Acasti Pharma Inc., of Laval, Quebec, manufactured the first cGMP batches of Capre, Acasti’s omega-3 pharmaceutical product candidate, produced with a proprietary continuous manufacturing process developed in partnership with Cordenpharma Chenôve, of Dijon, France. Capre is a described as a potentially best-in-class omega-3 drug derived of krill oil and being developed for the treatment of patients with hypertriglyceridemia.
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. won FDA breakthrough status for givosiran (ALN-AS1), the company’s investigational RNAi therapy targeting aminolevulinic acid synthase 1 for the prophylaxis of attacks in patients with acute hepatic porphyria. The candidate received a PRIME designation from the EMA earlier this year. Alnylam plans to initiate a phase III program for givosiran in late 2017.
Atreca Inc., of Redwood City, Calif., and the Dana-Farber Cancer Institute entered a research agreement to focus Atreca’s Immune Repertoire Capture (IRC) technology on the active immune responses of cancer patients whose disease is responding well to immunotherapy and other treatments. The research will apply the IRC technology, which identifies and generates sequences of functional, native antibodies and T-cell receptors from active human immune responses, with the aim of understanding why only subsets of patients benefit from therapy and to identify antitumor antibodies generated in those responses. The study will focus on patients with melanoma, non-small-cell lung cancer and renal cell cancer, with the potential to expand into other cancer types.
Catalyst Biosciences Inc., of South San Francisco, said the EMA’s Committee for Orphan Medicinal Products issued a positive opinion recommending orphan medicinal product designation for CB 2679d/ISU304 for the treatment of hemophilia B. CB 2679d/ISU304 is a next-generation coagulation factor IX variant.
Compugen Ltd., of Holon, Israel, said it anticipates a delay of several months in the submission of an investigational new drug (IND) application for COM-701, the clinical candidate antibody targeting CGEN-15029/PVRIG. Previously, the company had disclosed an expectation of IND filing in the fourth quarter of this year, but was recently informed by its manufacturing service provider for COM-701, a global contract development and manufacturing organization, that the batch of material manufactured for Compugen’s planned good laboratory practice toxicity studies was contaminated during the manufacturing process. The contamination was discovered during quality control procedures prior to release of the affected batch and, as such, was not used in any preclinical studies. That contamination has necessitated the production of a new batch of material for the execution of those studies.
Cure Pharmaceutical Inc., of Oxnard, Calif., said its partnership with Cannakids has entered a strategic research collaboration with Technion Research and Development Foundation Ltd., a wholly owned subsidiary of the Technion-Israel Institute of Technology, a science and technology university, in order to research how different cannabinoid compounds within cannabis strains can be used to treat various subtypes of cancer. Terms were not disclosed.
Eisai Co. Ltd., of Tokyo, said its U.S. subsidiary, Eisai Inc., entered a new joint research agreement with the Broad Institute to develop a new antimalarial medicine based on antimalarial drug targets the team identified last year. The Eisai and Broad joint development program for antimalarial medicines was established in September 2014 and has led to the identification and optimization of promising molecules, using hits obtained by screening Broad’s compound library for antimalarials as a starting point. Those compounds interact with a novel target in the malarial parasite (phenylalanine t-RNA synthetase), which results in inhibition of protein synthesis. The compounds exhibit potent in vitro and in vivo antimalarial activity, in the blood-, liver- and transmission-stages of the parasite life cycle. Those results were published in Nature.
Epimab Biotherapeutics Inc. and Wuxi Biologics Co. Ltd., both of Shanghai, agreed to work together to advance Epimab’s first candidate, EMB-01, toward an investigational new drug application (IND) filing. EMB-01, a bispecific antibody, has shown efficacy in multiple preclinical cancer models, the company said. Under the agreement, Wuxi will be responsible for establishing the chemistry, manufacturing and control portion for the EMB-01 development program up to filing of the IND, which is expected in mid-2018.
Evoke Pharma Inc., of Solana Beach, Calif., has enlisted the regulatory consulting and contract research organization Rho Inc. to assist it with the preparation and submission of its planned 505(b)(2) new drug application (NDA) for Gimoti, the company’s nasal delivery formulation of metoclopramide for the relief of symptoms associated with acute and recurrent diabetic gastroparesis in adult women. The company said it remains on track to initiate and complete a pharmacokinetic study in the second half of this year, with an NDA submission by late 2017 or early 2018.
