DUBLIN Dyno Therapeutics Inc., an early stage gene therapy firm applying artificial intelligence to advanced capsid engineering, has entered partnerships with Novartis AG and Sarepta Therapeutics Inc., in ophthalmic indications and muscle diseases, respectively, which have more than $2 billion in biobucks attached. Further financial details are scant, although it could receive more than $40 million in up-front, option and license payments during the research phase of the Sarepta alliance. The two deals are evidence of the market appetite for improved adeno-associated viral vectors. We had a significant amount of interest even before the company was formed, Dyno CEO and co-founder Eric Kelsic told BioWorld.

The phase III pivotal trial of mavacamten, an oral, allosteric cardiac myosin modulator for treating symptomatic, obstructive hypertrophic cardiomyopathy, from Myokardia Inc., of Brisbane, Calif., hit its primary and all secondary endpoints. The data show mavacamten was well-tolerated and demonstrated safety results comparable to placebo. Ninety-eight percent of patients enrolled completed the study. The company stock (NASDAQ:MYOK) was met by enthusiasm midday Monday, as shares swelled by 62%. Myokardia said it plans to submit an NDA in the first quarter of 2021. Myokardia is developing mavacamten to treat conditions in which excessive cardiac contractility and impaired diastolic filling of the heart are the underlying cause.

After being hit with the major market meltdown during March, public biopharmaceutical companies developing new medicines put that behind them with a dramatic surge in valuations in April. As a result, the BioWorld Drug Developers index recorded an almost 20% increase during the period, with that momentum continuing into early May.

By delivering the protein follistatin via gene therapy, researchers at Washington University in St. Louis were able to increase skeletal muscle mass, decrease inflammation and reverse obesity-related arthritis in mice who developed osteoarthritis as a result of a high-fat diet. They reported their results in the May 8, 2020, online issue of Science Advances.

HONG KONG The Japanese government is tightening its grip on its listed companies, including those working on promising COVID-19 treatments. On May 8, the Japanese Ministry of Finance releaseda list of 518 companies that would be subject to stricter restrictions on receiving foreign investments. Starting on June 7, foreign investors buying a stake of 1% or more in Japanese firms will be pre-screened, compared with the previous limit of 10%. In a statement, the ministry said the move was related to the degree of impact of the investment on maintaining the basis of production and technologies in the business sectors that relate to protection of national security, maintenance of public order, or safeguard of public safety.

DUBLIN Tolerogenixx GmbH is on track to move its cell-based immune tolerance induction therapy for kidney transplant recipients into a 200-patient phase IIb trial, following the publication of promising data from a phase Ib trial in 10 patients, in which all participants had a successful transplant at one-year follow-up, including those in a high-dose group on a reduced immunosuppression regimen.

Having a COVID-19 therapy approved through an emergency use authorization (EUA) is not the same as having access to it, even if its free. With one-third of the COVID-19 cases confirmed globally as of today and 28.5% of the deaths, the U.S. is getting 40% of the 1.5 million doses of remdesivir Gilead Sciences Inc. is donating worldwide. The federal government last week began doling out the 607,000 doses to the states, which are then charged with distributing them to the hospitals with the greatest need. But given the supply and manufacturing timeline for the first drug granted an EUA to treat severe COVID-19 cases, more than 300,000 eligible U.S. patients likely will not have access to the drug through the end of July, said Brian Abrahams, a senior analyst with RBC Capital Markets LLC.

HONG KONG South Koreas Hanmi Pharmaceutical Co. Ltd. has filed a new drug approval application for Rolontis (eflapegrastim) with the countrys Ministry of Food and Drug Safety. Rolontis, a biologic to treat neutropenia, is the first of its kind in South Korea and, according to Hanmi, the first to be developed using the companys Lapscovery platform.

About 15 months after closing its multibillion-dollar acquisition of Loxo Oncology Inc., Eli Lilly and Co. has secured an accelerated FDA approval for the first of the deal's headline assets, the RET kinase inhibitor selpercatinib, now branded as Retevmo. The green light, following a priority review, allows for marketing of the drug as a treatment for three types of tumors non-small-cell lung cancer, medullary thyroid cancer and other types of thyroid cancers in patients whose tumors have a rearranged during transfection alteration. The decision arrived well ahead of an earlier-projected third-quarter decision by the agency.

Three biopharma drugs are up for FDA approval this week, including one new chemical entity, dasotraline, from Sunovion Pharmaceuticals Inc. to treat binge eating disorder, and two other candidates that are part of supplemental filings for expanded oncology indications. Sunovions drug and Blueprint Medicines Corp.s Ayvakit (avapritinib) to treat fourth-line gastrointestinal stromal tumors (GIST), have PDUFA dates set for May 14, while Clovis Oncology Inc.s Rubraca (rucaparib) for prostate cancer is scheduled for May 15.

The articles from BioWorlds ongoing coverage of the COVID-19 coronavirus outbreak are available at http://www.bioworld.com/coronavirus. Note that we have added three critical tables which are constantly updated:

4D Pharma, 9 Meters, Abbvie, ADC, Amag, Appili, Artara, Astrazeneca, Bellerophon, Biomarin, Bluebird, Bridgebio, Burning Rock, Cellect, Cocrystal, Crispr, Daicchi, Dar, Faron, Histogen, Hummingbird, Infinity, Inmune, Intercept, Jazz, Kiniksa, Kura, Lilly, Merck, Mimedx, Myokardia, Navidea, Noxxon, Orexo, Oyster Point, Pliant, Plus, Portage, Protagonist, Protalix, Regeneron, Relief, Ritter, Strongbridge, Themis, Tiburio, Tolerogenixx, Tracon, Vertex

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Novartis, Sarepta join Dyno's enterprise to boldly go to new gene therapy frontier - BioWorld Online

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