Analysis of the UK Biobank from Alnylam Pharmaceuticals Inc., of Cambridge, Mass., demonstrated a significant association with V122I, a mutation of the transthyretin gene. The data showed V122I has an increased likelihood of polyneuropathy. The findings showed a need for broader assessment of a patient's overall health to look for multisystem manifestations of hereditary transthyretin amyloidosis, which often includes cardiomyopathy and polyneuropathy, the company said.

Anokion SA, of Lausanne, Switzerland, released preclinical data showing liver-targeted islet antigens (chromogranin-A) were protected from hyperglycemia in its BDC2.5 model of type 1 diabetes. The data showed tolerogenic T-cell mechanisms effective at preventing or reducing disease pathology, including mouse models of type 1 diabetes and multiple sclerosis. The preclinical model showed Anokion's liver-targeted antigens prime antigen-specific T cells toward tolerogenic phenotypes, including expression of inhibitory and exhaustion markers, clonal T-cell deletion, and the induction of regulatory T-cell responses. The mechanisms also translated to nonhuman primates using a novel model of antigen-specific T-cell tolerance in a cynomolgus macaque. Anokion just acquired Kanyos Bio Inc. and closed a $40 million series B. (See BioWorld, Sept. 11, 2019.)

Applied Therapeutics Inc., of New York, released preclinical data showing its aldose reductase inhibitor, AT-001, reduced cardiac damage in a mouse model of diabetic cardiomyopathy and showed promise inhibiting aldose reductase within a safe ranging dose. The data also showed that in humans, single and multiple ascending doses were well-tolerated. The data come from Applied Therapeutics' phase I/II trials of diabetic patients with heart failure.

Biogen Inc., of Cambridge, Mass., reported data from two real-world observational studies of Plegridy (peginterferon-beta-1a) and Avonex (interferon-beta-1a) in multiple sclerosis, which found that exposure to interferon-beta treatment before conception and/or during pregnancy is not expected to have adverse effects on pregnancy or infant growth outcomes. Data were presented at the European Committee for Treatment and Research in Multiple Sclerosis meeting in Stockholm.

Castle Creek Pharmaceutical Holdings, of Parsippany, N.J., will acquire Fibrocell Science Inc., an Exton, Pa.-based cell and gene therapy company, for $63.3 million. Fibrocell stockholders will receive an all-cash consideration of $3 per share. The sale is scheduled to close by year-end. Fibrocell's pipeline includes FCX-007, a late-stage gene therapy candidate for treating recessive dystrophic epidermolysis bullosa, a congenital and progressive orphan skin disease caused by the deficiency of the protein COL7, and FCX-013, a gene therapy candidate for treating moderate to severe localized scleroderma.

Crestone Inc., of Boulder, Colo., has been awarded a $17.8 million contract from the NIH to help develop a phase II study of CRS-3123, its candidate for treating Clostridioides difficile. The candidate is designed to block production of disease-causing pathogens and to minimally disrupt healthy gastrointestinal microbiota. The trial will be randomized, double-blinded and include three 10-day treatment arms of twice-daily doses of 200 mg or 400 mg CRS-3123 compared to vancomycin 125 mg four times per day at 30 sites across the U.S.

CTD Holdings Inc., of Alachua, Fla., said it received notice that an individual IND was cleared by the FDA for allowing investigational Trappsol Cyclo to a single pediatric patient diagnosed with Niemann-Pick disease type C. The company said it will provide the hydroxypropyl beta cyclodextrin drug to the patient under expanded access.

A committee from the board at Eidos Therapeutics Inc., of San Francisco, unanimously rejected a nonbinding proposal from Palo Alto, Calif.-based Bridgebio Pharma Inc. to purchase all its outstanding common stock. The proposal was for a fixed exchange ratio of 1.3 shares of common stock for each share of Eidos common stock. Bridgebio focuses on treating diseases caused by transthyretin amyloidosis.

Knopp Biosciences LLC, of Pittsburgh, said data demonstrating that compound subgroups from its library of potassium channel modulators selectively target Kv7 channel subtypes associated with a wide range of neurological and smooth muscle diseases, are being presented at the International Kv7 Channels Symposium in Naples, Italy. The data demonstrate that specific subgroups of Kv7 compounds selectively modulate different Kv7 channel subtypes across ranges of channel activation and inhibition, enabling selection of compounds capable of targeting neurological disorders associated principally with Kv7.2/7.3 channels and others associated principally with Kv7.4 channels. Knopp's lead Kv7 modulator, KB-3061, is in preclinical development for KCNQ2 epileptic encephalopathy, a rare neonatal disease characterized by seizures beginning in the first days of life and by significant developmental delay.

Merck Serono SA, a unit of Merck KGaA, of Darmstadt, Germany, reported real-world data from a post-hoc analysis of the Clarity and Clarity extension trials that found 75% of patients showed no disability progression at five years post-treatment with adenosine deaminase inhibitor Mavenclad (cladribine). No new safety findings from up to 10 years of clinical program follow-up and post-approval data were noted.

Vectura Group plc, of Chippenham, U.K., said that following an award of $89.7 million in damages by a jury in the U.S. District Court for the District of Delaware on May 3, presiding U.S. District Judge Richard Andrews has ruled on the parties' post-trial motions upholding the award, and ongoing royalties of 3% on U.S. sales of certain infringing Ellipta products from Glaxosmithkline plc, supplemental damages based on GSK's infringing sales of approximately $10.5 million, and pre-judgment interest at the prime rate of approximately $6.7 million.

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