Aeterna Zentaris Inc., of Charleston, S.C., received a letter from Nasdaq that it's no longer in compliance with listing rules because the bid price for its shares was below $1 per share for 30 consecutive business days. The company has until July 1 to cure the deficiency and is considering its options to regain compliance.
Amag Pharmaceuticals Inc., of Waltham, Mass., completed a strategic review and plans to divest Intrarosa (prasterone) and Vyleesi (bremelanotide). The company has received preliminary expressions of interest for the drugs.
Bayer AG, of Leverkusen, Germany, and Exscientia Ltd., of Oxford, U.K., established a three-year, multitarget collaboration to discover drugs for cardiovascular and oncology indications. Exscientia will use its artificial intelligence capabilities to identify compounds, which will ultimately be owned and developed by Bayer. Exscientia is eligible to receive up to €240 million (US$267 million) in up-front, research and milestone payments as well as royalties on sales of the drugs.
Biomed X GmbH, of Heidelberg, Germany, and Merck KGaA, of Darmstadt, Germany, have started a sixth research project, which will focus on the role of the intestinal epithelial barrier in the development and exacerbation of autoimmune diseases such as systemic lupus erythematosus and multiple sclerosis.
Briacell Therapeutics Corp., of Vancouver, British Columbia, said it has identified a new group of breast cancer patients responding to its immunotherapy and combination therapies. The clinical benefit rate in the Bria-IMT monotherapy studies for grade I/II patients with immune responses was 71% (5/7) even though patients were pretreated with a median of seven prior regimens, including chemotherapy. In the combination study of Bria-IMT with Keytruda, all three patients with grade I/II tumors had clinical benefit. Those patients had been heavily pretreated with 14 to 15 prior regimens. The Bria-IMT cell line derives from a grade II tumor biopsy.
Preclinical findings from Cannabics Pharmaceuticals Inc., of Tel Aviv, Israel, and Bethesda, Md., show the cannabinoid Cannabigerol (CBG) had a greater antitumor effect on human stomach and bone cancer cell lines compared to CBGA, the acidic form of the compound. CBG is a nonpsychoactive cannabinoid found in minute quantities in the raw cannabis plant and has anecdotally shown promise in having anti-inflammatory qualities and as an antibacterial agent.
Cohbar Inc., of Menlo Park, Calif., said its peptide analogues of a mitochondrially encoded peptide (MBT5) demonstrated potent and selective inhibition of human CXCR4 receptor in cell-based assays, with IC50 values in the low nanomolar concentration range. In a difficult-to-treat in vivo mouse model of melanoma, the B16F10 syngeneic tumor model, the combination of MBT5 analogue administered subcutaneously with the chemotherapeutic temozolomide showed enhanced antitumor activity, reducing tumor growth after 11 days by 61% compared to control animals. The reduction in tumor growth produced by the combination exceeded the effect of either temozolomide used as a single agent, which reduced tumor growth by 38% compared to control, or the murine checkpoint inhibitor anti-PD-1 antibody, which had no effect on tumor growth in this model. CXCR4 is overexpressed in more than 75% of cancers and high levels of the receptor are associated with poor survival prognosis.
CBX-12 (alphalex-exatecan) is the new lead development program at Cybrexa Therapeutics Inc., of New Haven, Conn. CBX-12 has shown efficacy in preclinical studies. CBX-12 leverages the same class of payload used in the antibody-drug conjugate (ADC) Enhertu (fam-trastuzumab deruxtecan-nxki, Daiichi Sankyo Co. Ltd.), which was recently approved by the FDA under accelerated approval in unresectable or metastatic HER2-positive breast cancer. CBX-12 delivers a topoisomerase I payload, but targets tumors without the need for a specific antigen. CBX-12 has potential synergy with PD-1 inhibitors and other immuno-oncology drugs, the company said.
Cytovia Therapeutics Inc., of New York, and the New York Stem Cell Foundation Research Institute plan to develop new disease treatments leveraging human stem cell research and gene-editing techniques. Cytovia leverages natural killer (NK) cells to make therapeutics more specific to cancer cells. Chimeric antigen receptor NK cells are genetically engineered to find and attack tumors.
Effector Therapeutics Inc., of San Diego, and Pfizer Inc., of New York, entered an exclusive worldwide license and collaboration agreement to develop small-molecule inhibitors of eukaryotic initiation factor 4E (eIF4E), an oncogenic driver located downstream from the RAS and PI3K signaling pathways. Effector receives $15 million up front and is eligible for additional potential $492 million in R&D funding, development and sales milestone payments. Effector will receive royalties on sales and has an option to enter into a co-promotion and profit and loss share arrangement in the U.S. EIF4E is an effector protein activated in a variety of human cancers and linked to poor prognosis and resistance to certain therapies.
