Alk­er­mes claims a suc­cess in PhI­II schiz­o­phre­nia head-to-head, but where’s the ben­e­fit for pa­tients?

Richard Pops

Alk­er­mes $ALKS has one shot at mak­ing ALKS-3831 a stand­out in the schiz­o­phre­nia field. For years now, in­ves­ti­ga­tors ex­pect­ed to be able to show that the drug per­forms pret­ty much the same as the gener­ic olan­za­p­ine in terms of ef­fi­ca­cy, but sig­nif­i­cant­ly bet­ter in terms of pre­vent­ing the weight gain — and all the risks that come with it — that af­flicts pa­tients on the old drug.

But in the first of two Phase III read­outs on Thurs­day evening, in­ves­ti­ga­tors were on­ly able to point to a sim­i­lar ef­fi­ca­cy with olan­za­p­ine with­out the ben­e­fit on weight gain.

El­liot Ehrich

Alk­er­mes CEO Richard Pops and R&D chief El­liot Ehrich were stick­ing with the top line and her­ald­ed the da­ta as a suc­cess. But in a wrap on side ef­fects the com­pa­ny al­so not­ed that weight gain was sim­i­lar for both drugs.

The stock dropped 5% as the da­ta hit Thurs­day evening, carv­ing out more than a bil­lion dol­lars in mar­ket cap.

“Those rates were sim­i­lar,” not­ed Ehrich dur­ing the Q&A with an­a­lysts Thurs­day evening.

The two ex­ecs, though, in­sist­ed that the 4-week study was sim­ply too short to ex­pect a sep­a­ra­tion on weight gain. That key ques­tion, they say, will have to wait for a 6-month study where pa­tients will have a chance to bal­ance the scale in fa­vor of the Alk­er­mes drug.

Based on its ear­li­er work, says Pops, it clear­ly takes time – three to six months — for the weight gain ad­van­tage to demon­strate it­self.

“Many physi­cians rec­og­nize the pow­er­ful ef­fi­ca­cy pro­file of olan­za­p­ine, but are hes­i­tant to pre­scribe it giv­en the se­vere weight gain and meta­bol­ic side ef­fects com­mon­ly as­so­ci­at­ed with its use,” said Christoph Cor­rell, M.D., pro­fes­sor of Psy­chi­a­try and Mol­e­c­u­lar Med­i­cine at Hof­s­tra North­well School of Med­i­cine. “A new an­tipsy­chot­ic with ro­bust ef­fi­ca­cy and a fa­vor­able weight and meta­bol­ic pro­file com­pared to olan­za­p­ine would be a wel­come ad­di­tion to the schiz­o­phre­nia treat­ment land­scape. This study con­firms a key el­e­ment of this pro­file, with a clear demon­stra­tion of ef­fi­ca­cy in a large, well-con­duct­ed clin­i­cal tri­al.”

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

BREAK­ING: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

Paul Hudson, Sanofi CEO (Photographer: Cyril Marcilhacy/Bloomberg via Getty Images)

Sanofi, Re­gen­eron’s Dupix­ent scores an­oth­er in­di­ca­tion with first-ever ap­proval for nodu­lar skin dis­or­der

Sanofi chief executive Paul Hudson told investors earlier this year that the Big Pharma was going to emphasize its sales kingpin Dupixent moving forward.

He wasn’t joking — the megablockbuster drug and sales king, recording just shy of $2 billion in sales this past quarter, has now officially secured its fifth indication from the FDA.

Sanofi and Regeneron, who jointly work on Dupixent development and commercialization, announced the new development on Thursday, saying that the FDA gave the all-clear to Dupixent to treat patients with prurigo nodularis, a rare autoimmune disorder characterized by a persistent, severe itch — and also visualized by hard, extremely itchy bumps known as nodules that form on the skin. The FDA noted in its announcement that it is the agency’s first approval for the disease.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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