UP­DAT­ED: Bio­gen pulls PRIME des­ig­na­tion for ad­u­canum­ab in Eu­rope; Mer­ck clos­es merg­er with Ac­celeron

Fol­low­ing news last week that Bio­gen re­ceived “a neg­a­tive trend vote” in Eu­rope for its con­tro­ver­sial Alzheimer’s drug ad­u­canum­ab, the EMA up­dat­ed a list of PRIME des­ig­na­tions in­di­cat­ing that some, in­clud­ing Bio­gen, pulled out of the EU’s pri­or­i­ty med­i­cines scheme, known as PRIME.

But Bio­gen said in a state­ment to End­points News that it ac­tu­al­ly pulled the PRIME des­ig­na­tion for ad­u­canum­ab in 2019, not­ing that it oc­curred “fol­low­ing the dis­con­tin­u­a­tion of the EMERGE and EN­GAGE stud­ies,” and that it “was a pro­ce­dur­al re­quire­ment at the time.”

Ad­u­canum­ab was first grant­ed en­try in­to the PRIME pro­gram back in May 2016, EMA said in its run­ning list of PRIME des­ig­na­tions.

The neg­a­tive news for Bio­gen in Eu­rope fol­lows an ex­treme­ly slow start in sell­ing the in­fused drug in the US. But the Cam­bridge, MA-based biotech is still await­ing a ma­jor de­ci­sion from the Cen­ters for Medicare and Med­ic­aid Ser­vices on whether the US fed­er­al gov­ern­ment will pay for use of the drug. A draft of that de­ci­sion is ex­pect­ed on Jan. 12. — Zachary Bren­nan

Mer­ck com­pletes ac­qui­si­tion of Ac­celeron

Mer­ck’s high­ly pub­li­cized ac­qui­si­tion of Ac­celeron Phar­ma is now com­plete, Mer­ck an­nounced this morn­ing — mak­ing it the biggest biotech merg­er of the year.

The $11.5 bil­lion deal to ac­quire the biotech and its lead pul­monary ar­te­r­i­al hy­per­ten­sion drug so­tater­cept had been high­ly pub­li­cized — and con­firmed days of ru­mors and in­sid­er leaks when it was of­fi­cial­ly an­nounced at the end of Sep­tem­ber.

“This is an im­por­tant and strate­gic op­por­tu­ni­ty for our com­pa­ny to con­tin­ue grow­ing our car­dio­vas­cu­lar port­fo­lio and pipeline,” Mer­ck pres­i­dent and CEO Rob Davis said in a state­ment.

So­tater­cept, which had been in test­ing to im­prove out­comes in pa­tients with PAH, is cur­rent­ly in Phase III tri­als as an add-on to cur­rent stan­dard of care treat­ments. — Paul Schloess­er

Deci­bel’s R&D deal with Re­gen­eron is get­ting an ex­ten­sion

Deci­bel Ther­a­peu­tics’ re­search col­lab­o­ra­tion with Re­gen­eron is get­ting ex­tend­ed.

The col­lab­o­ra­tion, which start­ed in 2017 to dis­cov­er and de­vel­op gene ther­a­pies for hear­ing loss, will be ex­tend­ed to Nov. 15, 2023, and Re­gen­eron will pay $10 mil­lion as an ex­ten­sion fee in Q4 2022.

Un­der the col­lab­o­ra­tion, Deci­bel is de­vel­op­ing three gene ther­a­py pro­grams with Re­gen­eron — tar­get­ing con­gen­i­tal, mono­genic hear­ing loss. Deci­bel plans to ini­ti­ate a Phase I/II clin­i­cal tri­al of DB-OTO, its lead gene ther­a­py prod­uct can­di­date, next year. Deci­bel is al­so ad­vanc­ing two oth­er gene ther­a­py pro­grams tar­get­ing oth­er mono­genic forms of hear­ing loss as part of the col­lab — AAV.103 and AAV.104.

