Le­vo Ther­a­peu­tics miss­es pri­ma­ry end­point in PhI­II tri­al of Prad­er-Willi drug — the lat­est set­back in a dis­as­ter-prone field

Mark­ing yet an­oth­er set­back in the Prad­er-Willi Syn­drome field, Le­vo Ther­a­peu­tics failed to hit its pri­ma­ry end­point in a Phase III study of in­tranasal car­be­tocin. But the biotech is now shift­ing its fo­cus to the sec­ondary end­points in an ef­fort to pluck vic­to­ry out of the jaws of de­feat.

The dis­or­der, char­ac­ter­ized by a false sense of star­va­tion, is caused by the ab­sence or dele­tion of a fa­ther’s chro­mo­some 15. Illi­nois-based Le­vo’s po­ten­tial ther­a­py in­volves a se­lec­tive oxy­tocin-re­cep­tor ag­o­nist.

Two dos­es of Le­vo’s LV-101 were test­ed against a place­bo across eight weeks. Topline re­sults show that the 9.6 mg dose missed the mark on pri­ma­ry mea­sure­ments, in­clud­ing im­prove­ment to hy­per­pha­gia and ob­ses­sive com­pul­sive be­hav­ior. But “sta­tis­ti­cal sig­nif­i­cance” was achieved with the sec­ondary dose of 3.2 mg, ac­cord­ing to the com­pa­ny.

“Con­sis­ten­cy in ben­e­fit/re­sponse was ob­served in the 3.2 mg dose arm across oth­er key sec­ondary end­points, in­clud­ing clin­i­cal glob­al im­pres­sion of change (CGI-C; p=0.027) and anx­i­ety and dis­tress be­hav­iors, as eval­u­at­ed by the PWS Anx­i­ety and Dis­tress Be­hav­iors Ques­tion­naire (PADQ; p=0.027). Nei­ther dose demon­strat­ed a sta­tis­ti­cal­ly sig­nif­i­cant ef­fect on the Chil­dren’s Yale-Brown Ob­ses­sive Com­pul­sive Scale (CY-BOCS),” the com­pa­ny an­nounced.

PWS has proved dif­fi­cult to treat.

Mil­len­do Ther­a­peu­tics’ lead drug liv­o­le­tide failed a piv­otal tri­al for the treat­ment of PWS in April, send­ing shares plum­met­ing. The drug didn’t im­prove hy­per­pha­gia or oth­er eat­ing-re­lat­ed be­hav­iors.

Zaf­gen’s ZGN-1258, its sec­ond at­tempt to treat rare cas­es of obe­si­ty brought on by PWS, hit a brick wall af­ter the FDA put a hold on the com­pa­ny’s PWS pro­gram in 2018, cit­ing CV safe­ty con­cerns. Zaf­gen was lat­er used as a shell for Lari­mar to re­verse its way to Wall Street.

Le­vo ex­ecs, though, say they like where they are at this point.

“This is a long-await­ed step to­wards ad­dress­ing the sub­stan­tial needs of in­di­vid­u­als liv­ing with PWS,” Le­vo CEO said in a pre­pared state­ment. “We are ex­cit­ed by these im­por­tant re­sults that were achieved af­ter decades of in­ter­est in ad­dress­ing the oxy­tocin de­fi­cien­cy in PWS. We are al­so pleased that our ef­forts to de­vel­op new tools for clin­i­cal eval­u­a­tion of this rare, neu­rode­vel­op­men­tal dis­or­der have en­hanced our un­der­stand­ing of the re­al-world im­pact LV-101 has on anx­i­ety and dis­tress be­hav­iors.”

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Zai Lab hauls in $761M from Hong Kong IPO to push Ze­ju­la, more bud­ding can­di­dates in Chi­na — re­port

Zai Lab is set to net more than $761 million from its secondary listing in Hong Kong after pricing the IPO at $72.51 (HKD$562) — just a hair below its Nasdaq closing price on Monday, Bloomberg and Nikkei Asian Review reported.

A pioneer in bringing Western drugs to China, co-founder and CEO Samantha Du has more than tripled Zai Lab’s market cap in the three years it’s been public in the US. The HKEX listing is designed to fund R&D and commercialization for the current portfolio while fueling new in-licensing pacts, the biotech wrote in a filing.

Tae Han Kim, Samsung Biologics CEO (SeongJoon Cho/Bloomberg via Getty Images)

Sam­sung Bi­o­log­ics nets $330M+ deal from As­traZeneca ahead of 'Su­per Plan­t' con­struc­tion

Just a few weeks after announcing plans to construct a $2 billion “Super Plant,” Samsung Biologics is keeping its foot on the gas.

The Korean CDMO has inked a $330.8 million manufacturing contract with AstraZeneca, the companies announced Monday evening, providing the British drugmaker the ability to expand production capabilities in the Asia market. Per terms of the deal, the partnership could be increased to $545.6 million.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

Francesco De Rubertis

Medicx­i's David Grainger and Francesco De Ru­ber­tis pump €200M in­to six com­pa­nies and what they say is a first-of-its kind fund

In what they’re billing as a first for biotech, David Grainger, Francesco De Rubertis and their team at Medicxi have put down a €200 million to sweep up stakes in six companies from their predecessor VC and pump new money into them.

Medicxi didn’t disclose which companies it was investing in but the portfolio draws from Index Ventures Life VI, one of the last funds the Medicxi team launched while they were still part of the multinational, tech-focused VC firm Index Ventures. That team kept advising Index on their life sciences portfolio even after they spun out to form their own firm in the middle of 2016.

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Scripps reach­es $10M set­tle­ment with gov­ern­ment over al­le­ga­tions NIH grants weren't prop­er­ly ac­count­ed for

Scripps Research Institute has settled a case with the Justice Department alleging claims of misappropriated funds, the US attorney for the district of Maryland announced late last week.

Prosecutors said the institute improperly used NIH-funded research grants for non-grant related activities, including working on new grant applications, teaching activities and other administrative tasks. As part of the settlement, Scripps has agreed to pay $10 million.

Eli Lilly CSO Dan Skovronsky (file photo)

UP­DAT­ED: #ES­MO20: Eli Lil­ly shows off the da­ta for its Verzenio suc­cess. Was it worth $18 bil­lion?

The press release alone, devoid of any number except for the size of the trial, added nearly $20 billion to Eli Lilly’s market cap back in June. Now investors and oncologists will get to see if the data live up to the hype.

On Sunday at ESMO, Eli Lilly announced the full results for its Phase III MonarchE trial of Verzenio, showing that across over 5,000 women who had had HR+, HER2- breast cancer, the drug reduced the odds of recurrence by 25%. That meant 7.8% of the patients on the drug arm saw their cancers return within 2 years, compared with 11.3% on the placebo arm.

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President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists. HHS con­tin­ues to claim Azar “will de­fer com­plete­ly to the FDA"

President Donald Trump, who seems intent on announcing a Covid-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.