Evotec SE, of Hamburg, Germany, and Leverkusen, Germany-based Bayer AG expanded a previous deal in women’s health with a five-year, multitarget collaboration to develop multiple candidates to treat polycystic ovary syndrome. Both companies will contribute drug targets and have access to targets from another recently formed partnership between New York-based Celmatix Inc. and Evotec. Bayer is responsible for clinical development and commercialization and Evotec receives €6.5 million (US$7.2 million) up front and €10 million in research payments over five years, which it will share with Celmatix in exchange for target identification and prioritization. Evotec and Celmatix are eligible to receive preclinical, clinical and sales milestones of potentially more than €330 million as well as royalties up to low double-digit percentages of net sales. Evotec is participating in a funding round allowing Celmatix to expand its broader, innovative, therapeutics initiatives. The companies are collaborating to develop preclinical programs for highly prevalent, yet underserved conditions affecting women’s reproductive health, including endometriosis, polycystic ovary syndrome and infertility.
Class B subordinate shares of Toronto’s FSD Pharma Inc. began trading on Nasdaq Jan. 9 under the symbol HUGE. The shares continued trading on the Canadian Securities Exchange under the same symbol. FSD is developing PP-101 (micro-palmitoylethanolamide plus pregabalin), its preclinical drug candidate for the treatment of symptoms related to fibromyalgia.
The not-for-profit gene therapy research organization Généthon and Sarepta Therapeutics Inc., of Cambridge, Mass., expanded their collaboration to develop a gene therapy approach for Duchenne muscular dystrophy. The agreement paves the way for a clinical trial this year. Généthon is commercializing the product, GNT-0004, in Europe, excluding the U.K., and Sarepta is responsible for the rest of the world. Généthon receives an undisclosed up-front payment, development and commercial milestones and royalties from Sarepta. Yposkesi, a contract development and manufacturing firm for gene therapy viral vector manufacturing, will handle production of clinical and large-scale commercial batches based on suspension-based production methods.
Gilead Sciences Inc., of Foster City, Calif., said it licensed a portfolio of broadly neutralizing antibodies (bNAbs) against HIV, including two clinical-stage agents, 3BNC-117 and 10-1074, from Rockefeller University. The agents have potential for use in HIV long-acting therapies for treatment and prevention, as well as cure strategies. Initial preclinical and clinical research has shown that HIV bNAbs can produce an enhanced, prolonged immune response to HIV, representing a promising new approach for HIV treatment or prevention in combination with other long-acting agents, or prolonged virologic remission in the absence of antiretroviral use. Rockefeller will receive an up-front payment and is eligible to receive cumulative milestone payments, as well as royalties on net sales. Additionally, Rockefeller will retain rights to perform nonclinical and early stage clinical research on the portfolio of HIV antibodies.
Hikma Pharmaceuticals plc, of London, and Cambridge, U.K.-based Arecor Ltd., said they entered an exclusive agreement to co-develop a new, ready-to-use injectable medicine in the U.S. through Hikma's affiliate, Hikma Pharmaceuticals U.S. Inc. The product is being developed using Arecor's drug formulation technology platform, Arestat, which enhances the properties of approved therapeutic proteins and peptides to deliver new reformulations of existing, complex products. Hikma will seek approval for the product under the FDA’s 505(b)(2) regulatory pathway, with filing expected in 2021. Arecor will be eligible to receive an up-front payment and further payments on the achievement of development, regulatory and commercial milestones. Hikma will be responsible for the manufacture and commercialization of the product. Arecor will retain the right to develop and commercialize the product in certain markets outside the U.S.
The Institute for Clinical and Economic Review (ICER) said it released a final evidence report that assessed the comparative clinical effectiveness and value of JAK inhibitors for treating rheumatoid arthritis (RA), including Rinvoq (upadacitinib, Abbvie Inc.), Xeljanz (tofacitinib, Pfizer Inc. ) and Olumiant (baricitinib, Eli Lilly and Co.), as compared to either placebo or Humira (adalimumab, Abbvie Inc.). ICER's report on those therapies was reviewed at the December meeting of the California Technology Assessment Forum (CTAF), one of ICER's three independent evidence appraisal committees, where members unanimously voted the evidence was adequate to demonstrate that, when added to a conventional disease-modifying antirheumatic drug (cDMARD), upadacitinib, tofacitinib and baricitinib all provide a superior net health benefit to cDMARDs alone. Separately, CTAF members voted unanimously that the evidence is adequate to demonstrate that the biosimilar infliximab-dyyb (Inflectra, Pfizer Inc.) is clinically equivalent to its reference biologic, Remicade (infliximab, Janssen Biotech Inc.). ICER's economic modeling demonstrated that upadacitinib plus a cDMARD achieves marginally higher quality of life than what is achieved with adalimumab plus a cDMARD, at similar costs. Based on that comparison with adalimumab, ICER's value-based price benchmark range for upadacitinib is between $44,000 and $45,000.
