Alligator Bioscience AB, of Lund, Sweden, said the company will increase its focus on its clinical development portfolio and reduce investments in nonclinical activities. The new strategy, expected to be fully implemented by the third quarter, will mean employee reductions impacting 12 positions, corresponding to more than 20% of the company's personnel. The measures will result in an annual cost reduction of more than SEK80 million (US$11.1 million), allowing the company to extend its cash runway for another 18 months.
Applied Biology Inc., of Irvine, Calif., said, based on a joint effort with Brown University researchers it has been concluded that the androgen receptor may be implicated in COVID-19 mortality risk; therefore, human type II pneumocytes androgen sensitivity should be tested as a potential treatment marker. The team submitted their discovery to publication in Dermatologic Therapy.
Beam Therapeutics Inc., of Cambridge, Mass., has licensed rights to the lentiviral transduction enhancer Lentiboost from Martinsried, Germany-based Sirion Biotech GmbH to use in its CAR T-cell products. Sirion will be entitled to undisclosed up-front and milestone payments and be eligible to receive royalties on future product net sales plus license fees tied to commercial success.
Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn., said it is collaborating with Cove, a company providing specialized care and access to innovative treatments for migraine sufferers via telemedicine services in the U.S. The arrangement will allow telemedicine evaluation for migraine sufferers during those times of limited access to routine office visits. Using Cove, patients consult with independent health care providers and may be prescribed the company’s recently FDA approved Nurtec ODT, a calcitonin gene-related peptide receptor antagonist, along with other migraine treatments.
Chromocell Corp., of North Brunswick, N.J., is making its Chromovert technology available to help accelerate research related to COVID-19 treatment and prevention. Chromovert, which uses rare cells that the company said are ideally suited for effective high-throughput screening, already has been successfully used to identify a novel, non-addictive pain blocker that is in clinical development. Also, Chromocell has identified a number of compounds in its pipeline that may help address COVID-19-related disease, and is submitting proposals to various governmental agencies in response to their recent calls for new programs.
Cullgen Inc., of San Diego, said its internal program to develop selective degraders that target key BRAF mutant proteins has been accepted for publication by the Journal of Medicinal Chemistry. The potentially first-in-class degraders developed by Cullgen efficiently degrade mutated, disease-causing forms of the BRAF protein and subsequently inhibit cancer cell growth, the company said. The company uses Usmite technology to identify and develop highly selective degraders for the treatment of cancer and other diseases.
Cytodyn Inc., of Vancouver, Wash., is collaborating with the U.K.’s Department of Health to provide emergency access to CCR5 antagonist leronlimab, an investigational medicine for COVID-19. The submission to the U.K. Medicines and Healthcare Products Regulatory Agency is expected to be made soon to include clinical trial sites in the U.K. Representatives from the U.K. are coordinating with Cytodyn’s international clinical research organization, Amarex Clinical Research, to prepare the requisite clinical agreements and to prepare for timely delivery of leronlimab. The FDA recently cleared the company to initiate a phase IIb/III trial for severe and critically ill COVID-19 patients for which enrollment is now underway.
Cytovia Inc., of New York, is expanding its programs to help urgently address the current COVID-19 crisis. The company works with natural killer (NK) cells. Using a bifunctional approach has the potential to minimize virus escape from the immune response, thereby inhibiting the intensification of the inflammation leading to acute respiratory syndrome, the company said. Specifically, the activation of NK cells through the NKp46 receptor aims to destroy the virus-infected cells while the other arm can either block the entry of the virus into epithelial cells or neutralize circulating viruses. The company is partnered with Austin, Texas-based Macromoltek Inc., an in-silico antibody drug design firm.
Gilead Sciences Inc., of Foster City, Calif., completed the transaction for Toro Merger Sub, Inc., a wholly owned subsidiary of Gilead, to acquire Forty Seven Inc., of Menlo Park, Calif., for $95.50 per share, net to the seller in cash, without interest, or about $4.9 billion.
Imaginab Inc., of Los Angeles, and ARTMS Products Inc., of Vancouver, British Columbia, entered a multiyear, nonexclusive partnership to explore a radiochemistry manufacture of Imaginab's lead asset, 89Zr CD8 Immunopet. ARTMS' technology will be deployed to enhance the manufacture of Imaginab's antibody, also known as 89Zr-Df-IAB22M2C. The CD8 Immunopet minibody binds to the CD8 receptor on human T cells and is used for non-invasive positron emission tomography (PET) imaging of CD8 T cells in patients as a pharmacodynamic and predictive marker for immunotherapy. Due to the zirconium-89 labeling of the minibody, it can be visualized by PET imaging.
