gross proceeds of approximately $267.6 million. Applied Molecular Transport Inc. filed to raise $100 million in an IPO. Axcella Health Inc. closed its public offering, grossing the company $60.1 million. Bellerophon Therapeutics Inc. priced its offering and a registered direct offering for expected gross proceeds of approximately $40 million. Bluebird Bio Inc. priced its public offering, grossing the company approximately $500 million. Contrafect Corp. priced its underwritten public offering for gross proceeds of approximately $52.5 million. Dynavax Technologies Corp. priced its underwritten public offering with gross proceeds expected to be approximately $70 million. Flexion Therapeutics Inc. priced its public offering that generated $90 million. Gamida Cell Ltd. raised $60 million from its offering. Gossamer Bio Inc. completed its public offering and priced $200 million of convertible senior notes due in 2027, collectively generating $325 million. Androgen receptor-related disease specialist Kiniksa Pharmaceuticals Ltd. closed its public offering, grossing the company $80 million. Kintor Pharmaceutical Ltd. raised $240 million on the Hong Kong Stock Exchange. The IPO was oversubscribed by 551 times. Krystal Biotech Inc. priced an underwritten public offering with gross proceeds expected to be about $125.1 million. Legend Biotech Corp. plans to raise up to $100 million to advance its BCMA-targeting CAR T candidate, LCAR-B38M, by listing on Nasdaq. Representing the highest amount ever raised by a public biopharma company on a U.S. exchange, Moderna Inc. priced a $1.34 billion follow-on offering to help fund worldwide manufacturing and distribution of its mRNA-1273 vaccine for COVID-19. Ocular Therapeutix Inc. priced an underwritten public offering for expected gross proceeds of approximately $45 million. Oyster Point Pharma Inc. closed a public offering for gross proceeds of about $120.8 million. Scpharmaceuticals Inc. priced its public offering grossing the company $50 million. Turning Point Therapeutics Inc. completed an underwritten public offering of common stock at $60 per share for gross proceeds of about $325 million.

Other public offerings: Diffusion Pharmaceuticals Inc. closed its registered direct offering, priced at-the-market under Nasdaq rules, for gross proceeds of approximately $12 million. Surface Oncology Inc. raised gross proceeds of approximately $28.9 million through its at-the-market facility. Tricida Inc. priced an offering of $175 million aggregate principal amount of its 3.50% convertible senior notes due 2027 in a private offering to qualified institutional buyers.

Venture funding: Amplyx Pharmaceuticals Inc. closed a $53 million series C extension, bringing the total for the series C round to more than $90 million. Atea Pharmaceuticals Inc. raised $215 million from a series D financing to support development of its COVID-19 antiviral, which is designed to inhibit the viral RNA polymerase enzyme. Calcimedica Inc. raised $15 million in a series C financing round. Cowen Healthcare Investments completed fundraising for Cowen Healthcare Investments III LP with $493 million of committed investments. Nautilus Biotechnology Inc. raised $76 million in a series B round. Octant Inc. raised $30 million in a series A financing. Open Orphan plc has raised £12 million (US$14.6 million) in a placing to accelerate development of COVID-19 challenge study models. SQZ Biotechnologies Co. said it completed a $65 million series D preferred stock financing.

Washington news

Responding to COVID-19’s wakeup call as it exposes the risks of relying heavily on foreign biopharma supply chains, the U.S. Department of Health and Human Services (HHS) signed a four-year, $354 million agreement with a team of private industry partners, led by Phlow Corp., to expand U.S. manufacturing of essential medicines at risk of shortage during the pandemic and in future public health emergencies. The HHS’ Biomedical Advanced Research and Development Authority (BARDA) contract, which can be extended for a total of $812 million over 10 years, could disrupt generic competition for essential medicines in the U.S. by bringing more drug and active pharmaceutical ingredient manufacturing back to America and introducing more efficient manufacturing technologies, while rallying the nonprofit sector of generic drug development.

Word on the Street: Industry responses to the issues of the week

“This is something very, very important. We have been engaged, with a lot of frustration. Clinical trial authorization is not for EMA, it is member states’ prerogative; we really tried hard to foster [coordination]. It could have been done faster with a much bigger trial, with a single protocol.”
EMA Director General Guido Rasi told the European Parliament’s public health committee, questioning if the huge number of small studies that are running will support regulatory decision-making

“One (Not So) Small Step for MRNA, One (Potential) Giant Leap for the Market...Promising phase I COVID-19 Vaccine Data.”
Headline of a research report from Cory Kasimov, analyst at J.P. Morgan, on preliminary phase I results from Moderna’s COVID-19 vaccine candidate, mRNA-1273

“BCL-2 is as an important mediator in restoring the normal apoptotic process in tumor cells, making this protein a vital target for the treatment of cancer. ZN-d5, a BCL-2 inhibitor, may offer patients with hematologic and epithelial malignancies a differentiated therapy option, both as a monotherapy and in combination with other agents, including our oral selective estrogen receptor degrader, ZN-c5.”
Anthony Sun, CEO, Zentalis Pharmaceuticals Inc., which raised $20 million in a series A financing round to support a Chinese joint venture called Zentera Therapeutics

“This contract with Astrazeneca is a major milestone in Operation Warp Speed’s work toward a safe, effective, widely available vaccine by 2021”
Alex Azar, secretary of the U.S. Department of Health and Human Services

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