Biopharma companies that are focused on cell therapies have reported promising clinical trial results in their ability to treat acute respiratory distress syndrome (ARDS), a condition that has shown to be a significant contributor to higher mortality in COVID-19 cases. In an Alliance for Regenerative Medicine (ARM) webinar on the topic, Michael Lehmicke, director, science and industry affairs at ARM, noted that developers are utilizing mesenchymal stem cells (MSCs) and other stromal cells to treat ARDS caused by COVID-19. The number of clinical trials now underway continues to increase with many more at the preclinical stage.

COVID-19 developments

Amyris Inc. and the Infectious Disease Research Institute (IDRI) signed a binding term sheet for a planned COVID-19 RNA vaccine technology program that combines IDRI's expertise against infectious diseases with Amyris' fermentation platform technology, with the goal to create semisynthetic squalene-based adjuvants at scale.

Codagenix Inc. will collaborate with Open Orphan plc, a specialist contract research organization pharmaceutical services company, to conduct a phase I study of a vaccine using a human challenge model, which involves deliberately infecting healthy volunteers. The study of Codavax-COVID, a codon deoptimized SARS-CoV-2 single-dose, intranasal candidate, will include a second dose as a surrogate viral challenge on day 28.

Inovio Inc. said its COVID-19 DNA vaccine, INO-4800, was effective in protecting nonhuman primates (specifically rhesus macaques) from live virus challenge 13 weeks after the last vaccination. The protective results were mediated by memory T- and B-cell immune responses from INO-4800 vaccination.

Moderna Inc. dosed the first of what could be as many as 30,000 healthy volunteers as it began its phase III COVID-19 vaccine trial. Those volunteers at increased risk of contracting COVID-19, with a large percentage being elderly or people with co-morbidities – those most at risk for life-threatening COVID-19 infections – will receive placebo or Moderna’s vaccine candidate, mRNA-1273.

PDS Biotechnology Corp. said its COVID-19 vaccine candidate, Versamune-CoV-2 (PDS-0203), appeared to induce "highly active and potent virus-specific CD8 killer and CD4 helper T cells within 14 days of treatment" in a preclinical study. The candidate pairs the company's T-cell-activating Versamune platform with a recombinant protein recognized by the human immune system that is derived from SARS-CoV-2.

Sanofi SA and Glaxosmithkline plc made new agreements to supply the U.S. and U.K. governments with a COVID-19 vaccine. The two companies also are in advanced discussions with the European Union to supply up to 300 million doses of a vaccine. In the largest Operation Warp Speed (OWS) deal so far, Sanofi, of Paris, and London’s GSK will receive up to $2.1 billion from the U.S. government to develop and deliver an initial 100 million doses of a recombinant protein-based COVID-19 vaccine. OWS now has awarded COVID-19 contracts totaling more than $8 billion.

Deal transactions

Astrazeneca plc broadened its antibody-drug conjugate (ADC) relationship with Daiichi Sankyo Co. Ltd. in a potential $6 billion development and commercialization deal. Astrazeneca is paying $350 million up front, with a further $325 million at one year and $325 million two years into the deal. Another $1 billion will be paid in milestones tied to regulatory approvals, with $4 billion to follow in sales-related milestones. They will work together on DS-1062, Daiichi’s trophoblast antigen2 ADC, which is in phase I development in multiple tumors that express the cell surface glycoprotein. The agreement builds on a March 2019 $6.9 billion pact between the two for Enhertu (trastuzumab deruxtecan), the HER2-targeted ADC now on course for blockbuster status, following FDA approval in December and U.S. launch at the start of this year.

Amag Pharmaceuticals Inc. and Palatin Technologies Inc. mutually terminated their January 2017 license agreement that granted Amag exclusive rights to develop and commercialize Vyleesi (bremelanotide), a melanocortin receptor agonist indicated for the treatment of acquired, generalized hypoactive sexual desire disorder in premenopausal women, in North America.

