Glaxosmithkline plc (GSK) waded into the competitive nonalcoholic steatohepatitis (NASH) waters by way of an exclusive global licensing deal (excluding China) with Arrowhead Pharmaceuticals Inc., under which GSK will develop and commercialize ARO-HSD, Arrowhead’s RNAi therapy targeting HSD17B13. The compound is undergoing a phase I/II trial. Arrowhead banks an up-front payment of $120 million and is eligible for $30 million at the start of phase II plus $100 million upon achieving a successful phase II trial readout along with dosing the first patient in a phase III experiment. If phase III results prove positive in NASH and ARO-HSD wins regulatory approval in major markets, Pasadena, Calif.-based Arrowhead would collect up to $190 million at the first commercial sale, and as much as $590 million in sales-related milestone payments, along with tiered royalties. Shares of the firm (NASDAQ:ARWR) were trading midday at $66.26, down $3.01.

Regeneron buys into Nykode’s vaccine platform in potential $925M deal

DUBLIN – A meaty licensing deal with Regeneron Pharmaceuticals Inc. is as good a time as any to unveil a new corporate identity. Nykode Therapeutics AS, the Norwegian immunotherapy firm formerly known as Vaccibody, is banking $30 million up front, another $20 million as an equity investment, and could earn up to $875 million more in development and commercial milestones across a multiproduct deal to develop vaccines for cancer and infectious disease. Oslo-based Nykode could also earn tiered sales royalties, ranging from high single digits to low double digits in percentage terms, while Tarrytown, N.Y.-based Regeneron is also picking up the tab on all research, development and commercialization costs.

FDA approves Aadi’s Fyarro as first treatment in rare cancer

The first approval for treating advanced malignant perivascular epithelioid cell tumor (PEComa) in adults has been awarded by the FDA to Aadi Bioscience Inc.’s Fyarro (sirolimus protein-bound particles for injectable suspension; albumin-bound). The treatment showed clinically durable responses in mTOR inhibitor-naïve patients with locally advanced unresectable or metastatic PEComa, buoying the approval. Clinical studies also demonstrated an acceptable and manageable safety profile. PEComa is a rare mesenchymal tumor, a subset of soft-tissue sarcomas with an undefined cell of origin. They are found primarily in women and malignant cases are extremely rare. The approval was awarded two days before the Nov. 26 priority NDA’S PDUFA date.

Newco News: Loqus23 targeting DNA damage repair processes for Huntington’s, other TRDs

LONDON – Newco Loqus23 Therapeutics Ltd. has closed a £7 million (US$9.4 million) extension of its seed round, providing the means to progress its lead small-molecule program in Huntington’s disease through lead optimization. Cambridge, U.K.-based Loqus23 aims to discover orally available small molecules to inhibit aberrant DNA damage repair processes that are at the heart of Huntington’s and other triplet repeat diseases (TRDs) caused by abnormal trinucleotide repeat expansion. These include myotonic dystrophy type 1, fragile X syndrome and spinocerebellar ataxia.

COVID-19 spurring antimicrobial resistance

The Pan American Health Organization (PAHO) is echoing fears that the COVID-19 pandemic will result in a surge of drug-resistant infections, saying surges already are being seen in several countries in the Americas, including Argentina, Ecuador, Guatemala, Paraguay and Uruguay. In addition to the infections, a recent PAHO epidemiological alert showed a spike in the emergence of bacteria resistant to antibiotics in the region. PAHO officials are attributing much of the surge to increased hospitalizations due to the pandemic and inappropriate antibiotic use in treating patients with COVID-19 infections.

Clover to receive $36.9M from CEPI for COVID-19 vaccine development

The Coalition for Epidemic Preparedness Innovations (CEPI) has agreed to provide Clover Biopharmaceuticals Ltd. with an additional $36.9 million to support development of SCB-2019, a recombinant trimeric spike-protein subunit vaccine candidate that could become one of the first protein-based COVID-19 vaccines commercialized globally through the COVAX Facility. The latest capital injection brings CEPI’s total investment in the asset to $397.4 million following earlier support for preclinical and clinical testing, as well as scale-up of manufacturing for the candidate.

Daiichi Sankyo releases top-line phase III data for FLT3 inhibitor quizartinib

Daiichi Sankyo Co. Ltd. has released top-line results from the phase III Quantum-First study of quizartinib in patients with newly diagnosed FLT3-ITD positive acute myeloid leukemia showing the candidate met the trial’s primary endpoint of overall survival (OS). The readout showed that patients treated with quizartinib, in combination with standard induction and consolidation chemotherapy and then with quizartinib again, had a statistically significant and clinically meaningful improvement in OS compared to those who received standard treatment alone, Ken Takeshita, global head of R&D at Daiichi Sankyo, told BioWorld.

Hahn faces House grilling on political interference at FDA

Former FDA Commissioner Stephen Hahn is being asked to spill the beans on political interference at the U.S. agency during the emergence of COVID-19 last year. Under the guise of investigating the federal government’s response to the pandemic “in order to better understand what went wrong, identify ways to improve the country’s response and determine what corrective steps are necessary to ensure our nation is better prepared for any future public health crisis,” Rep. James Clyburn (D-S.C.), chair of the U.S. House Select Committee on the Coronavirus Crisis, wrote to Hahn Nov. 22, demanding all documents he has related to a host of allegations of administration interference in the FDA’s authorization decisions on potential pandemic treatments. As part of the subcommittee’s investigation, Clyburn also asked Hahn to sit for a transcribed interview Dec. 13.

Delays in FDA foreign inspections becoming a concern

U.S. trading partners are raising concerns about the FDA’s continued delays, due to the COVID-19 pandemic, in inspecting foreign drug manufacturing facilities. Those delays, which can impact drug approvals, were one of the regulatory issues India trade officials raised at the 12th ministerial-level meeting of the India-United States Trade Policy Forum Nov. 23. While U.S. officials acknowledged the concern, they provided no timeline for the FDA to resume its full foreign inspection program. Instead, they reiterated that the agency will conduct prioritized inspections when there is minimal risk to company and FDA officials and it will continue to use remote evaluation techniques for regulatory decisions as appropriate.

Also in the news

Aadi, AB Science, Aligos, Amryt, Aptevo, Astrocyte, Beigene, Bionomics, Bristol Myers Squibb, Dnatrix, Dragonfly, Evrys, Galapagos, Gilead, Ingeneron, Johnson & Johnson, Kancera, Marvel, Mission, Neonmind, Neurophth, Ocuphire, Oncotelic, Oramed, Qurient, RDIF, Reveragen, Revive, Santhera, Scynexis, Sellas, Seqirus, Serum, Simcere, Sterna, Supernus, Synlogic, Theratechnologies, Tonix, Transgene, Vipergen