Mer­ck touts PhI­II da­ta bur­nish­ing $500M plat­form — what kind of bar does it set for a chron­ic cough drug?

Roy Baynes sur­prised some an­a­lysts dur­ing Mer­ck’s in­vestor day last June — its first in five years — when he shone a sur­pris­ing spot­light on gefapix­ant. The P2X3 in­hibitor, he said, has the po­ten­tial to be “a pipeline in a prod­uct,” start­ing with a pro­gram in chron­ic cough.

Topline re­sults from the Phase III tri­als ap­pear to be bear­ing out his en­thu­si­asm. The read­out is al­so set­ting a bar that some see biotech ri­val Bel­lus Health clear­ing eas­i­ly.

When giv­en twice dai­ly at the 45 mg dose, gefapix­ant met the pri­ma­ry end­point, where pa­tients saw a sig­nif­i­cant de­crease in cough fre­quen­cy over a 24-hour pe­ri­od ver­sus place­bo. That was true in both COUGH-1 (12 weeks) and COUGH-12 (24 weeks). The 15 mg reg­i­men, though, failed to achieve the same ef­fect.

That’s all Mer­ck is di­vulging un­til an up­com­ing med­ical meet­ing. In a brief state­ment, Baynes high­light­ed that the con­di­tion is of­ten re­frac­to­ry and un­ex­plained.

For Baird an­a­lyst Bri­an Sko­r­ney, the news rep­re­sents the “best-case sce­nario” for Bel­lus, which al­so works with the P2X3 mech­a­nism but whose pitch cen­ters on a more se­lec­tive ap­proach that avoids taste-al­ter­ing side ef­fects that have marred Mer­ck’s re­sults in the past.

“We be­lieve the ab­sence of [taste] da­ta from Mer­ck’s press re­lease sug­gests they are con­tin­u­ing to see sig­nif­i­cant taste al­ter­ations in their clin­i­cal tri­als,” he wrote in a note.

Taste al­ter­ations, or dys­geu­sia, oc­curred in rough­ly half of the pa­tients who took gefapix­ant in Phase II, he point­ed out, com­pared to less than 4% in the place­bo arms. Mean­while, on­ly a sin­gle pa­tient out of 40 who took Bel­lus’ BLU-5937 in Phase II re­port­ed dys­geu­sia.

More da­ta from Bel­lus’ Phase II tri­al are ex­pect­ed in mid-2020. The Cana­di­an biotech’s shares on the Nas­daq trad­ed up 8.49% to $6.26.

But Mara Gold­stein of Mizuho likes Mer­ck’s chances. The side ef­fects were tran­sient, lead­ing to on­ly one pa­tient dis­con­tin­u­a­tion in the ear­ly tri­als fea­tur­ing a 50 mg dose.

“If ap­proved, this would be the first in­di­ca­tion for a drug in this set­ting, pro­vid­ing MRK with a po­ten­tial first-to-mar­ket ad­van­tage in spite of the like­ly loss/al­ter­ation of taste,” she wrote.

The phar­ma gi­ant al­so has much more re­sources to push gefapix­ant way be­yond chron­ic cough. Af­ter buy­ing Af­fer­ent Phar­ma and its P2X3 plat­form for $500 mil­lion up­front in 2016, it’s plot­ted a whole range of pro­grams in en­dome­tri­al-re­lat­ed pain, sleep ap­nea and oth­er sen­so­ry re­lat­ed func­tions.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Try­ing to shake up the Parkin­son's par­a­digm, Ab­b­Vie sub­mits NDA for con­tin­u­ous, 24-hour in­fu­sion ther­a­py

AbbVie is approaching the FDA with a new therapy to potentially treat Parkinson’s disease, using prodrugs of two medications commonly used for the condition.

The Big Pharma submitted its NDA for ABBV-951, a solution of levodopa and carbidopa prodrugs being evaluated in advanced Parkinson’s patients who don’t respond well to oral therapy, AbbVie announced Friday morning. Researchers are hoping a positive Phase III study that reads out in late October will help move things along quickly at the agency.

Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Fu­ji­film con­tin­ues its biotech build­ing spree with new fa­cil­i­ty in Chi­na

A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand.

Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development.

According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services. Cell culture media itself often requires customization of formulas and protocols to achieve the desired quantity and quality of therapeutic desired. Fujifilm Irvine Scientific is offering these services from its headquarters in California and Japan to its customers globally, as well as in China now.

Emer Cooke, EMA director (AP Photo/Geert Vanden Wijngaert)

Ahead of FDA, EMA rec­om­mends au­tho­riz­ing new gene ther­a­py treat­ment for ul­tra-rare dis­ease

Aromatic amino acid decarboxylase (AADC) deficiency is an ultra-rare genetic disease that leaves patients unable to produce certain hormones in the brain, such as dopamine and serotonin, usually leading to developmental delays, weak muscle tone and inability to control the movement of the limbs. It can also lead to multiple organ failure.

To date, there have been no treatments approved for AADC deficiency, which has been identified in less than 150 patients.

Ather­sys tries to post-hoc-an­a­lyze its way out of an­oth­er tri­al fail for stroke stem cell ther­a­py

Athersys’ stem cell therapy has failed yet again.

In a 206-person trial conducted in Japan, Athersys’ stem cell therapy for stroke failed its primary endpoint of “excellent outcome,” a combined measure of three stroke recovery scores.

While a greater percentage of patients in the treatment group reached the primary endpoint compared to placebo, that difference was not statistically significant.

Siddhartha Mukherjee (Brian Ach/Getty Images for The New Yorker)

All Blue's $733M bid to ac­quire Zymeworks turns hos­tile as board bat­tles back — af­ter a biotech celebri­ty jumps in

Yesterday, the team at All Blue Capital — bent on the takeover of a badly battered Zymeworks — brought in celebrated oncologist, Pulitzer prize-winning writer and biotech exec Siddhartha Mukherjee to add some glitz to their proposed board. But they’re still not winning over any converts.

This morning, Zymeworks’ board officially turned this acquisition offer into a hostile showdown, rejecting the unsolicited offer and marshaling its forces to prevent a buyout at $10.50 per share.

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