Forward Pharma A/S, of Copenhagen, has filed a notice of appeal of the Patent Trial and Appeal Board decision that ended the interference proceeding between the Forward 11/576,871 patent application and an issued patent of Cambridge, Mass.-based Biogen Inc. The appeal was filed to the U.S. Court of Appeals for the Federal Circuit and seeks to have the decision overturned and the interference reinstated. The appeal is expected to last 10 months or longer, according to Forward. The case involves Biogen’s Tecfidera (dimethyl fumarate) for multiple sclerosis, which competes with a Forward compound.
Heptares Therapeutics Ltd., of London, a wholly owned subsidiary of Sosei Group Corp., published a high-resolution X-ray crystal structure of the full-length glucagon-like peptide-1 (GLP-1) receptor bound to a peptide agonist in Nature. Findings provide insight into the molecular mechanism of action of GLP-1 peptides and their interactions with the receptor, and the research further validates and enables the application of structure-based methods to the design of optimized peptide therapeutics and small molecules targeting the GLP-1 receptor, and related G protein-coupled receptors, for treating a range of diseases.
I2 Pharmaceuticals Inc., of Boulder, Colo., said it has acquired all of the antibody and engineered protein technology of Redwood City, Calif.-based Sea Lane Biotechnologies LLC, including several potential product candidates. The acquisition consists of three distinct classes of protein therapeutics, including human antibody libraries, human B-cell derived antibodies, and a new class of engineered protein therapeutics called Surrobodies, which have the potential to be developed as cancer therapeutics. Financial terms were not disclosed.
Imidomics SL, of Barcelona, Spain, said it will collaborate with Summit, N.J.-based Celgene Corp. to advance the discovery and development of treatments to address patients with immune-mediated inflammatory diseases affecting large patient populations. Ulcerative colitis, Crohn’s disease, psoriatic arthritis, rheumatoid arthritis, psoriasis and lupus erythematosus are among the conditions covered. Under the terms of the collaboration agreement, Celgene will have access to Imidomics’ database integrating clinical and molecular data across the areas of interest, using it to identify new targets and biomarkers, support the development of new therapeutic candidates and next-generation companion diagnostics, and enable patient population stratification for trials. Imidomics will receive project funding and a potential royalty stream for products resulting from the collaboration.
Immune Therapeutics Inc., of Orlando, Fla., said it has signed a memorandum of understanding (MOU) for the distribution in Côte d’Ivoire (the Ivory Coast) for Lodonal, an immune system regulator for the management of HIV/AIDS. The MOU is among Immune Therapeutics, Washington-based GB Pharma Holdings and local firm Rougier Pharma Afrique SA and sets forth a framework for the future standard terms and conditions for distributing Lodonal in Côte d’Ivoire. The company anticipates distribution and sales of Lodonal in Côte d’Ivoire by the end of 2017. Rougier is leading the approval process with the support of Immune Therapeutics and GB Pharma. Once drug approval is obtained in Côte d’Ivoire, Rougier intends to expand distribution to Benin, Togo, Burkina Faso, Niger and Gabon after facilitating the approvals in those countries as well.
Inspyr Therapeutics Inc., of Westlake Village, Calif., said it is purchasing Lewis and Clark Pharmaceuticals, of Charlottesville, Va., in an all-stock transaction. The resulting integrated biopharmaceutical company will focus on advancing a broad pipeline of therapies to treat cancer, inflammation and other serious diseases. Inspyr’s lead product candidate, mipsagargin, a prodrug therapeutic for cancer, is being developed initially for hepatocellular carcinoma. Lewis and Clark’s pipeline includes potentially first-in-class adenosine receptor modulator-based compounds currently in advanced preclinical development as oncology or anti-inflammatory agents.
