Aavantibio Inc., of Gainesville, Fla., which is developing a gene-targeting therapy for Friedreich's ataxia, said it has been awarded Muscular Dystrophy Association Venture Philanthropy funding totaling $1.07 million to advance its phase II trial of a gene replacement therapy for the disease. The company will now be able to begin production of the clinical gene vector for its program prior to starting trials this year.
Actinium Pharmaceuticals Inc., of New York, said it entered an agreement with the University of California, Davis, which will utilize the company's antibody radiation-conjugate apamistamab-I-131 for targeted conditioning and replace the chemotherapy conditioning being used in an ongoing phase I/II stem cell gene therapy clinical trial. In the study, patients with relapsed or refractory HIV-related lymphoma are being treated with autologous stem cell gene therapy. Until now, conditioning in the trial, as is typical, used a multidrug chemotherapy regimen administered over several days. That approach is nontargeted, associated with toxicities that impairs patients and restricts the use and efficacy of cellular therapy. Apamistamab-I-131, which requires just one therapeutic administration, will displace the nontargeted chemotherapy to condition patients in a targeted manner with the goal of reducing conditioning-related toxicities and improving patient outcomes.
Adaptive Biotechnologies Corp., of Seattle, said it entered a global diagnostic agreement with Genentech, a member of the Roche Group, of Basel, Switzerland, to utilize its next-generation sequencing-based Clonoseq assay to assess minimal residual disease (MRD) status in response to venetoclax, a selective B-cell lymphoma-2 inhibitor, in the registrational phase III Cristallo study for the treatment of newly diagnosed people with chronic lymphocytic leukemia (CLL), plus additional future venetoclax studies in CLL. The companies will partner to incorporate the assay to measure MRD status as a primary endpoint in the study. Adaptive will receive up-front and sample testing payments to advance the development and potential expedited approval of venetoclax in that setting.
Adare Pharmaceuticals Inc., of Lawrenceville, N.J., and Stans, Switzerland-based NLS Pharmaceutics Ltd. said they will collaborate to develop mazindol (MZD) controlled-release product candidates for the treatment of narcolepsy and attention deficit hyperactivity disorder. The enhanced collaborative formulations are intended to deliver an effective pharmacokinetic profile and are expected to improve patient compliance and disease management. MZD is a norepinephrine reuptake inhibitor previously approved by the FDA for the treatment of obesity.
Akelos Inc., of New York, which is currently developing and commercializing a non-opioid, antihyperalgesic drug to treat chronic and neuropathic pain, said its scientific founder, Peter Goldstein, has been awarded a $1.75 million two-year grant from the NIH to develop a non-opioid alternative for treating neuropathic pain. Research will be focused on the HCN1 ion channel for primary sensory neurons, which plays a pivotal role in the individual’s experience of pain.
Allele Biotechnology and Pharmaceuticals Inc., of San Diego, and Astellas Pharma Inc., of Tokyo, through its Massachusetts-based subsidiary, Astellas Institute for Regenerative Medicine, entered a licensing agreement to expand Astellas’ access to Allele’s induced pluripotent stem cell technologies for various cell therapy programs. Under the terms, Astellas will pay Allele an up-front fee and milestones, product-based royalties and potentially manufacturing fees.
Allogene Therapeutics Inc., of South San Francisco, and Springworks Therapeutics Inc., of Stamford, Conn., said they entered a clinical trial collaboration agreement that will cover the evaluation ALLO-715, an investigational anti-B-cell maturation antigen (BCMA) AlloCAR T therapy, in combination with Springworks’ investigational gamma secretase inhibitor, nirogacestat, in patients with relapsed or refractory multiple myeloma. In preclinical models, nirogacestat has been shown to increase the cell surface density of BCMA and reduce levels of soluble BCMA, thereby enhancing the activity of BCMA-targeted therapies. Allogene will sponsor and conduct the phase I study to evaluate the safety, tolerability and preliminary efficacy of the combination, and will assume all development costs associated with the study.
Alphaeon Corp., of Newport Beach, Calif., said it completed a reorganization to simplify its corporate structure by splitting into two business units, each of which will retain the same shareholder base that was in place prior to the change. Aeon Biopharma Inc. will now focus on developing its botulinum toxin complex, ABP-450 (prabotulinumtoxinA) injection, as a therapeutic application for debilitating medical conditions. Its second unit, Alphaeon1 LLC, will be the vehicle for Alphaeon stockholders to continue to hold the approximately 8.7 million shares of Evolus Inc., previously held by the company, subject to securities law restrictions on resales by affiliates for six months under Rule 144.
