Abcellera Inc., of Vancouver, British Columbia, reported preclinical data for LY-CoV1404, a second antibody candidate from its collaboration with Eli Lilly and Co., of Indianapolis, targeting COVID-19. Results showed LY-CoV1404 binds to a rarely mutated region of the SARS-CoV-2 spike protein and neutralizes all currently known variants of concern, including those first identified in the U.K. (B.1.1.7), South Africa (B.1.351), Brazil (P.1), California (B.1.426 and B.1.429) and New York (B.1.526). LY-CoV1404 also is highly potent, which Abcellera said could have implications for reducing the amount of antibody necessary for clinical dosing, and potentially enabling a subcutaneous route of administration for either treatment or prophylaxis of COVID-19. The drug has been added to Lilly’s ongoing Blaze-4 phase II trial in patients with mild to moderate COVID-19.
Abpro Corp., of Woburn, Mass., reported preclinical results showing ABP-201, a bispecific antibody co-targeting VEGF and ANG2 in development for treating angiogenetic eye pathologies, reduced neovascular lesion formation and vascular leakage in the rodent model of choroidal neovascularization. The drug also performed equally or better than Eylea (aflibercept, Regeneron Pharmaceuticals Inc.) for inhibition of lesion formation and vascular leakage. Data were presented at the Association for Research in Vision and Ophthalmology meeting.
Alligator Bioscience AB, of Lund, Sweden, said it entered a joint research agreement with Bioarctic AB, of Stockholm, under which Alligator will employ its antibody generation technologies to develop new product candidates in the neurodegenerative field. Financial terms were not disclosed.
Aphios Corp., of Woburn, Mass., said research demonstrated that orally administered nanoparticle-encapsulated Bryostatin-1 activates alpha-secretase and PKC isoforms in vitro, and facilitates acquisition and retention of spatial learning in vivo in an Alzheimer’s disease mouse model.
Aura Biosciences Inc., of Cambridge, Mass., presented preclinical data at the Association for Research in Vision and Ophthalmology, showing that lead virus-like drug conjugate AU-011, currently in phase II development for choroidal melanoma, showed potential for the treatment of choroidal metastases from breast and lung cancer, which are the most common causes of metastases in the choroid. In both cell line panels, AU-011 demonstrated potent cell binding and cell killing activity with EC50 in the picomolar range. In vivo, a single systemic administration in a syngeneic breast cancer mouse model demonstrated potent cytotoxic activity with significant reduction of tumor growth, further corroborating data previously obtained in other cancer models. The company also evaluated the distribution of AU-011 with both intravitreal and suprachoroidal administration in an orthotopic rabbit model of choroidal melanoma, which demonstrated both routes of administration achieved tumor distribution to the choroid and to the tumor.
Biocartis Group NV, of Mechelen, Belgium, said it signed a new agreement with Astrazeneca plc, of Cambridge, U.K., aimed at providing access to Idylla EGFR testing products at selected hospital sites in Biocartis’ European and global distributor markets to support identification of patients with EGFR mutations. Terms were not disclosed.
Cartesian Therapeutics Inc., of Gaithersburg, Md., disclosed a $2 million competitive R&D award from the National Heart, Lung, and Blood Institute of the U.S. NIH for clinical development of Descartes-30, the first RNA cell therapy for respiratory diseases. This is the fourth SBIR grant awarded to Cartesian.
Edgewise Therapeutics Inc., of Boulder, Colo., disclosed the publication of the first cross-sectional, retrospective study to describe selective elevation of fast but not slow skeletal muscle fiber injury biomarkers in the blood of patients with Becker and Duchenne muscular dystrophy (DMD) in Muscle & Nerve. Previous studies have demonstrated that DMD patient muscle is more prone to fast fiber injury compared to slow fiber injury. This study extends those findings to examine fiber-type specific biomarkers of muscle injury in patient blood.
Effector Therapeutics Inc., of San Diego, disclosed a $5 million cooperative agreement sponsored by the Defense Advanced Research Projects Agency (DARPA) and the Defense Health Agency to fund a planned phase Ib study of zotatifin (eFT-226), as an antiviral agent in an outpatient setting for those with mild to moderate COVID-19 disease. The agreement is a subaward of a DARPA research program awarded to the Quantitative Biosciences Institute at the University of California, San Francisco. Zotatifin is an investigational small-molecule inhibitor of eukaryotic initiation factor 4A.
Enesi Pharma Ltd., of Oxford, U.K., said it successfully developed an Implavax-enabled, solid-dose recombinant H7N9 pandemic influenza vaccine and delivered proof-of-concept results in validated preclinical models. The project was conducted as part of a government initiative focused on developing alternative technologies.
