A cou­ple months ahead of its PDU­FA date, Bris­tol My­ers rolls out long-term da­ta for mava­camten

It’s al­most time for the FDA to make a de­ci­sion on mava­camten, the myosin in­hibitor at the cen­ter of Bris­tol My­ers Squibb’s $13 bil­lion MyoKar­dia buy­out. With a Jan. 28 PDU­FA date loom­ing, the phar­ma gi­ant is now tak­ing the wraps off some long-term da­ta it hopes will bol­ster the drug’s case for ap­proval.

Heart pa­tients who re­ceived mava­camten in a co­hort of the long-term MA­VA-LTE study saw sig­nif­i­cant re­duc­tions in me­di­an lev­els of a hor­mone called NT-proB­NP (58% at week 24 and 67% at week 48), Bris­tol My­ers re­port­ed at this year’s Amer­i­can Heart As­so­ci­a­tion con­fer­ence. High­er than nor­mal NT-proB­NP lev­els are in­dica­tive of heart fail­ure.

Bris­tol My­ers called this a “sus­tained re­duc­tion,” adding that im­prove­ments in left ven­tric­u­lar re­lax­ation and di­as­tolic func­tion were al­so seen al­though no sig­nif­i­cant changes were ob­served in left-ven­tric­u­lar stroke vol­ume.

The phar­ma gi­ant is shoot­ing for an ap­proval in symp­to­matic ob­struc­tive hy­per­trophic car­diomy­opa­thy, or oHCM: a con­di­tion in which the mus­cu­lar wall be­tween the two bot­tom cham­bers of the heart be­comes thick­er than nor­mal, block­ing blood flow out of the heart. In ad­di­tion to be­ing at an in­creased risk of heart fail­ure and stroke, pa­tients with HCM of­ten suf­fer fa­tigue and short­ness of breath that keep them from per­form­ing dai­ly ac­tiv­i­ties.

Mava­camten was “well tol­er­at­ed” in the long-term study, Bris­tol My­ers said, though nine pa­tients paused their treat­ment be­cause left ven­tric­u­lar ejec­tion frac­tions (a mea­sure­ment of how much blood is be­ing pumped out of the heart’s left ven­tri­cle) were too low. Eight of them re­sumed treat­ment at a low­er dose af­ter re­cov­ery, while one dis­con­tin­ued the tri­al per­ma­nent­ly, the com­pa­ny said.

BMS picked up mava­camten in its MyoKar­dia buy­out last Oc­to­ber and got its NDA ac­cept­ed back in March. The drug claims to work by de­creas­ing the ex­cess bridg­ing be­tween myosin and actin, there­by coun­ter­act­ing the hy­per­tro­phy and hy­per-con­trac­til­i­ty ex­pe­ri­enced by pa­tients with oHCM.

Roland Chen

In May, Bris­tol My­ers re­leased more Phase III da­ta at the vir­tu­al Amer­i­can Col­lege of Car­di­ol­o­gy meet­ing show­ing that pa­tients on mava­camten post­ed a clin­i­cal­ly sig­nif­i­cant in­crease over place­bo in scores on the Kansas City Car­diomy­opa­thy Ques­tion­naire, a catch-all sum­ma­ry of symp­toms and qual­i­ty of life mark­ers, at 30 weeks. Mava­camten boost­ed ob­struc­tive car­diomy­opa­thy pa­tients’ scores by more than 20 points over place­bo, where a five-point in­crease was con­sid­ered “small but clin­i­cal­ly sig­nif­i­cant,” Bris­tol My­ers said in a re­lease.

“We’re very ex­cit­ed,” Bris­tol My­ers’ se­nior VP and head of car­dio­vas­cu­lar de­vel­op­ment Roland Chen said of the da­ta be­ing pre­sent­ed at AHA. “They re­in­force our con­fi­dence of mava­camten as a po­ten­tial med­i­cine for pa­tients with hy­per­trophic car­diomy­opathies.”

