9 Meters Biopharma Inc., of Raleigh, N.C., reported preliminary results from a collaboration with Gustave Roussy, of Villejuif, France, on a 14-month research project initiated in March last year using NM-102, a long-acting, degradation-resistant peptide, believed to be gut-restricted, and presumed to prevent gut microbial metabolites and antigens from trafficking into systemic circulation. The researchers found that NM-102 was effective alone or when combined with immune checkpoint inhibitors (ICIs) in a preclinical transgenic mouse model of spontaneous aggressive skin melanoma. In addition, the combination of NM-102 with ICIs improved survival compared to ICIs alone. The company said it intends to move forward with an IND-enabling pathway to further develop NM-102.
Abpro Corp., of Woburn, Mass., said an article has been published in Nature Communications, titled, “Characterization of neutralizing antibody with prophylactic and therapeutic efficacy against SARS-CoV-2 in rhesus monkeys.” ABP-300, referred to as MW-05 in the publication, neutralizes COVID-19 by binding to the receptor binding domain (RBD) of the SARS-CoV-2 spike protein, blocking the viral interaction with the angiotensin-converting enzyme 2 receptors of the host, which are critical for viral entry and infection. Through that mechanism of action, ABP-300 not only completely and safely neutralizes COVID-19 in animal models but could potentially do so with superior safety and efficacy than other monoclonal antibodies in development, the company said. The study also reported ABP-300 was shown to have a protective effect by alleviating the lung lesions caused by SARS-CoV-2 in the monkeys whether the antibody was injected before or after the virus challenge.
Aeterna Zentaris Inc., of Charlestown, S.C., said that, through a wholly owned subsidiary, it has entered an amendment of its existing license agreement with Novo Nordisk Biopharm Ltd., a unit of Novo Nordisk A/S, of Bagsvaerd, Denmark, related to the development and commercialization of macimorelin. The original agreement provided Novo Nordisk the exclusive right to commercialize macimorelin in the U.S. and Canada, and it is currently marketing the product in the U.S. under the trade name Macrilen for the diagnosis of adult growth hormone deficiency (AGHD). Aeterna continues to retain all rights to macimorelin outside of the U.S. and Canada, but Novo Nordisk has agreed to make an immediate up-front payment of €5 million (US$5.91 million), which accelerates and replaces the $5 million later-stage regulatory approval milestone that it would have been required to pay only upon successful achievement of regulatory approval of macimorelin in the U.S. for the diagnosis of childhood-onset growth hormone deficiency (CGHD). All other future potential commercialization milestone payments remain unchanged. The companies are developing the expanded use of macimorelin for the diagnosis of CGHD. Aeterna has begun the startup phase for the clinical safety and efficacy study evaluating macimorelin for the diagnosis of CGHD, which is expected to be initiated in the first quarter of next year.
AIkido Pharma Inc., of New York, said positive results from a study of antiviral activity of FDA-approved drugs have been published in the Proceedings of the National Academy of Sciences. The publication reports studies on newly discovered antiviral compounds that were developed using a computer modeling approach, and the lead compounds were found to have broad-spectrum antiviral activity, inhibiting influenza virus, Ebola, Marburg, MERS-CoV, SARS-CoV and SARS-CoV2. The company previously executed a master license agreement with University of Maryland, Baltimore (UMB) for specific antiviral compounds discovered by UMB that seek to inhibit replication of multiple viruses, including Influenza virus, SARS-CoV, SARS-CoV2, MERS-CoV, Ebolavirus and Marburg virus.
Assembly Biosciences Inc., of South San Francisco, and Door Pharmaceuticals LLC, of Bloomington, Ind., signed an exclusive, two-year collaboration and option agreement focused on the development of a novel class of hepatitis B virus core protein modulators. Door’s platform targets functions of core protein distinct from viral assembly and that have the potential to interfere with viral nucleic acid including cccDNA transcription, providing a complement to Assembly’s current portfolio, the companies said. Financial details were not disclosed.
Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn., said Richard Granstein at Weill Cornell Medicine in New York, will initiate an investigator-led clinical trial with a Biohaven CGRP-receptor antagonist for the treatment of plaque psoriasis. The investigator-led clinical trial will explore whether treatment will reduce the severity of disease and percentage of area affected as measured by patients' Psoriasis Activity Severity Index score after 16 weeks of treatment as compared to placebo. In addition, the study will assess the potential impact on itch and patient quality-of-life measures, Biohaven said. Separately, the company said it had entered a value-based contract agreement for Nurtec ODT (rimegepant) with Prime Therapeutics LLC, of Eagan, Minn. The drug is an orally disintegrating tablet version of the CGRP antagonist. Terms of the outcomes-based agreement were not disclosed. In January, a meeting of the Midwest Comparative Effectiveness Public Advisory Council, a committee of the Institute for Clinical and Economic Review, found evidence to be insufficient to demonstrate superior net health benefit for rimegepant or any of the two other acute migraine treatments it evaluated when compared to triptans.