Esteve Pharmaceuticals SA, of Barcelona, licensed rights to its pain treatment, E-58425, in the U.S. to Kowa Pharmaceuticals America Inc., a unit of Nagoya, Japan-based Kowa Co. Ltd. Esteve will supply the product. Financial terms of the deal weren’t disclosed. E-58425, a cocrystal form of the anti-inflammatory celecoxib and the analgesic tramadol, is currently under review by the FDA with a decision expected in 2021.
Evotec SE, of Hamburg, Germany, and the University Hospital Erlangen disclosed a partnership regarding molecular analyses of biospecimens from the German Chronic Kidney Disease (GCKD) cohort study. The research team will make use of Evotec's Evopanomics and Evopanhunter platforms for deep molecular profiling of patient samples, the company said. Started in 2009, the GCKD was designed to be the world's largest cohort study on chronic kidney disease.
Exelixis Inc., of Alameda, Calif., and Gamamabs Pharma SA, of Toulouse, France, disclosed an agreement under which Exelixis will, upon the future closing of the asset purchase and subject to certain conditions to closing, acquire all rights, title and interest in Gamamabs’ AMHR2 antibody technology. Exelixis will pay Gamamabs $5 million upon signing of the agreement, make additional payments upon completion of closing conditions, and make additional milestone payments after closing, contingent upon various events. Once the transfer is completed, Exelixis will control 100% of Gamamabs’ AMHR2 franchise technology, including all assets pertaining to Gamamabs’ monoclonal antibody drug product murlentamab (GM-102).
Gain Therapeutics Inc., of Bethesda, Md., reported preclinical data for its structurally targeted allosteric regulators (STARs) GBA1 Parkinson’s disease program, GT-02287 and GT-02329, at the XXVI International Association of Parkinsonism and Related Disorders World Congress. In human fibroblasts isolated from patients with GBA1-related Gaucher disease, both STAR compounds stabilized GCase and enhanced its enzymatic activity. In primary rat dopaminergic neurons, both compounds were neuroprotective and lowered alpha-synuclein pathology. When given orally to mice, GT-02329 enhanced GCase activity and protein levels in the striatum. GT-02287 reduced alpha-synuclein accumulation in rat models of Parkinson’s disease. In rats treated with rotenone to produce certain features of the human disease, GT-02287 improved locomotor activity.
Gemini Therapeutics Inc., of Cambridge, Mass., reported preclinical data on GEM-103, its human recombinant complement factor H, at the 2021 Association for Research in Vision and Ophthalmology Virtual meeting. The drug reduced the severity of choroidal neovascularization in a laser-induced mouse model of wet age-related macular degeneration. The reduction in severity, as measured by fluorescent angiography and optical coherence tomography, was comparable to the reduction produced by Eylea (aflibercept, Regeneron Pharmaceuticals Inc.).
Gensight Biologics SA, of Paris, published the results of Reality, a natural history study of 44 patients with Leber hereditary optic neuropathy, in Eye. The fitted curve for best-corrected visual acuity over time showed a marked loss of vision during the acute phase of the disease, which was followed by a continuous deterioration of vision over the three-year follow-up.
Hcmed Innovations Co. Ltd., of Taipei, Taiwan, said it initiated a strategic partnership with CSL Behring, a unit of CSL Ltd., of Melbourne, Australia, to develop CSL’s plasma-derived immunoglobulin, CSL-787, for administration using Hcmed's vibrating-mesh nebulizing device. Hcmed will lead development of the customized nebulizing device, optimized for CSL’s formulation, with CSL retaining sole responsibility to develop and commercialize the combination product. Hcmed is set to receive an up-front payment and device and development milestones and, upon execution of a subsequent agreement for commercial supply of the device, royalties from global net sales.
Hoth Therapeutics Inc., of New York, plans to develop its HT-KIT mRNA frame shifting therapeutic for multiple orphan diseases, including aggressive systemic mastocytosis, systemic mastocytosis with associated hematological neoplasm, mast cell leukemia, acute myeloid leukemia and gastrointestinal stromal tumors.
Hutchmed (China) Ltd., formerly Hutchison China Meditech Ltd., of Hong Kong, said trading in shares under its new corporate name is expected to begin on May 5 after issuance of a replacement certificate of incorporation under the name in the Cayman Islands. The company’s tickers remain unchanged. On May 4, its American depositary shares (NASDAQ:HCM) lost 82 cents, finishing at $27.18.
