Roche shares surge as a dou­ble-shot win in I/O and he­mo­phil­ia rais­es block­buster ex­pec­ta­tions

Roche has scored a win with one of the key im­muno/on­col­o­gy tri­als of the year, post­ing sig­nif­i­cant top-line gains for pro­gres­sion-free sur­vival and risk of death for a seg­ment of lung can­cer pa­tients get­ting first-line treat­ment with a triple com­bo of its check­point ther­a­py Tecen­triq, Avastin and chemo. And it swept a dou­ble head­er ear­ly this morn­ing with their re­port of an­oth­er suc­cess­ful late-stage study for its new­ly ap­proved he­mo­phil­ia A drug Hem­li­bra, fur­ther set­ting the stage for a painful show­down with mar­ket leader Shire.

The back-to-back suc­cess­es drove up Roche’s shares $RHH­BY by more than 6% this morn­ing, adding $12 bil­lion in mar­ket cap as in­vestors ral­lied to ev­i­dence that the phar­ma gi­ant could suc­cess­ful­ly put the patents to a trio of fran­chise drugs in the rear view mir­ror while join­ing Mer­ck in the front ranks of lung can­cer con­tenders. Mer­ck shares dipped 2%.

First, Tecen­triq.

San­dra Horn­ing

Some an­a­lysts had al­ready raised doubts that Roche could field a triple with Avastin and chemo in the all-im­por­tant mar­ket for first-line treat­ment of peo­ple with ad­vanced non-squa­mous non-small cell lung can­cer. Avastin could look too tox­ic and Mer­ck’s Keytru­da/chemo com­bo looked like the right ap­proach for now.

But IM­pow­er 150 suc­ceed­ed where As­traZeneca’s com­bi­na­tion of Imfinzi (dur­val­um­ab) and the CT­LA 4 im­munother­a­py treme­li­mum­ab had flunked out — at least in the first shot at PFS. Roche’s top-line re­port notes that it still has to cap­ture a ma­ture snap­shot of over­all sur­vival da­ta in the first half of next year.

In the mean­time the lead­ers in I/O, in­clud­ing Mer­ck $MRK, now face a new ri­val in one of the biggest can­cer mar­kets they’re scrap­ping over in a fren­zied race for mar­ket su­prema­cy. That will like­ly have se­ri­ous con­se­quences for Mer­ck, which worked hard to wres­tle the lead from Bris­tol-My­ers Squibb $BMY — an­oth­er play­er still in con­tention. And As­traZeneca $AZN has made lung can­cer a top pri­or­i­ty for its own I/O fran­chise.

The win by Roche al­so bol­sters the ar­gu­ment in fa­vor of adding chemo to the drug reg­i­men, with an on­go­ing de­bate over the ad­van­tages and dis­ad­van­tages of in­clud­ing chemo or an­oth­er im­munother­a­py.

“Many ex­perts in the field will be re­lieved be­cause there has been un­cer­tain­ty … I think this will re­al­ly en­cour­age many of us to use this com­bi­na­tion up­front,” on­col­o­gist Ste­fan Zim­mer­mann told Reuters.

Roche chief med­ical of­fi­cer San­dra Horn­ing says that the com­pa­ny is now test­ing the triple in a range of can­cer types. And the full da­ta set on the triple will ar­rive at a con­fer­ence in De­cem­ber.

Now Hem­li­bra.

John­ny Mahlangu

Roche just won an ac­cel­er­at­ed ap­proval of this drug for he­mo­phil­ia A on Fri­day, with da­ta from HAVEN 1 and 2. In HAVEN 3, Roche re­peat­ed its ear­li­er suc­cess and added a beat, demon­strat­ing su­pe­ri­or­i­ty to fac­tor VI­II pro­phy­lax­is. And it did it with­out trig­ger­ing any new cas­es of throm­bot­ic events that dogged its ear­li­er tri­als, forc­ing reg­u­la­tors to add a black box warn­ing on the la­bel.

“It is well es­tab­lished that pro­phy­lax­is is the pre­ferred ap­proach for treat­ment of he­mo­phil­ia A, but this can re­quire fre­quent in­tra­venous in­fu­sions, and some pa­tients on pro­phy­lax­is can still ex­pe­ri­ence bleeds, while oth­ers pre­fer on-de­mand treat­ment,” said John­ny Mahlangu, who’s on the fac­ul­ty of health sci­ences at the Uni­ver­si­ty of the Wit­wa­ter­srand and NHLS in Jo­han­nes­burg, South Africa. “Giv­en its po­ten­tial to be dosed through sub­cu­ta­neous in­jec­tion on­ly once week­ly or every oth­er week, Hem­li­bra may pro­vide a fur­ther ef­fec­tive pro­phy­lac­tic treat­ment op­tion for more peo­ple with he­mo­phil­ia A and help al­le­vi­ate some of the ad­min­is­tra­tion bur­den as­so­ci­at­ed with cur­rent treat­ment.”

The suc­cess­es weighed on he­mo­phil­ia play­er Shire, which is fac­ing a more pow­er­ful con­tender for its mar­ket share in he­mo­phil­ia, and As­traZeneca. Both ri­vals saw their shares slip against the Roche spike this morn­ing.

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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