Shares of Travere Therapeutics Inc. (NASDAQ:TVTX) were trading midday at $15.83, down $6.78 or almost 30%, after the firm disclosed top-line primary efficacy results from the pivotal phase III study called Duplex with sparsentan, a dual endothelin angiotensin receptor antagonist, in focal segmental glomerulosclerosis (FSGS). The results fell short of the primary efficacy endpoint, estimated glomerular filtration slope over 108 weeks of treatment, though secondary and top-line exploratory endpoints trended favorably for the drug, which was cleared as Filspari by the U.S. FDA for IgA nephropathy (IgAN). Some investors fretted over whether the Duplex results in FSGS, characterized by kidney scarring, might read through to two-year data with the drug in IgAN from the phase III effort called Protect.

Avadel preps to launch newly approved Lumryz 

Now that Avadel Pharmaceuticals plc has final U.S. FDA approval for its narcolepsy drug, Lumryz, the company has priced the sodium oxybate formulation at $64.67 per gram, matching the cost of competitor Xyrem/Xywav from Jazz Pharmaceuticals plc’s Xywav is taken twice nightly, while Lumryz is taken just once. The GABA B receptor agonist Xywav (sodium oxybate/potassium oxybate/calcium oxybate/magnesium oxybate) gained FDA approval in July 2020 for treating cataplexy and excessive daytime sleepiness for those with narcolepsy. Also strengthening its hand, Lumryz received orphan drug exclusivity from the FDA. Avadel is planning its commercial launch to be around the beginning of June. Avadel stock (NASDAQ:AVDL) had advanced a confident 14% at midday to about $13.35 per share.

Antiva targets CIN with topical tablet, raises $53M

A precancerous condition caused by the human papillomavirus (HPV) affects up to 300,000 American women who are diagnosed each year, and yet there are no treatments, just preventive vaccines introduced in 2006 – targeted to younger generations prior to the first sexual encounter. That leaves a large proportion of the female population stuck with a “wait-and-see” approach that involves continuous monitoring of their HPV infection through pap smears to detect cellular changes that could lead to cervical cancer. South San Francisco-based Antiva Biosciences Inc. is seeking to find a better response to this condition known as high-grade cervical intraepithelial neoplasia (CIN2,3) with its lead topical therapeutic ABI-2280, a prodrug of an acyclic nucleoside phosphonate that is currently in phase I trials.

Newco news: Series A on horizon for artificial interferon specialist ILC Therapeutics

With its sights set on a series A and an IPO following a £3.5 million (US$4.4 million) investment round in 2021, Scottish biotech ILC Therapeutics Ltd. is hoping to make waves with a sublingual interferon antiviral to treat COVID-19. The USP for the company’s lead, Alfacyte, is the fact that it’s an artificial version of interferon, so has less of a propensity to cause the flu-like symptoms that can come from treatment with natural kinds, which hike levels of cytokines and interleukins. As a hybrid interferon that is composed of interferon alpha-10 and interferon alpha-12, Alfacyte is “up to 10,000 times less likely” to cause adverse effects, according to ILC CEO Alan Walker.

HELP! Sanders faces mutiny amid bounty of support for US drug pricing bills

In its first markup of the 118th Congress, the Senate Health, Education, Labor and Pensions (HELP) Committee, under the new leadership of Sen. Bernie Sanders (I-Vt.), today devolved into a mutiny of sorts as the committee members started to take up four bipartisan bills aimed at taming prescription drug prices. The mutiny had more to do with the process than the bills themselves, a fact Ranking Member Bill Cassidy(R-La.) made clear in his opening comments when he called Sanders out for reneging on the compromises the two had worked out in scheduling the markup session. He also noted that he been given the technical assistance notes and Congressional Budget Office score for only one amendment being offered at the meeting, although the documents were available for the other three as well. “That’s not the way to run a committee. . . At some point, process matters,” he said. With other members, both Republican and Democrat, sharing Cassidy’s concerns, Sanders recessed the markup until further notice.

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