Amid the intense activity related to COVID-19 research and development, the FDA did make some time to approve several new medicines. Ahead of the scheduled Aug. 20 PDUFA date, Seattle Genetics Inc. won FDA clearance for the oral small-molecule breast cancer therapy tucatinib, a tyrosine kinase inhibitor branded Tukysa. Designed as highly selective for HER2 without significantly knocking down EGFR, Tukysa proved its worth in the HER2Climb study, which compared Tukysa in combination with Herceptin (trastuzumab, Roche Holding AG) plus capecitabine to Avastin/capecitabine in patients with locally advanced unresectable or metastatic HER2-positive disease.

Following a priority review, partners Astrazeneca plc and Merck & Co. Inc. gained a green light from the FDA for U.S. marketing of the oral MEK1/2 inhibitor Koselugo (selumetinib), the first FDA-approved treatment for the rare genetic disorder neurofibromatosis type 1 (NF1).

Urogen Pharma Ltd.’s mitomycin gel, an orphan drug branded Jelmyto, also received FDA approval three days ahead of the April 18 PDUFA date, offering patients the first non-surgical option for low-grade upper tract urothelial cancer (LG-UTUC) and granting the company with its first marketed product.

Deals of the week

In a $2 billion deal, the Blackstone Group Inc. will acquire 50% of royalties owed to Alnylam Pharmaceuticals Inc. on the global sales of inclisiran, which is designed to treat hypercholesterolemia. The twice annually injectable proprotein convertase subtilisin/kexin type 9 (PCSK9) gene inhibitor is being reviewed by the FDA. The drug could become the first siRNA based PCSK9 inhibitor to win FDA approval.

Legochem Biosciences Inc. has partnered with Iksuda Therapeutics Ltd. to develop oncological antibody-drug conjugates (ADC). Under the global research collaboration and license agreement, Legochem is set receive up to $407 million from development, regulatory and commercial milestone payments, as well as royalties on the sales of any resulting ADC products. It is also entitled to a prearranged percentage of sublicense revenue should Iksuda sign a license agreement with other companies. Iksuda gained certain rights to use Conjuall, Legochem’s linker and conjugation platform, as well as Legochem cancer-selectively activated payloads, to research, develop and commercialize targeted cancer therapeutics.

MEI Pharma Inc. signed a deal with Kyowa Kirin Co. Ltd. that is focused on phase II-stage ME-401 for B-cell malignancies. The arrangement means $100 million up front for MEI, which could bank up to $582.5 million more if development, regulatory and commercial goals are reached.

COVID-19 research and trials

7 Hills Pharma has launched a COVID-19 vaccine program for older adults. It will evaluate its lead compound, 7HP-349, a next-generation small-molecule integrin activator, as an oral adjuvant with a novel recombinant coronavirus vaccine, laying the foundation for potential clinical testing late this year. 48Hour Discovery Inc., a University of Alberta biotechnology spin-off company that develops peptide-based therapeutic drugs from billion-scale libraries of molecules, said it initiated drug discovery projects for therapeutic targets related to COVID-19. AI Vivo said its systems pharmacology platform powered by artificial intelligence has identified the top candidate drugs that are highly likely to be effective in treating COVID-19 disease. Five of the top-ranked drugs on the list that have already entered clinical trials for COVID-19, include: chloroquine, dexamethasone, sirolimus, tacrolimus and thalidomide. Applied DNA Sciences Inc. completed design qualification, production and shipment of five COVID-19 vaccine candidates to Takis Biotech Srl. The vaccine candidates were produced by the company’s LinearDNA manufacturing systems. Fujifilm Corp. said it increased manufacturing capacity at its Toyama Chemical Co. Ltd. and Wako Pure Chemical Co. Ltd. sites to increase production of RNA polymerase inhibitor Avigan (favipiravir) for treating COVID-19 patients.

The U.S. Biomedical Advanced Research and Development Authority has committed up to $483 million to accelerate Moderna Inc.’s mRNA vaccine candidate, mRNA-1273, in efforts to fight coronavirus (SARS-CoV-2). The support would enable the company to supply millions of doses per month in 2020 and tens of millions per month in 2021 if the vaccine candidate is successful in the clinic.

Biogen Inc. said that together with Broad Institute of MIT and Harvard, and Partners Healthcare a consortium has been established that will build and share a COVID-19 biobank that will help scientists study a large collection of de-identified biological and medical data to advance knowledge and search for potential vaccines and treatments. Stony Brook Medicine has launched an FDA-approved, randomized investigational new drug trial to determine whether plasma from people who have recovered from COVID-19 can aid in the recovery of patients currently fighting the disease. As part of that effort, the Long Island medical school is partnering with Chembio Diagnostics Inc., whose COVID-19 rapid serological point-of-care test will be used to identify potential plasma donors. CNS Pharmaceuticals Inc. said independent research by the University of Frankfurt found 2-deoxy-D-glucose (2-DG) reduced replication of SARS-CoV-2 by 100% in in vitro testing. The research shows, CNS said, that inhibiting glycolysis with non-toxic concentrations of 2-DG completely prevented SARS-CoV–2 replication in Caco-2 cells. Cytodyn Inc. reported that more than 25 COVID-19 patients were administered leronlimab under the FDA’s emergency investigational new drug authorization. The rate of response, the company said, in mild to moderate patients has been “very promising,” with the first five patients treated being removed from oxygen. Gilead Sciences Inc. said a cohort analysis showed most of the 53 individuals hospitalized with severe complications of COVID-19 infection who were treated with the antiviral remdesivir on an individual compassionate use basis improved clinically with no new safety signals. Nearly two-thirds (64%, n=34/53) were on mechanical ventilation at baseline, including four also on extracorporeal membrane oxygenation. Treatment resulted in improved oxygen support class for 68% (n=36/53) over a median follow-up of 18 days from first dose. More than half on mechanical ventilation were extubated (57%, n=17/30) and 47% (n=25/53) were discharged from the hospital following treatment.