Infinity Pharmaceuticals Inc., of Cambridge, Mass., said it is working with clinical trial investigators and sites to continue patient treatment with minimal interruption. New patient enrollment across its clinical programs is being assessed for each individual patient, the company said, adding that it expects some delays to patient enrollment and site initiation due to COVID-19. Infinity said it has not experienced any disruption to its drug supply for ongoing clinical trials and that it does not expect any to occur. Infinity has several studies underway for advancing IPI-549, an oral immuno-oncology candidate that selectively inhibits PI3K-gamma.
Ingeneron Inc., of Houston, said results of an FDA-approved, randomized, controlled, feasibility study of treating symptomatic partial-thickness rotator cuff tears (sPTRCT) with fresh, uncultured, unmodified, autologous adipose-derived regenerative cells (UA-ADRCs) show assessment at 24 and 52 weeks post treatment meant significantly higher mean American Shoulder and Elbow Surgeons Standardized Shoulder Assessment Form total scores (indicating reduced pain and improved shoulder function) of the subjects in the UA-ADRC group compared to the subjects in the corticosteroid group at both time points. The study, performed by Sanford Health, is the first randomized controlled trial in which cells isolated from patients’ own adipose tissue were compared to another therapy option for treating sPTRCT, the company said. The data were published in the Journal of Orthopaedic Surgery and Research.
Intellistem Technologies Inc., of San Francisco, said it has developed a peptide-based vaccine candidate (IPT-001) intended for use against COVID-19. The vaccine was developed using the novel coronavirus and engineered using the Intellipeptidome platform to target both the spike (S) and nucleocapsid (N) proteins. The company said it is in the process of an IND submission and in the final stages of preparing for a phase I trial, anticipated to start as early as September 2020.
Iqvia Holdings Inc., of Danbury, Conn., and Research Triangle Park, N.C., created what it calls the first comprehensive online screener and trial matching tool for all U.S. COVID-19 trials. The platform combines publicly available information, Iqvia data and tools, and an algorithm generating a questionnaire dependent on previous responses to match patients and investigators to accelerate ongoing COVID-19 clinical research projects in the U.S. The platform is designed to accelerate phases I to IV trials.
Karyopharm Therapeutics Inc., of Newton, Mass., said it plans to initiate a global randomized clinical trial for low-dose oral selinexor in hospitalized patients with severe COVID-19. Selinexor, marketed as Xpovio, is approved at higher doses by the FDA to treat relapsed or refractory multiple myeloma. Selinexor is an oral, selective inhibitor of nuclear export compound that blocks the cellular protein XPO1. It also facilitates transporting several viral proteins from the nucleus of the host cell to the cytoplasm1 and amplifies pro-inflammatory transcription factor activity. Selective inhibitor of nuclear export compounds may interfere with key host protein interactions with SARS-CoV-2, the virus that causes COVID-19, the company said.
La Jolla Pharmaceutical Co., of San Diego, said it is providing Giapreza (angiotensin II) for named patient emergency use in patients with septic shock due to COVID-19 at University Hospital Münster in Germany. Giapreza was recently approved by the European Commission but is not yet commercially available in Europe. According to German law, authorized medicines can be made available prior to commercial availability to fulfill special patient needs. In December 2017, Giapreza was approved by the FDA as a vasoconstrictor indicated to increase blood pressure in adults with septic or other distributive shock.
Leading Biosciences Inc., of Carlsbad, Calif., said it developed an approach to treat COVID-19 patients with acute respiratory distress syndrome and multiple organ dysfunction syndrome by using LB-1148, a serine protease inhibitor designed to neutralize the activity of potent digestive proteases that can cause a range of complications and organ dysfunction if they escape the gastrointestinal tract through a compromised mucosal barrier. Most forms of shock, including septic shock, feature disruption of the gastrointestinal mucosal barrier, leading to the escape of digestive enzymes accelerating inflammation, cytokine storm, autodigestion and organ failure. The drug is being developed to reduce recovery times and shorten hospital lengths of stay.
Lodo Therapeutics Corp., of New York, said it achieved a preclinical milestone in its 2018 multitarget collaboration with Genentech, a unit of Roche Group AG, of Basel, Switzerland. Lodo is using its genome mining and biosynthetic cluster assembly platform to identify molecules with therapeutic potential against multiple disease-related targets of interest to Genentech.
Metrion Biosciences Ltd., of Cambridge, U.K., said it contributed to two peer-reviewed papers as a member of the FDA’s Comprehensive in vitro Proarrhythmia Assay (CiPA) initiative, designed to extend the use of advances in electrophysiology-based cardiac ion channel screening, in silico predictive modelling and human induced pluripotent stem cell-derived cardiomyocytes to improve predictability of the cardiac liability of drug candidates. Metrion’s research formed part of the first stage of the proposed harmonization work. A paper published on March 27, 2020, in Nature Scientific Reports that reviewed data from a multiyear, multisite collaboration across industry, academia and the FDA to optimize experimental protocols and reduce experimental variability concluded that high-quality in vitro cardiac ion channel data are required for accurate and reliable characterization of the risk of delayed repolarization and pro-arrhythmia in the human heart and to guide subsequent studies and regulatory submissions. A paper published online in Toxicology and Applied Pharmacology used automated patch clamp data from the CiPA initiative to address the lack of statistical quantification of variability, enabling the consortium to establish a more systematic approach to estimate hERG block potency and safety margins to predict cardiac arrhythmia.