Biomed X GmbH, of Heidelberg, Germany, said it started a joint research group in collaboration with Boehringer Ingelheim GmbH, of Ingelheim, Germany, at the Biomed X Innovation Center. The aim of the new research project is to investigate myelination deficits of the adolescent brain in connection with the development of schizophrenia, with the goal of identifying extrinsic (neuronal) and intrinsic (oligodendrocytic) factors affecting myelination as potential new targets for the prevention of schizophrenia in high-risk individuals.
Crestone Inc., of Boulder, Colo., got a hand from the Cystic Fibrosis Foundation for preclinical development of Crestone’s antibiotic lead series to treat nontuberculous mycobacteria infections, focusing on M. abscessus. The series’ efficacy will be tested at Colorado State University and Johns Hopkins University. The compounds selectively target MmpL3, a membrane transporter of trehalose monomycolate essential for cell wall synthesis. Financial details were not released.
Curis Inc., of Lexington, Mass., and New York’s Darwinhealth joined for a multiyear scientific research collaboration to better understand the role of the transcription factor MYC in the mechanism of Curis’ fimepinostat. Oral fimepinostat is a synthetic small molecule that inhibits the activity of histone deacetylase, and phosphatidyl-inositol 3 kinase, across a number of tumor subtypes. While no financial specifics were released, Darwinhealth will be eligible for milestone payments and royalties for applications.
Dinaqor AG, of Pfäffikon, Switzerland, and London’s University College London agreed to a research collaboration and exclusive license agreement to develop gene therapies for treating monogenic cardiomyopathies. The deal involves advancing Dinaqor’s two cardiac gene therapy programs into the clinic. Dinaqor will obtain exclusive worldwide rights to any therapies developed as part of the collaboration and UCL will be entitled to sales royalties on approval.
Eagle Pharmaceuticals Inc., of Woodcliff Lake, N.J., said it agreed on terms with the University of Pennsylvania for an exclusive worldwide license agreement to develop dantrolene sodium for the potential treatment of people living with Alzheimer’s disease, including an agreement to fund additional research and provisions regarding commercialization of products developed under the license. Research previously showed that intranasal administration of dantrolene sodium provided therapeutic effects on memory and cognition in a mouse model of Alzheimer’s.
Editas Medicine Inc., of Cambridge, Mass., and Sandhill Therapeutics Inc., of Dallas, agreed on a strategic research collaboration, license and option agreement to combine their genome editing and cell therapy technologies to discover, develop and manufacture allogeneic engineered natural killer cells and non-alpha beta T-cell medicines to treat cancer. Editas receives the exclusive license to Sandhill’s technology to research, develop and commercialize immuno-oncology engineered cell medicines for solid tumors and an option to expand the license to two additional areas. Sandhill receives an up-front payment, development and sales-based milestone payments and royalties on sales. No financial details were released.
ERS Genomics Ltd., of Dublin, licensed its CRISPR genome-editing technology intellectual technology to Tokyo’s Daiichi Sankyo Co. Ltd. to support its internal research and development. Eric Rhodes, ERS’ CEO, said the deal is the company’s first large pharma licensee in Japan. Financial details were not released.
Eusa Pharma Inc., of Hemel Hempstead, U.K., and Beigene Ltd., of Beijing, entered an exclusive development and commercialization agreement for Sylvant (siltuximab) and Qarziba (dinutuximab beta). Eusa granted Beigene exclusive rights to Sylvant in greater China and to Qarziba in mainland China. Beigene will fund and undertake all clinical development and regulatory submissions in the territories and will launch and commercialize both products once approved. Eusa will receive an up-front payment and be eligible to receive payments upon the achievement of regulatory and commercial milestones up to a total of $160 million. Eusa will also be eligible to receive tiered royalties on future product sales. Sylvant is approved in more than 40 countries to treat idiopathic multicentric Castleman’s disease. Qarziba is the only EMA-approved targeted immunotherapy to treat high-risk neuroblastoma. Both products have been listed for fast-track approval in China by the NMPA.