Biontech SE and Regeneron Pharmaceuticals Inc. will collaborate on a clinical trial combining Biontech’s BNT-111, an mRNA cancer immunotherapy, and Libtayo (cemiplimab), a fully human anti-PD-1 therapy, for treating melanoma.

Centogene NV and Molecular Health GmbH will collaborate exclusively to initiate the Real-Life Data and Innovative Bioinformatic Algorithms (RIBA) project with epilepsy as the first indication. RIBA aims to foster a precision medicine environment to accelerate, de-risk and improve the development of new orphan drugs, based on the combination of large real-life datasets in rare disease with big data, artificial intelligence and computational know-how.

Cerevel Therapeutics Inc. and Cyclica Inc. started a research collaboration aimed at accelerating the discovery of medicines for neurological diseases. Cerevel will use Cyclica’s Ligand Express and Ligand Design artificial intelligence platforms to screen, identify, design and evaluate compounds directed at prespecified targets.

F-star Therapeutics Ltd. is gaining a Nasdaq listing via a reverse merger with Spring Bank Pharmaceuticals Inc., with F-star’s shareholders due to own 61% of the new entity, to be named F-star Therapeutics Inc. The deal, which has the agreement of both sides, requires F-star to raise $25 million in a private round at the point of closure, giving the merged company cash of $40 million for clinical development of its four programs over the next two to three years.

Sangamo Therapeutics Inc. signed up its sixth big pharma/biotech partner: Novartis AG. The three-target deal will use Sangamo's zinc finger protein transcription factors to up-regulate undisclosed genes to treat autism spectrum disorder and other neurodevelopmental disorders. Sangamo will receive $75 million up front and is eligible for up to $720 million in milestone payments; it is also due tiered royalties on sales of drugs developed through the collaboration.

UCB SA, a Belgian company developing an antibody targeting a toxic protein tied to Alzheimer’s disease (AD), said Roche Holding AG has negotiated an exclusive global license to the potential therapy for $120 million up front, plus almost $2 billion in milestone payments following positive proof of concept for the anti-tau candidate, UCB-0107, in AD.

Zentalis Pharmaceuticals Inc. will work with Eli Lilly and Co. to evaluate the combination of ZN-c5, its oral selective estrogen receptor degrader candidate, and Verzenio (abemaciclib), Lilly’s CDK4 and 6 inhibitor, in patients with ER-positive/HER2-negative advanced breast cancer.

Leading U.S. life sciences clusters maintain their rankings

There were no surprises among the latest rankings in JLL’s Life Sciences Outlook report, which tracks geographic shifts in life sciences innovation, operations and facilities investments, and includes an analysis of markets actively investing in their life sciences sectors. Boston, San Francisco and San Diego once again occupied the top three spots as the leading U.S. life science clusters. Companies located in those regions were also able to attract the lion's share of venture capital funding, which is one of the metrics JLL uses in its annual ranking system, along with such categories as market occupancy rate, employment concentration, employment growth, life sciences establishments concentration and average asking rent. Percentage weightings are assigned to each category to arrive at a final score for each cluster.

Financial transactions

Public offerings: Arcturus Therapeutics Holdings Inc. priced an underwritten public offering for gross proceeds of approximately $173 million. Aurinia Pharmaceuticals Inc. closed its offering for gross proceeds of approximately $200 million. Biontech SE closed its underwritten offering for gross proceeds of approximately $512 million. Bioxcel Therapeutics Inc. raised gross proceeds of $200 million. Essa Pharma Inc. closed its public offering netting the company approximately $45 million. TCR2 Therapeutics Inc. raised gross proceeds of $124 million. Zentalis Pharmaceuticals Inc. priced a public offering for gross proceeds of about $144.4 million.