Kaleido Biosciences Inc., of Lexington, Mass., said it has begun a research collaboration with Janssen’s World Without Disease Accelerator, part of the Janssen Pharmaceutical Cos. of New Brunswick, N.J.-based Johnson & Johnson. It will explore the potential for the company’s Microbiome Metabolic Therapies (MMT) to prevent the onset of childhood allergy and other atopic, immune and metabolic conditions by driving specific microbiome features that support an appropriate maturation of the infant immune system. The ex vivo screening platform will be used to identify MMT candidates that support the growth of specific beneficial microbes. They will then be further evaluated for their ability to prevent atopic conditions such as infant allergy.
Nanoviricides Inc., of Shelton, Conn., said it has completed genetic toxicology testing required to support the IND application for NV-HHV-101, moving toward clinical trials for topical dermal treatment of the shingles rash as the initial indication. The compound was found to be safe in terms of potential genotoxicity in the suite of tests that were performed and did not induce mutations in bacteria and did not cause chromosomal damage in human cells.
Principia Biopharma Inc., of South San Francisco, reported an expansion of its Bruton tyrosine kinase (BTK) franchise with PRN-473 topical, a reversible covalent BTK inhibitor. The company also announced suspension of PRN-1371, an FGFR inhibitor for bladder cancer to focus its product portfolio on immune-mediated diseases. PRN-473 topical is entering a phase I trial to evaluate its safety, tolerability and pharmacokinetics. It is the company’s third BTK inhibition molecule to enter the clinic, joining rilzabrutinib, an oral, reversible covalent BTK inhibitor designed to treat systemic diseases (pemphigus, immune thrombocytopenia and IgG4-RD) and SAR-442168/PRN-2246, the company’s partnered central nervous system-penetrating BTK inhibitor that targets immune and inflammatory diseases of the CNS.
Redx Pharma plc, of Alderley Park, U.K., said it has elected RXC-007 as its next drug development candidate for the treatment of fibrosis. It is a selective Rho-associated coiled-coil containing protein kinase 2 (ROCK2) inhibitor, an enzyme which sits at a nodal point in cell signaling pathways believed to be central to fibrosis. Preclinical data with compounds from the company’s ROCK2 program have demonstrated robust antifibrotic effects in a range of industry-standard in vivo models. Specifically, RXC-007, which is orally bioavailable, met fibrosis endpoints in disease models of liver and lung fibrosis. The company aims to initiate a phase I study with RXC-007 in the first half of 2021, while evaluating clinical development pathways in idiopathic pulmonary fibrosis and potentially in other disease areas.
Scholar Rock Holding Corp., of Cambridge, Mass., said it earned a $25 million milestone payment from Foster City, Calif.-based Gilead Sciences Inc. for the successful demonstration of efficacy in preclinical in vivo proof-of-concept studies. This is the most advanced program of the collaboration focused on the discovery and development of potent and selective inhibitors of latent transforming growth factor-beta (TGFβ) activation for the treatment of fibrotic diseases. The collaboration, signed in December 2018, allows Scholar Rock to retain exclusive worldwide rights to discover, develop and commercialize certain TGFβ inhibitors for oncology and cancer immunotherapy.
Solid Biosciences Inc., of Cambridge, Mass., said changes to its organizational structure that reduce its workforce by approximately one-third are designed to help advance SGT-001, a gene transfer candidate for the treatment of Duchenne muscular dystrophy (DMD). Last month, the company reported biomarker data from two patients that provide evidence that SGT-001 is biologically active with differentiated properties, which Solid believes warrants further evaluation. In November, the SGT-001 IGNITE DMD trial was placed on clinical hold by the FDA. Going forward, the company said it will focus on conducting its analyses of SGT-001 to determine how to address the clinical hold and resume dosing. The activities supporting the company’s other research and development programs will be curtailed.
Tacitus Therapeutics Inc., of New York, said, in collaboration with the Mount Sinai Health System, it launched a program to develop stem cell therapies initially targeting blood cancers and related clotting disorders. The first therapy, HSC-100, is being investigated in a phase I trial for treatment of hematological malignancies.
Wuxi Biologics, of Shanghai, and Barcelona, Spain-based Almirall SA, said they entered a strategic collaboration agreement that will allow Almirall to leverage the Wuxibody platform to develop bispecific antibodies for dermatological diseases. In return, Wuxi Biologics will receive an up-front payment as well as development, regulatory and commercial milestone payments for each bispecific antibody generated from that platform and will also be entitled to royalties based on global sales generated by those projects.