Immunicum AB, of Stockholm, will said preclinical data for ilixadencel and DCP-001, to be presented at the 18th Cancer Immunotherapy meeting, showed that ilixadencel plus an anti-CTLA4 checkpoint inhibitor completely eradicated tumors in 50% to 80% of the treated animals in a CT26 colon carcinoma animal model. DCP-001 induced production of various pro-inflammatory cytokines and chemokines by peripheral blood mononuclear cells. Immature monocyte-derived dendritic cells took up the DCP-001 vaccine, which was enhanced when cells were exposed to a CD47 blockade.
Logicbio Therapeutics Inc., of Lexington, Mass., presented results from a retrospective study of the disease course in pediatric patients living with severe methylmalonic acidemia (MMA) caused by a deficiency of the mitochondrial enzyme methylmalonyl-CoA mutase (MMUT) in a poster presentation at the American College of Medical Genetic and Genomics annual clinical genetics meeting. The non-interventional natural history study collected data via chart review from 18 people diagnosed with severe MMA by newborn screening, including those managed medically from birth to 3 years of age (cohort 1) and pediatric-age recipients of liver transplant (cohort 2), who were observed for one year prior to and up to three years following transplantation. Results showed significant and sustained decrease in methylmalonic acid levels post-transplantation compared to baseline. While not approaching normalization, levels showed reduced variability, suggesting improved metabolic stability.
Moderna Inc., of Cambridge, Mass., established an agreement with Gavi, the vaccine alliance, to supply 500 million doses of the COVID-19 Vaccine Moderna. The company plans to deliver 34 million doses in the fourth quarter of 2021. Gavi will have the option to procure the remaining 466 million doses in 2022. The doses will be sold at Moderna’s lowest tiered price and be made available to the 92 Gavi COVAX Advance Market Commitment low- and middle-income countries. In separate news, Moderna said it expanded the Moderna Technology Center in Norwood, Mass., increasing production and lab space from approximately 300,000 square feet to approximately 650,000 square feet by renovating existing space and acquiring a 240,000-square-foot building on the same campus. The expansion is expected to support a 50% increase in production of Moderna’s COVID-19 vaccine at the manufacturing site, expected to occur in late 2021 and early 2022.
Moonlake Immunotherapeutics AG, of Zug, Switzerland, licensed sonelokinab (M-1095/ALX-0761), a trispecific nanobody IL-17A/F inhibitor with an albumin binding site, from Merck KGaA, of Darmstadt, Germany. Moonlake plans to develop the drug for multiple inflammatory diseases in dermatology and rheumatology. Financial terms of the deal weren’t disclosed. Moonlake was founded in 2021 with an investment by Merck, among others.
Nflection Therapeutics Inc., of Wayne, Pa., said its scientists and researchers from the Tsai Lab at Moffitt Cancer Center and the Sarin Lab at the Stanford University School of Medicine presented preclinical data at the Society for Investigative Dermatology's 2021 virtual meeting suggesting that its topical gel, NFX-179, prevented cutaneous squamous cell carcinoma (cSCC) in a UV-induced mouse model. At 28 days, NFX-179 gel reduced the formation of new cSCCs by an average of 92% at doses of 0.5% and greater, with no apparent systemic or skin toxicities. In a split-back randomized controlled study in five mice per group, near-complete suppression of cSCC was seen only in the drug-treated area. The team also reported that NFX-179 inhibited the growth of human cSCC cell lines in a dose-dependent manner, penetrated human skin and inhibited ERK signaling in human cSCC explants.
A team of researchers in the fields of microbiology and artificial intelligence said they launched Novobiome SA, of Paris, to develop live biotherapeutics targeting diseases that affect the nexus of liver and intestinal diseases and the gut microbiome, using an ex vivo R&D platform known as Novosift to analyze the symbiotic relationship between the microbiota and the human intestine. The Novosift platform incorporates high-throughput functional metagenomics, human intestinal organoids, high resolution fluorescent confocal microscopy and a deep learning AI algorithm. The company’s first candidate, ADC-001, which comprises an anti-inflammatory bacterium member of the Coriobacteriia class, targets hepatic conditions such as nonalcoholic fatty liver disease and nonalcoholic steatohepatitis, with the goal of entering the clinic by late 2022 or early 2023.
Ocugen Inc., of Malvern, Pa., reported data from scientists at the Indian Council of Medical Research-National Institute of Virology that showed sera from Covaxin-vaccinated people was able to neutralize several SARS-CoV-2 variants: B.1.617 (India, double mutant), B.1.1.7 (U.K.), B.1.1.28 (Brazil P2) and a heterologous strain.