Initial public offerings: Allovir Inc., a late clinical-stage cell therapy company, priced its upsized IPO of 16.25 million shares of common stock at $17 per share for gross proceeds of about $276.25 million. Annexon Inc. closed its IPO for gross proceeds of approximately $250.8 million. Harmony Biosciences Holdings Inc. filed with the SEC for an IPO of up to $100 million. Inozyme Pharma Inc. closed its IPO grossing the company $128.8 million.

Venture funding: Biotheryx Inc. closed a $35 million series D financing. Versant Ventures is committing $35 million in series A funding to Bright Peak Therapeutics Inc. Enthera Pharmaceuticals Srl closed a €28 million (US$32.9 million) series A round to take programs in type 1 diabetes and inflammatory bowel disease (IBD) into phase I development. MBX Biosciences Inc. raised $34.6 million in a series A financing. Nura Bio Inc., a company working to discover and develop new neuroprotective medicines, has closed a $73 million series A financing. Omega Therapeutics Inc. completed an $85 million financing. Praxis Precision Medicines Inc. raised approximately $110 million in a series C1 financing. Vico Therapeutics BV raised $31 million in its series A financing round to advance therapies for rare CNS diseases.

Regulatory news

The U.S. Presidential Advisory Council on Combating Antibiotic-Resistant Bacteria will hold a virtual meeting Sept. 9 and 10 to discuss the impact of COVID-19. Topics to be discussed will include the COVID-19 mortality rate due to secondary acquired infections, antibiotic stewardship practices during a pandemic, and the intersection of antimicrobial resistance and emergency preparedness, according to a notice to be published in the Federal Register.

The FDA issued a draft guidance on setting endotoxin limits during development of oncology drugs administered parenterally, except for intraocular administration, to treat serious and life-threatening cancers based on histology or stage of disease. The draft looks at a risk-based approach of weighing the potential risks of not evaluating endotoxin levels in all components of a multidrug regimen against the potential benefits to patients. Comments on the guidance should be submitted by Sept. 27, 2020.

The FDA issued a report on fiscal 2021 PDUFA user fees. The base revenue amount is about $1.06 billion, prior to adjustments for inflation, capacity planning, additional full-time employees, operating reserve and additional direct costs. Officials will adjust for inflation increases for the year by using two separate measures, one for personnel compensation and benefits (PC&B) and one for non-PC&B costs.

The NIH is seeking interest in collaborative research to develop autologous CD22 CAR T cells (m971BBZ lentivirus transduced) for the treatment of B-cell acute lymphocytic leukemia. The goal is the transfer of clinical development of the CD22-CAR program from National Cancer Institute to a drug development company. The collaborator will be responsible for the rapid scale-up and clinical manufacture of the agent to support the pivotal trial and subsequent BLA.


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

“We've approached this challenge really at two levels. First, picking vaccination – vaccinating centers and experts in areas of current and anticipated exposure to infection. Think of this as figuring out what the right ZIP code to go after or to go to. And second, working with the local investigators and communities in those ZIP codes so that we can enable those who are at highest risk for both infection and disease to have the opportunity to enroll.”
Tal Zaks, chief medical officer, Moderna Inc., speaking on a conference call on the dosing of the first of what could be as many as 30,000 healthy volunteers who will receive placebo or its COVID-19 vaccine candidate, mRNA-1273

“Based on our animal data, we expect this vaccine to be effective with a single dose but will also evaluate a repeat dose to assess potential for boosting and as a model for protection from wild-type challenge.”
Sybil Tasker, chief medical officer, Codagenix Inc., which will collaborate with Open Orphan plc to conduct a phase I study of Codavax-COVID using a human challenge model, which involves deliberately infecting healthy volunteers

“The technical thresholds for developing and manufacturing biosimilars are high. There are only a handful of biopharmaceutical companies in the world capable of entering the EU and U.S. markets to compete.”
Scott Liu, CEO, Henlius Biotech Inc., which won EMA approval for its Zercepac, a biosimilar to Roche Holding AG’s Herceptin (trastuzumab)



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