Nervgen Pharma Corp., of Vancouver, British Columbia, restated its previous guidance on plans to initiate a phase I study in the fourth quarter of 2020 and a phase II trial in spinal cord injury in the second half of 2021 of its lead protein tyrosine phosphatase sigma inhibitor, subject to additional impact from the COVID-19 pandemic on suppliers, FDA review times and financing. To conserve cash, the company said it reduced or suspended external consulting contracts unless directly related to development programs or financing. Denis Bosc, vice president of chemistry, manufacturing and controls is leaving the company immediately, and his departure is not expected to affect development timelines. Remaining members of the executive team agreed to a temporary compensation reduction in exchange for a one-time grant of additional stock options, and some nonexecutive staff agreed to a temporary salary reduction in exchange for a one-time stock option grant while others received termination notices. Nervgen said it granted 280,000 incentive stock options to directors and officers and an additional 96,000 incentive stock options to employees that may be exercised at $1.13 per share for up to 10 years. Nervgen went public in 2019 on the TSX Venture Exchange, where it trades as NGEN. Shares closed even on April 7 at CA$1.15 (US82 cents).
Pancella Inc., of Toronto, said it is set to receive advisory services and up to $400,000 from the National Research Council of Canada Industrial Research Assistance Program to help advance R&D of a two-step safety switch dubbed Pankiller. Pancella’s existing safety switch, known as Failsafe, is designed to eliminate rogue or deregulated therapeutic cells. The Pankiller system enhances that platform and provides a mechanism to remove a graft entirely once it has completed its therapeutic purpose, offering an alternative to the removal of therapeutic cells through irradiation.
Portola Pharmaceuticals Inc., of South San Francisco, said the Centers for Medicare & Medicaid Services established a permanent J-code for Andexxa (coagulation factor Xa [recombinant], inactivated-zhzo) to facilitate reimbursement in the hospital outpatient setting. The code, J7169, will take effect on July 1, 2020, and is expected to replace the temporary C-code.
Reneuron Group plc, of London, said it initiated a research program to evaluate use of its exosomes as a delivery vehicle for viral vaccines. Previously presented, unpublished data showed the company’s exosomes can be loaded with biologically active cargo and delivered preferentially to specific sites in the body. The new research is designed to establish whether the potency of SARS-CoV-2 vaccines in development can be enhanced using Reneuron's expertise in exosome isolation, modification and manufacture at scale. Although the company's prioritized internal research projects continue to progress, Reneuron said the COVID-19 pandemic is affecting most studies, including in the U.S., where the company is running trials with stem cell therapy candidates ReN-001 to treat stroke disability and ReN-003 to treat retinitis pigmentosa. Follow-up continues on participants already treated in both studies, but the company expects delays in additional recruitment and plans to provide updated guidance on timing of top-line data in due course. The company also said its licensing partner in China, Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd., continues to pursue development of ReN-001, a CTX neural stem cell therapy candidate, in its licensed territory, where applications were filed to open sites for Reneuron’s ongoing phase IIb Pisces III trial in stroke disability. Separately, Reneuron said it signed a research agreement with an unnamed major pharmaceutical company to explore the use of its exosomes to deliver gene silencing sequences created by the pharma. In the initial phase of the collaboration, Reneuron is responsible for manufacturing exosomes and loading them with the gene silencing sequences. Financial terms were not disclosed.
Sanofi SA, of Paris, said it finalized the planned restructuring of Praluent (alirocumab) with Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. Effective April 1, Sanofi assumed sole responsibility for Praluent outside the U.S., with Regeneron bearing sole responsibility in the U.S. The companies agreed to support each other’s manufacturing needs in the near term. Sanofi said it continues to work on the related antibody restructuring for Kevzara (sarilumab), given that U.S. and global programs are evaluating the IL-6 receptor antagonist to treat individuals hospitalized with severe and critical COVID-19 infection.
Timber Pharmaceuticals LLC, of Woodcliff Lake, N.J., said it was awarded the second tranche of $500,000 of a $1.5 million grant from the FDA’s Office of Orphan Products Development orphan products clinical trials grants program after reaching certain milestones in the development of TMB-001 (topical isotretinoin). Timber is advancing the candidate to treat congenital ichthyosis, a rare genetic keratinization disorder that leads to dry, thickened and scaling skin, and is enrolling participants with moderate to severe disease in the phase IIb Control study. Timber is slated to assume the NYSE American listing of Biopharmx Corp., of Campbell, Calif., through a reverse merger expected to close in the second quarter of 2020.