Plus Therapeutics Inc., of Austin, Texas, said it formed two collaborations to support process development and analytical chemistry activities for the cGMP manufacturing of lead asset rhenium nanoliposome (RNL), in development to treat recurrent glioblastoma. A preclinical, clinical and process development agreement with Invicro LLC, of Boston, a unit of Konica Minolta Inc., covers characterization of current manufacturing process and development of in-process manufacturing controls for the RNL active pharmaceutical ingredient (API) and final drug product and provides future clinical trial imaging support and drug development consulting. A separate pact allows contract research organization Eurofins Biopharma Inc. to develop and validate test methods for purity, composition and identity of the API in RNL.
Proqr Therapeutics NV, of Leiden, the Netherlands, and RTW Investments LP said newly formed Yarrow Biotechnology Inc. in-licensed exclusive rights to Proqr’s antisense oligonucleotide technology to develop and commercialize therapies for an undisclosed non-ophthalmic target. Proqr is eligible to receive up to $115 million in up-front and milestone payments plus single-digit royalties on net sales and the right to receive an undisclosed equity percentage in the form of Yarrow common shares . Proqr will conduct certain preclinical activities, with reimbursement from Yarrow for research costs, while Yarrow assumed responsibility to continue development and commercialization activities. In conjunction with the agreement, Gerard Platenburg, chief innovation officer at Proqr, is set to join Yarrow’s board.
Recce Pharmaceuticals Ltd., of Sydney, Australia, said RECCE-327 showed bactericidal activity against six antibiotic-resistant ESKAPE pathogens, including drug-resistant mutations known as Superbugs and two additional pathogens on the World Health Organization priority list. Independent studies showed RECCE-327 exceeded 99.9% effectiveness against the full suite of ESKAPE pathogens within hours of exposure to the candidate, a bactericidal permeability protein inhibitor.
Relief Therapeutics Holding AG, of Geneva, said it signed a binding term sheet covering acquisition of the outstanding shares of privately held Applied Pharma Research SA (APR), of Balerna, Switzerland. At the transaction’s close, APR shareholders are set to receive CHF22 million (US$24.1 million) in cash plus additional cash relating to APR's working capital position, and CHF50 million in Relief common registered shares. APR shareholders also are eligible for contingent payments comprising a combination of cash and Relief common registered shares, predicated on the achievement of established milestones. Relief holds an exclusive right for 60 days to negotiate and close the transaction based on terms and conditions in the term sheet.
Samsung Bioepis Co. Ltd., of Incheon, Korea, presented data comparing SB-11 (ranibizumab) to Lucentis (ranibizumab, Roche Holding AG) at the Association for Research in Vision and Ophthalmology Annual Meeting 2021. The amino acid sequence, post-translational modifications, higher order structure and size and charge variants of SB-11 were similar to those of Lucentis. SB-11’s binding activity based on VEGF-A binding assay was 98% potencies based on human umbilical vein endothelial cells antiproliferation and VEGF-A neutralization assay of 101% and 99%, respectively.
Sesen Bio Inc., of Cambridge, Mass., said it expanded its commercial leadership team and engaged contract sales organization Syneos Health for U.S. field sales support for lead candidate Vicineum (oportuzumab monatox), an epCAM modulator under review by the FDA to treat non-muscle invasive bladder cancer.
Stoke Therapeutics Inc., of Bedford, Mass., reported preclinical data showing in vitro protein up-regulation and improved mitochondrial function in OPA1 protein-deficient fibroblasts derived from people with autosomal dominant optic atrophy (ADOA), primarily caused by OPA1 protein deficiency. The data showed antisense oligonucleotides from the company’s TANGO platform reduced non-productive exon inclusion and increased total OPA1 mRNA expression in three patient fibroblast cell lines with different mutations, increased expression of multiple OPA1 protein isoforms by approximately 35% to 47% and produced dose-dependent improvement in mitochondrial bioenergetics. The data were presented at the Association for Research in Vision and Ophthalmology 2021 annual meeting and will be discussed at the upcoming American Society of Gene and Cell Therapy annual meeting.
Twoxar Inc., of Palo Alto, Calif., said it plans to rebrand as Aria Pharmaceuticals to reflect the artistry and complexity that defines its scientific approach to developing medicines from discovery to FDA approval. The company also said it is transitioning from discovery collaborations with pharmaceutical companies to advancing its own pipeline, beginning with IND-enabling studies for preclinical candidates in chronic kidney disease, lupus and idiopathic pulmonary fibrosis.
Xoma Corp., of Emeryville, Calif., earned a $500,000 milestone payment from Janssen Biotech Inc., a unit of New Brunswick, N.J.-based Johnson & Johnson, as a result of the first patient being treated in a phase III trial of cetrelimab.