David Meeker, Rhythm Pharmaceuticals CEO

Rhythm Phar­ma knocked off stride as mixed PhI­II obe­si­ty da­ta for its sole big drug look ‘meh’ to many

Just a few weeks af­ter Rhythm Phar­ma­ceu­ti­cals $RYTM snagged an FDA OK for set­melan­otide in 3 rare ge­net­ic dis­or­ders that trig­ger obe­si­ty, the biotech is strug­gling to make a good case for a cru­cial mar­ket ex­pan­sion.

Rhythm CEO David Meek­er spot­light­ed the pos­i­tive da­ta at hand for Bardet-Biedl syn­drome and Al­ström syn­drome, two oth­er ail­ments that trig­ger an in­sa­tiable hunger. But their 3 pa­tients with Al­ström syn­drome all failed the pri­ma­ry end­point in the study while the BBS ef­fi­ca­cy rate dwin­dled sig­nif­i­cant­ly from its Phase II re­sults, drag­ging down the over­all re­sults that left in­vestors and an­a­lysts won­der­ing just how big this suc­cess re­al­ly was.

Mur­ray Stew­art

Al­to­geth­er 34.5% of the 31 pa­tients in the tiny study hit the key end­point of at least a 10% drop in weight. The study al­so suc­ceed­ed on sec­ondary end­points.

Stifel’s Derek Archi­la has been track­ing Rhythm’s progress, and he had been ex­pect­ing the biotech to hit a high­er thresh­old on suc­cess. But do­ing some quick back-of-the-en­ve­lope math, he fig­ured about 40% of the BBS pa­tients hit the end­point, giv­ing the biotech a shot at a new mar­ket in­di­ca­tion worth about $390 mil­lion in peak sales.

“While at first blush these re­sults may ap­pear mediocre (our base case was a 40-50%) there are a few key fac­tors to con­sid­er,” he wrote Tues­day morn­ing, high­light­ing the AS flop as well as a pos­si­ble con­found­ing fac­tor in re­cruit­ing ado­les­cents for the study, which might have been weight­ed against suc­cess.

Phil Nadeau al­so high­light­ed the pos­i­tive.

While the BBS re­spon­der rate in the Ph III (39%) fell be­low that seen in the Ph II (67%), any im­prove­ment in weight com­bined with the dra­mat­ic re­duc­tions in hunger score demon­strat­ed in Phase III is mean­ing­ful for these pa­tients, who typ­i­cal­ly ex­pe­ri­ence on­go­ing weight gain over time as­so­ci­at­ed with de­bil­i­tat­ing hy­per­pha­gia

The “meh” over­all re­sults, though, ex­plain the 5% drop in share price this morn­ing. But Rhythm ex­ecs are push­ing ahead.

“(T)hese re­sults re­in­force the po­ten­tial val­ue of the MC4R path­way as a ther­a­peu­tic tar­get for some rare ge­net­ic dis­eases of obe­si­ty and un­der­score our be­lief that obe­si­ty is a com­plex, mul­ti­fac­to­r­i­al dis­ease,” said Mur­ray Stew­art, the CMO at Rhythm.

Bio­mark­er 'roadmap­s' and the fu­ture of can­cer R&D; Cur­tain rais­es on #AS­CO22; Pfiz­er, No­var­tis tack­le drug ac­cess; 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.

While this was not a week for earth-shattering news, there were certainly a lot of interesting tidbits. If you found this recap helpful, please recommend it to your friends and colleagues. We’ll see you on the other side of the long weekend.

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Keep­ing pres­sure on Am­gen, Mi­rati draws mixed re­views on lat­est cut of KRAS da­ta

As the close runner-up to Amgen’s Lumakras in the KRAS race, any data cut from Mirati’s adagrasib continues to draw scrutiny from analysts. And the latest batch of numbers from ASCO is a decidedly mixed bag.

While a quick comparison suggests that adagrasib spurred slightly more responses and led to a longer overall survival than Lumakras among a group of non-small cell lung cancer patients, its duration of response appears shorter and the safety profile continues to spark concern.

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Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

In search of elu­sive NASH break­through, Pfiz­er spot­lights com­bo ap­proach

Pfizer’s second crack at steering a NASH candidate through a battered field seems to be going better than the first.

The pharma giant has scored the FDA’s fast track designation for an experimental combination therapy as a treatment for NASH with liver fibrosis. The combo consists of ervogastat, a diacylglycerol O-acyltransferase 2 inhibitor (DGAT2i), and clesacostat, an acetyl-CoA carboxylase inhibitor (ACCi).

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Ann is one of ViiV Healthcare's newest spokespeople as the retired school administrator speaks up about her HIV status.

GSK's Vi­iV de­buts next evo­lu­tion in HIV med Dova­to cam­paign with new spokes­peo­ple and new mes­sage

When Ann saw the first TV commercials for HIV medicine Dovato, she didn’t see herself represented. So the 74-year-old retired school administrator who’s been living with HIV since 1998, reached out to GSK’s ViiV Healthcare and asked why not?

Now Ann is one of three people starring in ViiV’s latest Dovato campaign called “Detect This.” The next-step evolution in the branded campaign plays on the word “detect” — often used in describing HIV status under control as undetectable — but in this case, uses the word as a directive for people to understand they can use fewer medicines.

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Switzer­land to de­stroy over 600,000 ex­pired dos­es of Mod­er­na Covid vac­cine

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

AS­CO ab­stracts mint some ear­ly win­ners, but Io­vance, Spring­Works get hit by mas­sive loss­es

Before cancer-focused biotechs start their trek to the first in-person American Society of Clinical Oncology (ASCO) annual conference since the pandemic began, investors have taken a good look at the teasers for the data scheduled to be presented — and started placing bets.

With its power to confer overnight fame, ASCO is a stage where impressive or surprise debut performances can go a long way. On the other hand, disappointing details could be punishing.

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Nassim Usman, Catalyst Biosciences CEO

Af­ter $60M Ver­tex deal, group of Cat­a­lyst share­hold­ers claims biotech could’ve sold as­sets three years ago

Catalyst Biosciences was down to five employees in March, and the biotech needed to do something after two rounds of layoffs, a nixed collaboration and a culling of its hemophilia program.

In came Vertex, with $60 million to buy up the South San Francisco biotech’s preclinical complement drugs, which target the system that bridges the body’s innate and adaptive immune response and a class most known for Ultomiris and Soliris. The deal includes CB 2782-PEG, the dry AMD drug that Biogen no longer wanted in March.

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Levi Garraway, Roche CMO (Genentech)

Roche's CD20xCD3 does­n't beat Gen­mab at ORR, but sets bar for CR da­ta on lym­phoma drug

On its way to potentially becoming the first to market with a CD20xCD3 bispecific for aggressive lymphoma, Big Pharma’s largest R&D spender has some more data to pad its case with an FDA filing slated for later this year.

Roche dropped some more details from a Phase II expansion study of its fixed-duration glofitamab, to be presented at next week’s ASCO annual meeting, in patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL). The patients had received a median of three prior therapies.

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