Mabvax Therapeutics Holdings Inc., of San Diego, said it entered a sponsored research agreement with Memorial Sloan Kettering Cancer Center (MSK) for the development of chimeric antigen receptor (CAR) T-cell therapeutics using antibody-targeting sequences derived from the company’s fully human antibodies for pancreatic, small-cell lung and other solid tumor cancers. The research will evaluate sequences from the monoclonal antibody clinical development candidate Humab-5B1 in xenograft models of pancreatic and small-cell lung cancer. Mabvax will provide funding to evaluate the efficacy of multiple CAR T-cell products incorporating several different antibody-targeting sequences. The company has provided a second set of antibody-targeting sequences to MSK for development into new CAR T-cell constructs for the treatment of solid tumors, and the center will evaluate the new CAR T-cell constructs and test in both in vitro and in animal models of solid tumors with a goal of completing IND-enabling preclinical studies.
Nicox SA, of Sophia Antipolis, France, said it received FDA approval for Zerviate (cetirizine ophthalmic solution 0.24 percent), the first topical ocular formulation of the antihistamine, for the treatment of ocular itching associated with allergic conjunctivitis. The efficiency of the drug was established in three randomized, double-masked, placebo-controlled, conjunctival antigen challenge trials in patients with a history of allergic conjunctivitis. Onset and duration were evaluated in two of those trials in which Zerviate demonstrated, statistically and clinically, significantly less ocular itching compared to vehicle at 15 minutes and eight hours after treatment.
Novo Nordisk A/S, of Bagsvaerd, Denmark, said the FDA approved the BLA for Rebinyn (nonacog beta pegol), an extended half-life factor IX molecule, for the treatment of adults and children with hemophilia B. It is indicated for on-demand treatment and control of bleeding episodes and the perioperative management of bleeding around the time of surgery.
Pfizer Inc., of New York, said a supplemental new drug application (sNDA) for Sutent (sunitinib) has been accepted for filing by the FDA. If approved, the sNDA would expand the use of Sutent to include it as an adjuvant treatment in adult patients at high risk of recurrent renal cell carcinoma following nephrectomy. The PDUFA date is in January 2018.
Promis Neurosciences Inc., of Toronto, said it identified a therapeutic epitope target on misfolded forms of TDP43, implicated in the development and progression of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The company has filed a provisional patent application for that target with the U.S. Patent Office. TDP43 plays a critical role in the response of cells to oxidative stress, but in ALS, FTD and other neurodegenerative diseases, it can lose its normal function, forming intracellular aggregates of misfolded TDP43 that disrupt cellular energy generation and normal protein degradation.
Proqr Therapeutics NV, of Leiden, the Netherlands, said it received FDA fast track designation for QR-110, the lead molecule in its ophthalmology pipeline being developed for the treatment of patients with Leber’s congenital amaurosis type 10 (LCA 10), a rare genetic disease that causes individuals to lose sight, often in the first years of life. QR-110 is an investigational RNA oligonucleotide targeting LCA 10 due to the p.Cys998X mutation, which is one of the most prevalent forms of gene-related blindness in children, the company said, and currently there are no disease-modifying therapies commercially available or in clinical development.
Sandoz, of Holzkirchen, Germany, a Novartis AG division, said the EMA has accepted for regulatory review the marketing authorization applications for biosimilars to North Chicago-based Abbvie Inc.’s Humira (adalimumab) and New Brunswick, N.J.,-based Johnson & Johnson’s Remicade (infliximab), both of which are used to treat immunological diseases. Sandoz is seeking approval for biosimilar adalimumab and infliximab for use in all indications of their respective reference medicines.
Servier SAS, of Neuilly-sur-Seine, France, and Iltoo Pharma SAS, of Paris, entered an exclusive license option agreement for the development and commercialization of the ILT-101 drug candidate (low-dose interleukin-2) for the treatment of autoimmune diseases, with systemic lupus erythematosus as the first target indication. The option shall be triggered in 2018 when the results of the ongoing phase II study in lupus patients are known. Under the terms of the agreement, and depending on the results of the trial, Servier may decide to exercise the license option to develop and commercialize ILT-101 worldwide in the treatment of autoimmune diseases, except for the U.S. and Japan, where Iltoo retains exclusive rights. Iltoo a will receive €8 million (US$8.9 million) immediately, and subsequent payments comprising one up-front payment upon exercise of the option, plus development and sales milestones worth a total of €200 million. The agreement also provides for double-digit royalty payments on future sales.