“We are pleased that Re­gen­eron has elect­ed to ex­tend the re­search term, which ex­tends our ac­cess to Re­gen­eron’s world-lead­ing ge­nom­ic and ge­net­ic tech­nolo­gies, and ther­a­peu­tic dis­cov­ery and de­vel­op­ment ex­per­tise,” said Deci­bel’s CEO Lau­rence Reid. — Paul Schloess­er

Ka­ma­da buys port­fo­lio from Saol Ther­a­peu­tics in deal with $95M up­front

Is­raeli biotech Ka­ma­da ac­quired a port­fo­lio of four FDA-ap­proved prod­ucts from Saol Ther­a­peu­tics for $95 mil­lion up­front.

The prod­ucts, which are “plas­ma-de­rived hy­per­im­mune com­mer­cial prod­ucts” ac­cord­ing to a com­pa­ny state­ment, have an ex­pect­ed com­bined an­nu­al glob­al rev­enue of be­tween $40 and $45 mil­lion, with ap­prox­i­mate­ly 95% of sales be­tween the US and Cana­da.

The four ac­quired prod­ucts in­clude:

  • Cy­togam (Cy­tomegalovirus Im­mune Glob­u­lin In­tra­venous [Hu­man]) for the pro­phy­lax­is of cy­tomegalovirus dis­ease as­so­ci­at­ed with the trans­plan­ta­tion of the kid­ney, lung, liv­er, pan­creas, and heart.
  • Win­rho SDF, which is in­di­cat­ed to in­crease platelet counts to pre­vent ex­ces­sive he­m­or­rhage in the treat­ment of non-splenec­tomies, for Rho(D)-pos­i­tive chil­dren with chron­ic or acute im­mune throm­bo­cy­tope­nia (ITP), adults with chron­ic ITP, and chil­dren and adults with ITP sec­ondary to HIV in­fec­tion.
  • He­pagam B, a he­pati­tis B im­mune glob­u­lin prod­uct in­di­cat­ed to both pre­vent he­pati­tis B virus re­cur­rence fol­low­ing liv­er trans­plan­ta­tion in he­pati­tis B sur­face anti­gen pos­i­tive (HB­sAg- pos­i­tive) pa­tients and pro­vide post-ex­po­sure pro­phy­lax­is.
  • Varizig, which con­tains an­ti­bod­ies spe­cif­ic to the vari­cel­la zoster virus, and is in­di­cat­ed for post-ex­po­sure pro­phy­lax­is of vari­cel­la (chick­en­pox) in high-risk pa­tient groups, in­clud­ing im­muno­com­pro­mised chil­dren, new­borns, and preg­nant women.

Ac­cord­ing to Ka­ma­da CEO Amir Lon­don, the com­pa­ny hopes to ex­pand fur­ther in­to the US next year. Along­side the $95 mil­lion up­front to Saol, Ka­ma­da will pay up to an ad­di­tion­al $50 mil­lion in sales mile­stones un­til 2034. — Paul Schloess­er

Ed­i­tor’s note: Ar­ti­cle cor­rect­ed to note that Bio­gen ac­tu­al­ly pulled its PRIME des­ig­na­tion from the EMA in 2019 when it dis­con­tin­ued the two stud­ies of its Alzheimer’s drug. Re­analy­ses of those halt­ed stud­ies helped ad­u­canum­ab win its ap­proval in the US.

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.

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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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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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.

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.

Aim­ing for fourth nod, Sarep­ta files an­oth­er DMD gene ther­a­py to FDA; Ax­some head­ed to­ward mi­graine re­sub­mis­sion

Sarepta Therapeutics has filed the data needed for an FDA accelerated approval, which would be the biotech’s fourth if granted by the agency.

The biotech has yet to complete confirmatory trials for those first three conditional nods. The filing for its fourth Duchenne muscular dystrophy treatment, disclosed Thursday, is not a surprise. Sarepta said in late-July it would do so after releasing positive results for the Roche-partnered gene therapy.

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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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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