Exonate Ltd., of Cambridge, U.K., entered a strategic collaboration agreement with Janssen Pharmaceuticals Inc., one of the Janssen Pharmaceutical Cos. of Johnson & Johnson. Exonate will work with Janssen Research & Development LLC scientists to develop an eye drop treatment for retinal vascular diseases such as wet age-related macular degeneration and diabetic macular edema by using mRNA targeted therapies. Exonate has developed small molecules that inhibit the production of pro-angiogenic vascular endothelial growth factor (VEGF) through the selective inhibition of serine/threonine-protein kinase-mediated VEGF splicing. The agreement was facilitated by Johnson & Johnson Innovation. Financial terms were not disclosed.
Fauna Bio Inc., of Berkeley, Calif., which has developed a hibernation biobank, has entered a research collaboration with Bagsvaerd, Denmark-based Novo Nordisk A/S to find new therapies for obesity by investigating molecules and pathways that increase metabolic rate in hibernating mammals. The goal is to investigate cellular physiology at key time points during the metabolically intense arousal periods in hibernation. The results of this work will provide insight into the specific genes, proteins and signaling networks that underlie metabolic switch points, the companies said. Understanding how animals survive extreme conditions may help in treating obesity and metabolic diseases, as well as conditions such as strokes, heart attacks and Alzheimer's disease. Fauna Bio will receive an up-front payment and research support for activities conducted in the collaboration, while granting Novo Nordisk an exclusive option to license targets identified under the collaboration.
Horizon Discovery Group plc, of Cambridge, U.K., signed a collaboration and license agreement with Mammoth Biosciences Inc., of South San Francisco, to access Mammoth’s CRISPR platform and thereby facilitate delivery of a new generation of genetically engineered cells to produce biotherapeutics. The companies will collaborate to optimize certain CRISPR tools to rapidly develop proprietary CHO cell lines. Deal terms were not disclosed.
Illumina Inc., of San Diego, and Roche Holdings AG, of Basel, Switzerland, disclosed a 15-year, nonexclusive collaboration agreement to broaden the adoption of distributable next-generation sequencing-based testing in oncology. As part of the deal, Illumina will grant Roche rights to develop and distribute in vitro diagnostic tests on Illumina’s Nextseq 550Dx System, as well as on its future portfolio of diagnostic sequencing systems, including the forthcoming NovaseqDx. Roche will in turn collaborate with Illumina to complement Illumina’s pan-cancer assay, Trusight Oncology 500 (TSO 500), with new companion diagnostic claims. The financial terms of the deal were not disclosed.
Indapta Therapeutics Inc., of San Francisco, and Basel-based CDMO Lonza AG announced a strategic partnership in which Lonza will manufacture Indapta’s off-the-shelf, allogeneic G-NK cell therapy under cGMP for use in clinical studies. Process development and manufacturing will take place in Lonza's cell and gene therapy manufacturing facility in Houston.
Iovance Biotherapeutics Inc., of San Carlos, Calif., obtained a license from Novartis AG, of Basel, Switzerland, to develop and commercialize IOV-3001, an antibody cytokine engrafted protein. Iovance will make an undisclosed up-front payment to Novartis as well as low single-digit milestones involved in initiation of patient dosing in various phases of clinical development for IOV-3001 and approval of the product in the U.S., EU and Japan. Novartis is also entitled to low-to-mid single-digit royalties from commercial sales of the product. IOV-3001 is an engineered IL-2 CDR graft which targets IL-2R beta-gamma-expressing cells and limits IL-2R alpha-beta-gamma-dependent Treg activation. The protein has an improved half-life leading to a better exposure while minimizing Cmax possibly reducing the side effects associated with IL-2 protein. Iovance will focus on GMP manufacturing of IOV-3001 during 2020 and may initiate IND-enabling activities as early as 2021. Separately, Iovance and Cellectis SA, of Paris, signed a research collaboration and exclusive worldwide license agreement whereby Iovance will license certain Talen technology from Cellectis in order to develop tumor-infiltrating lymphocytes (TIL) that have been genetically edited to create more potent cancer therapeutics. The deal allows Iovance to use Talen technology for multiple gene targets to modify TILs for therapeutic use in several cancer indications. Iovance will pay undisclosed development, regulatory and sales milestone payments to Cellectis, as well as royalty payments based on net sales of Talen-modified TIL products.