Bone Therapeutics SA, of Gosselies, Belgium, said Catalent Pharma Solutions Inc., of Somerset, N.J., has completed the acquisition of Bone’s manufacturing subsidiary, Skeletal Cell Therapy Support SA, disclosed in October.
Covis Group S.à r.l., of Luxembourg, completed its transaction with Amag Pharmaceuticals Inc., of Waltham, Mass., via the tender offer for all of the outstanding shares of common stock of Amag at $13.75 per share in cash and subsequent merger. The combined organization will operate as part of the Covis Pharma Group and will be led by Covis CEO Michael Porter.
Dicerna Pharmaceuticals Inc., of Lexington, Mass., said the FDA accepted the IND filed by partner Eli Lilly and Co., of Indianapolis, for a phase I trial testing RNAi candidate LY-3561774, triggering a milestone payment of $10 million to Dicerna.
Elevatebio Inc., of Cambridge, Mass., and Agbiome Inc., of Research Triangle Park, N.C., said that Lifeedit Therapeutics Inc., of Research Triangle Park, N.C., has joined Elevatebio’s portfolio of therapeutic, technology and manufacturing companies. Lifeedit combines a genome editing platform, derived from Agbiome’s microbial library, with Elevatebio’s expertise in the discovery and development of new cell and gene therapies. Lifeedit will continue to develop internal cell and gene therapies while further strengthening its platform.
Innocan Pharma Corp., of Herzliya, Israel, launched two therapeutic studies on small and large animals examining the therapeutic efficacy of Innocan's cannabidiol loaded liposome platform technology in relevant diseases. These studies follow the results of previous studies which demonstrated the prolonged release of cannabidiol for at least three weeks into the blood of mice and rats after a single injection. The new studies will be conducted by the Hebrew University of Jerusalem.
Johnson & Johnson, of New Brunswick, N.J., disclosed the expansion to the partnership between its Janssen Pharmaceutical Cos. and the U.S. Biomedical Advanced Research and Development Authority for the ongoing development of Janssen's COVID-19 vaccine candidate. Janssen will commit about $604 million and the authority will commit about $454 million to support the ongoing phase III Ensemble trial evaluating the candidate as a single dose in up to 60,000 volunteers worldwide.
Kala Pharmaceuticals Inc., of Watertown, Mas., provided an update on its plans to launch Eysuvis (loteprednol etabonate ophthalmic suspension) 0.25% for the short-term (up to two weeks) treatment of the signs and symptoms of dry eye disease. The firm nearly completed the hiring and onboarding of the first wave of its planned sales force expansion ahead of the virtual launch meeting in early December. Kala also established a wholesale acquisition cost, which will allow the company to begin submitting payer contract bids to pharmacy benefit managers, commercial and Medicare part D plans. The drug was approved in October.
Lead Pharma Holding BV, of Oss, the Netherlands, signed a collaboration and license agreement with Roche Holding AG, of Basel, Switzerland, to develop oral small molecules to treat a broad range of immune-mediated diseases. The pair will collaborate in research activities up to the selection of a preclinical candidate, after which Roche will be responsible for further development and global commercialization. Lead will receive an up-front payment of €10 million (US$11.8 million), and will be eligible to receive research funding and preclinical milestone payments; total potential payments including research, development, regulatory and sales milestones may add up to as much as €260 million, plus royalties on worldwide sales.
Merck & Co. Inc., of Kenilworth, N.J., said the Bill & Melinda Gates Foundation agreed to support a pivotal phase III study assessing islatravir as a once-monthly oral pre-exposure prophylaxis option in women and adolescent girls at high risk for acquiring HIV-1 infection in sub-Saharan Africa. The IMPOWER 22 study of the nucleoside reverse transcriptase translocation inhibitor is expected to begin by early 2021. The foundation intends to provide grant funding to the International Clinical Research Center at the University of Washington’s department of global health, which is collaborating with Merck on IMPOWER 22. The grant will support ICRC’s work with experienced trial sites in sub-Saharan Africa to enroll, follow and retain trial enrollees. As sponsor, Merck will supply the medicine, pursue regulatory and customs approvals and manage the trial, including site monitoring and data reporting. Merck will fund IMPOWER 22 in the U.S.
Minoryx Therapeutics SL, of Barcelona, disclosed the publication in Neurobiology of Disease of preclinical data on its lead compound, leriglitazone, showing potential therapeutic action and protection against neurodegeneration, in particular against Friedreich’s ataxia. The data support the ongoing development of the compound, which improves impairments that are derived from frataxin loss, the genetic deficiency that causes the disease.
Pfizer Inc., of New York, completed the transaction to spin off its Upjohn business and combine it with Mylan NV, of Canonsburg, Pa., to form Viatris Inc. Under the terms of the transaction, which was structured as an all-stock reverse Morris trust, Upjohn was spun off to Pfizer stockholders by way of a pro rata distribution and immediately thereafter combined with Mylan. The combined company was renamed Viatris and will begin trading (NASDAQ:VRTS) on Nov. 17.