Halix BV joined a consortium guided by the University of Oxford to provide GMP manufacturing services supporting large-scale production of the COVID-19 vaccine ChAdOx1 nCoV-19, which is being developed by the university’s Jenner Institute. Macromoltek Inc. has applied its computational design technology to help its partner, Cytovia Therapeutics Inc., develop a potential treatment of COVID-19. Redhill Biopharma Ltd. said following the preliminary findings of clinical improvement in the first two COVID-19 patients treated with opaganib (Yeliva, ABC-294640) in Israel, three additional patients have been treated with opaganib, four of whom have demonstrated measurable improvement within days following treatment initiation. All five patients suffered from moderate to severe acute respiratory symptoms related to SARS-CoV-2 infection, required supplemental oxygenation and were hypoxic despite being treated with supplemental oxygen. Starpharma Ltd. said its Vivagel active astrodimer sodium (SPL-7013) was shown in laboratory studies to have significant antiviral activity against the coronavirus that causes COVID-19.

Companies represented in the expert group brought together by the World Health Organization (WHO) to work on the development of COVID-19 vaccines have signed a pledge to strengthen collaboration and sharing of data. In a declaration on WHO’s website, companies including Sanofi SA, Moderna Inc., Inovio Inc., Clover Biopharmaceuticals Inc., Cansino Biologics Inc., Curevac AG and Novavax Inc., that have some of the most advanced vaccine candidates, say they will continue efforts to strengthen the “unprecedented” cooperation, “to help reduce inefficiencies and duplication of effort.”

Financial transactions

Public offerings: Athersys Inc. priced its underwritten public offering for gross proceeds of approximately $50.1 million. Calithera Biosciences Inc. priced its underwritten public offering, with gross proceeds expected to be $31.25 million. Crinetics Pharmaceuticals Inc. priced an underwritten public offering of 7.15 million shares at $14 each. The underwriters have been granted a 30-day option to purchase up to an additional 1.07 million shares. The gross proceeds are expected to be $100.1 million. Keros Therapeutics Inc. closed its IPO for total gross proceeds of $110.4 million. Lantern Pharma Inc. filed to raise up to $29 million in an IPO.

Other public offerings: Coherus Biosciences Inc. priced an offering of $200 million aggregate principal amount of 1.5% convertible senior subordinated notes due 2026 in a private offering. Scynexis Inc. entered a common stock purchase agreement for up to $20 million with Aspire Capital Fund LLC. Stealth Biotherapeutics Corp. completed a $20 million private placement with Morningside Venture (I) Investments Ltd.

Venture funding: 54gene has raised $15 million in series A funding. Avectas Ltd. completed a $20 million series C financing. Cerevance Inc. raised $45 million in a series B round designed to propel the discovery and development of therapies for treating CNS diseases such as Alzheimer’s disease. Qiming Venture Partners said that it has established a new $1.1 billion fund to target early stage health care and technology investments.

Regulatory news

The FDA is extending the deadline until June 5, 2020, to apply for a funding opportunity to examine the use of real-world data (RWD) to generate real-world evidence (RWE) in regulatory decision-making. The agency said it is particularly interested in projects that compare the use of RWD and generation of RWE with more traditional approaches and methods for data collection and evidence generation. The scope of the funding opportunity includes projects that focus on exploring and conducting innovative clinical trials, explaining ways to address challenges to using RWD in research studies, exploring the use of innovative technologies, designing and conducting pilot projects, determining endpoints that RWD can capture, and evaluating reliability considerations around the use of RWD.

The push in Congress to drive down U.S. prescription drug costs has taken a backseat to all things COVID-19, but that reprieve for drug companies may be about to end as freshman members of the House urge their leaders to include drug pricing proposals in the next coronavirus relief bill. “We have consistently heard from our constituents that costs are prohibitive to obtaining their medications, a problem that is exacerbated during a crisis,” the lawmakers said in a letter to House Speaker Nancy Pelosi (D-Calif.) and Minority Leader Kevin McCarthy (R-Calif.). Provisions making prescription drugs both accessible and affordable should be a top priority in the relief package, the lawmakers said, pointing to the “tens of millions of Americans” who have lost their jobs and, thus, their health coverage because of COVID-19 shutdowns.

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

"Everyone's daily lives have been disrupted during the COVID-19 pandemic, and in this critical time we want patients to know that the FDA remains committed to making patients with rare tumors and life-threatening diseases, and their unique needs, a top priority."

Richard Pazdur, the FDA's Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA's Center for Drug Evaluation and Research, commenting on the agency’s approval of MEK1/2 inhibitor Koselugo (selumetinib), the first FDA-approved treatment for the rare genetic disorder neurofibromatosis type 1

"In addition to pushing out timelines, clinical trial disruptions also put significant up-front investment for companies at risk and delay potentially meaningful therapeutic agents from reaching patients, and we anticipate further setbacks as all trials for non-life-threatening conditions experience some form of pause."

RBC Capital Markets analyst Brian Abrahams

"Ongoing trials have potential to benefit millions of people with debilitating chronic diseases long after the coronavirus pandemic has ended. Furthermore, discontinuing ongoing trials wastes previously invested resources and squanders the time and effort of participants who may have already completed the trial."

Mary McDermott, of Feinberg School of Medicine at Northwestern University, and Anne Newman, of University of Pittsburgh Graduate School of Public Health, in a viewpoint published in JAMA

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