The Janssen Pharmaceutical Cos. of Johnson & Johnson, of New Brunswick, N.J., announced a significant investment to expand the presence and capabilities of Janssen Research & Development LLC, in the San Francisco Bay area. The investment will support the leasing and construction of an R&D facility in the global innovation center of South San Francisco. Janssen personnel at the new facility will focus on infectious and retinal diseases, data science and emerging modalities within discovery, product development and supply. The expanded facility is projected to more than double Janssen’s presence in South San Francisco and is anticipated to be fully operational in 2022, upon completion of construction.
Mediwound Ltd., of Yavne, Israel, expanded its agreement with Swiss firm Genfa Medica SA, granting it the exclusive right to market and distribute Nexobrid in Ukraine, Lithuania, Latvia and Estonia, adding to its current rights to distribute Nexobrid in Russia. Nexobrid, currently approved in the EU and other markets, is a topically administered biologic that removes eschar in patients with deep partial and full-thickness thermal.
Moderna Inc., of Cambridge, Mass., said that by building on the clinical validation of systemic delivery of mRNA provided by data from its antibody against the chikungunya virus (mRNA-1944) program, it will launch into new therapeutic areas, including autoimmune and inflammatory diseases. The company also will expand its pipeline of vaccines in the near term, following six positive phase I trial readouts from its infectious disease portfolio and the initiation of a phase II study for its cytomegalovirus vaccine (mRNA-1647). Moderna is developing two potential medicines, mRNA-6231 and mRNA-6981, designed to engage peripheral tolerance pathways to dampen autoimmune activation and help restore immune homeostasis, thereby reducing autoimmune pathology. mRNA-6231 is an mRNA encoding for a long-acting IL-2 mutein designed to preferentially activate and expand the regulatory T-cell population, dampening the immune response. mRNA-6981 is an mRNA encoding for PD-L1 and is designed to augment cell surface levels of PD-L1 on myeloid cells, providing co-inhibitory signals to self-reactive lymphocytes.
Neurobo Pharmaceuticals Inc., of Boston, said its merger with Gemphire Therapeutics Inc., of Ann Arbor, Mich., closed. The new company, known as Neurobo Pharmaceuticals, now trades on Nasdaq under NRBO.
Obseva SA, of Geneva, and China’s Yuyuan Bioscience Technology Co. Ltd. said they entered a sublicense agreement to develop and commercialize nolasiban for improving clinical pregnancy and live birth rates in women undergoing embryo transfer following in vitro fertilization in the People's Republic of China. Nolasiban is an oral oxytocin receptor antagonist, for which two phase III studies have been completed in Europe. Under the terms, Yuyuan has the exclusive rights to develop and commercialize nolasiban in the PRC and will fund all development and registration activities. Both companies plan to collaborate on the subsequent global development of nolasiban, but Obseva will retain worldwide rights to the product outside of the PRC. In addition, both companies will seek to expand their collaboration in China on other projects. Financial terms of the deal are not being disclosed.
Pear Therapeutics Inc., of Boston, said it entered an agreement with Apricity Health LLC to develop up to two product candidates for precision management of patients undergoing cancer treatment. Apricity, which leverages technology and analytics to address the expertise gap in real-world medicine, is developing its first product candidate, Apricityrx, to enable intelligent monitoring and precision management of patients receiving immune checkpoint therapies.
Tauriga Sciences Inc., of New York, said it amended the certificate of incorporation to change its subsidiary’s name to Tauriga Pharma Corp., to reflect the decision to focus on development of a pharmaceutical product line to address nausea symptoms related to chemotherapy treatment in patients.
Titan Pharmaceuticals Inc., of South San Francisco, said it signed an agreement for its Probuphine (buprenorphine) implant to be included on the Federal Supply Schedule, providing U.S. veterans and other federal government agencies with access to Titan's six-month maintenance treatment for opioid use disorder in eligible patients. The final contract is effective Jan. 15 and has a five-year term.
Trellis Bioscience Inc., of Redwood City, Calif., is receiving an award of up to $3.2 million in nondilutive funding, with the possibility of $3.8 million more if certain project milestones are met, from CARB-X to develop a monoclonal antibody designed to disrupt the protective biofilm that makes bacteria resistant to antibiotics. Trellis is developing TRL-1068, which it said works by extracting a key scaffolding protein, thereby rendering the bacteria more susceptible to attack by the immune system as well as more sensitive to conventional antibiotics. TRL-1068 has been shown to enhance antibiotic activity in highly drug-resistant strains of gram-positive and gram-negative bacteria. The CARB-X award will support antibody manufacturing for phase I.