Pharvaris GmbH, of Zug, Switzerland, said it's preparing to initiate an on-demand study of PHVS-416, a soft capsule formulation of its bradykinin B2 receptor antagonist PHA-121, for the treatment of hereditary angioedema (HAE) attacks in 2021. At the virtual American College of Asthma, Allergy and Immunology Annual Scientific Meeting, the company presented new pharmacokinetic and pharmacodynamic data suggesting that "rapid onset of action and prolonged efficacy with a single dose of PHA-121 can be expected in the treatment of acute HAE attacks.”
PTC Therapeutics Inc., of South Plainfield, N.J., said a review of 66 patient case reports found that symptoms of aromatic L-amino acid decarboxylase deficiency (AADC-d) that contribute to high disease burden started from birth in 29% of cases and accumulated over time. By age 6, 28% of patients were so severely disabled that they experienced symptoms in six core domains. Now, an MAA for PTC-AADC (eladocagene exuparvovec), the company's experimental therapy for the condition, is under review, with an EMA CHMP opinion expected in the first half of 2021, it said.
Receptor Life Sciences Inc., of Seattle, was granted a Schedule I research license from the U.S. DEA. The company is developing oral and inhaled cannabinoid medicines.
Retrophin Inc., a San Diego-based company founded by the now-jailed former hedge fund manager Martin Shkreli, has changed its corporate name to Travere Therapeutics Inc., pronounced "truh-veer," it said. The company, now led by CEO Eric Dube, is advancing candidates for rare disorders in nephrology, hepatology and metabolism. Its pipeline is led by sparsentan, which is being investigated in pivotal phase III trials for rare kidney diseases.
Spectrum Antimicrobials Inc., of Petaluma, Calif., a subsidiary of Collidion, disclosed work on SPC-069, a candidate designed to have a threefold mechanism of action against pathogens that may affect the respiratory system. First, it achieves viral and bacterial destruction through a non-selective reaction pathway, causing fundamental damage to viral structures that are responsible for causing infection. SPC-069’s mechanism of action against bacteria causes damage to the bacterial cell membrane followed by interruption of ATP production, where such damages are irreversible. The secondary mode of action for SPC-069 involves reduction of inflammation through the possible stabilization of mast cells, reducing the overall release of histamines. Lastly, it has a non-selective reaction with general cellular receptors that may be responsible for the reduction of ACE II receptor activity. That may potentially inhibit the spike proteins of the virus from attaching to the receptor which leads to the infection of healthy cells, the company said. ACE II has been identified as the primary entry point for coronaviruses which cause illnesses such as cold or flu.
Synthetic Biologics Inc., of Rockville, Md., engaged Alliance Global Partners to assist it in exploring and evaluating a range of strategic options. On Oct. 2, the company said a phase IIb trial of one of its two lead candidates, SYN-010, in people with constipation-predominant irritable bowel syndrome would be discontinued after an interim futility analysis suggested it was unlikely to meet the study's primary endpoint. It's continuing to develop SYN-004 (ribaxamase), an oral enzyme designed to prevent dysbiosis of the gut microbiome, and SYN-020, an intestinal alkaline phosphatase.
Vertex Pharmaceuticals Inc., of Boston, advised its shareholders to reject a "mini-tender" offer from TRC Capital Investment Corp., of Ontario. The unsolicited offer price of $210 per share (NASDAQ:VRTX) fell below the company's current shore value, of $224 per share.
Vivus Inc., of Campbell, Calif., filed an amended Chapter 11 bankruptcy plan. Subject to confirmation of the plan by the U.S. Bankruptcy Court for the District of Delaware, it will emerge from bankruptcy as a wholly owned subsidiary of IEH Biopharma LLC. The company will continue to manufacture, sell and provide physician and patient support for its commercial products, Qsymia (phentermine and topiramate) capsules for weight management in adults and Pancreaze (pancrelipase) for the treatment of exocrine pancreatic insufficiency due to cystic fibrosis or other conditions.
Xoma Corp., of Emeryville, Calif., earned a $2 million milestone payment from Takeda Pharmaceutical Co. Ltd., of Osaka, Japan, in connection with the first patient being dosed in its phase II study to evaluate safety, tolerability, and efficacy of mezagitamab (TAK-079) in participants with generalized myasthenia gravis. Mezagitamab is an anti-CD38 antibody that resulted from the companies’ collaboration.
Xortx Therapeutics Inc., of Calgary, Alberta, reported results of an analysis of approximately 800 patients hospitalized with COVID-19 who had measurements of uric acid at the time of or during hospitalization. Acute kidney injury (AKI) was documented early in the infection, with 36% of individuals having evidence of AKI at admission. An additional 23% developed AKI during hospitalization. More than 50% of individuals admitted to hospital with AKI had very high serum uric acid levels with a mean of greater than 8 mg/dL. Nearly 90% of patients with severe hyperuricemia had a MAKE-D event (a 100% increase or more in serum creatinine from baseline or dialysis or death). Xortx is developing XRx-101, which is designed to lower uric acid.