If ap­proved in a cou­ple months, Bris­tol My­ers says it’s ready to be­gin rolling out the drug in 2022.

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.

David Baker working with a student on their protein design (Jason Mast)

Sci­en­tists are fi­nal­ly learn­ing how to de­sign pro­teins from scratch. Drug de­vel­op­ment may nev­er be the same

SEATTLE — It’s a cloudy Thursday afternoon in mid-July and David Baker is reclining into the futon in his corner office at the University of Washington, arms splayed out like a daytime talk show host as he coaches another one of his postdocs through the slings and arrows of scientific celebrity.

“Be jealous of your time,” he says, before plotting ways of sneaking her out of Zooms. “It’s this horrible cost to science that you’re tied up in some stupid meeting.”

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Amidst R&D reshuf­fle, Ver­tex ex­pands its pres­ence in Boston, aim­ing to be­come num­ber one

Vertex Pharmaceuticals has been one of the buzzier names in the bustling Boston biotech scene, but now the company is looking to vault to number one status — at least in terms of physical footprint.

At a ribbon cutting on Tuesday for its new Jeffrey Leiden Center for Cell and Genetic Therapies at the Boston Seaport, Vertex announced it would embark on a new project: The company will build a 344,000 square foot facility in the seaport to accommodate the company’s growing R&D needs, especially in its cell and gene therapies program.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,200+ biopharma pros reading Endpoints daily — and it's free.

Patty Murray, D-WA (Graeme Sloan/Sipa USA)(Sipa via AP Images)

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,200+ biopharma pros reading Endpoints daily — and it's free.

Warren Buffett, Berkshire Hathaway CEO

Berk­shire Hath­away pulls out of Ab­b­Vie, Bris­tol My­ers Squibb in­vest­ments

It looks like Warren Buffett is sticking to ice cream and railroads for the moment.

The billionaire CEO of Berkshire Hathaway backed out of two major holdings in the pharma industry, Forexlive first reported, including a $410 million investment in AbbVie and a $324.4 million stake in Bristol Myers Squibb.

The move comes after Berkshire abandoned its Teva shares just last quarter, Bloomberg reported.

Long-ex­pect­ed UK lay­offs im­mi­nent for No­var­tis fol­low­ing sale

Nearly a year ago, more than 200 workers at Novartis’ Grimsby, UK, facility were able to hang on to their jobs after the pharma closed a Switzerland site as a part of its workforce restructuring plan. Now, it looks like those employees’ time is up, as the site has been sold, Grimsby Telegraph reported today.

The manufacturing site has been sold to Humber Industrials, a subsidiary of International Process Plants. None of the current staff members will be working with the new owners, however.

Clay Siegall (Photo by Dimitrios Kambouris/Getty Images for Gabrielle's Angel Foundation)

UP­DAT­ED: Clay Sie­gall re­signs from Seagen amid in­ves­ti­ga­tion in­to do­mes­tic vi­o­lence claims

A week after Seagen revealed that longtime CEO Clay Siegall was on leave due to an allegation of domestic violence, he has resigned.

Since that shocking revelation, more details about the claims have emerged into the public eye. As Endpoints News reported, Siegall was arrested on April 23. A police report about that night and a subsequent temporary restraining order described a pattern of abusive behavior against his wife and a physical altercation that left her with multiple bruises. Siegall denied the claims.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,200+ biopharma pros reading Endpoints daily — and it's free.

FDA lob­bies Con­gress over rare dis­ease court rul­ing with wide im­pli­ca­tions

Usually reserved for making decisions on drug applications or enforcing what Congress stipulates, the FDA is now dipping its toe into the wild world of congressional politics as it attempts to fix a major court decision that could have a chilling effect on rare disease R&D.

The case in question from last October saw a US appeals court overturn a prior FDA court win, saying that the agency never should’ve approved a rare disease drug because a previously approved but more expensive drug with the same active ingredient has orphan drug exclusivity barring such an approval.