David Meeker, Rhythm Pharmaceuticals CEO and chairman

Rhythm gets sec­ond OK for ge­net­ic obe­si­ty drug Im­civree, swings in­to roy­al­ty deal

An­oth­er OK, an­oth­er 2,000 po­ten­tial pa­tients.

On Thurs­day, Rhythm Phar­ma­ceu­ti­cals re­ceived its sec­ond FDA green light for its obe­si­ty man­age­ment drug for those with ul­tra-rare ge­net­ic dis­eases, this time for Bardet-Biedl syn­drome, a dis­ease that im­pacts a num­ber of or­gans but with ear­ly-on­set obe­si­ty as a ma­jor symp­tom. And the com­pa­ny al­so en­gi­neered a roy­al­ty deal net­ting it up to $100 mil­lion.

The FDA ap­proved the drug, known as set­melan­otide and mar­ket­ed as Im­civree, for pa­tients 6 years and up af­ter Phase III da­ta in 31 Bardet-Biedl pa­tients showed it de­creased BMI by around 8% com­pared to place­bo. Rhythm chair­man and CEO David Meek­er not­ed that while BMI was not a per­fect mea­sure­ment, the com­pa­ny opt­ed to use it over weight be­cause its study in­clud­ed pe­di­atric pa­tients who were still grow­ing.

“Part of the noise in the world of obe­si­ty is that [peo­ple say] ‘it’s an epi­dem­ic,’ and every­body thinks they un­der­stand it, and physi­cians think they un­der­stand it, and a lot of peo­ple think it’s a lifestyle choice,” Meek­er, for­mer CEO of Gen­zyme, told End­points News.

But while peo­ple are slow­ly un­der­stand­ing obe­si­ty is a dis­ease, Rhythm is show­ing that it’s not one dis­ease, but many dis­eases, he con­tin­ued, adding that re­searchers are find­ing some clear ge­net­ic dri­vers be­hind obe­si­ty.

In No­vem­ber 2020, Rhythm re­ceived its first ap­proval for Im­civree for pa­tients with dis­rup­tions to their POMC, PC­SK1 and LEPR genes. The Bardet-Biedl ap­proval is a bit dif­fer­ent, as pa­tients need a clin­i­cal di­ag­no­sis of the syn­drome, which has been linked to 23 dif­fer­ent ge­net­ic mu­ta­tions, in­stead of a gene test con­fir­ma­tion of a spe­cif­ic mu­ta­tion as they did with the first ap­proval.

On­ly one minute af­ter an­nounc­ing the Bardet-Biedl ap­proval, Rhythm dis­closed it was en­ter­ing a roy­al­ty deal with the apt­ly named Health­Care Roy­al­ty. As part of the deal, Rhythm will get an ini­tial $37.5 mil­lion, fol­lowed by an ad­di­tion­al $37.5 mil­lion pend­ing Eu­ro­pean ap­proval for Im­civree in Bardet-Biedl syn­drome and $25 mil­lion for cer­tain sales goals. In ex­change, Rhythm will pay Health­care Roy­al­ty tiered roy­al­ties be­gin­ning in the low dou­ble dig­its and falling to the low sin­gle dig­its af­ter cer­tain rev­enue points on Im­civree’s glob­al sales.

In 2021, Rhythm made a mod­est $3.2 mil­lion from Im­civree sales, though it will like­ly eclipse that num­ber in 2022, with $1.5 mil­lion in sales al­ready in the first quar­ter.

The mon­ey from Health­care Roy­al­ty will keep Rhythm go­ing un­til the sec­ond half of 2024, as it tries to great­ly broad­en Im­civree’s in­di­ca­tion in­to oth­er dis­eases that im­pact the hunger sig­nal path­way up­on which it acts. But the FDA doled out a CRL for Im­civree in Al­ström syn­drome, an­oth­er ul­tra-rare ge­net­ic dis­ease that caus­es vi­sion and hear­ing loss, di­a­betes and obe­si­ty, since those pa­tients did not reach the pri­ma­ry end­point for the study.

In to­tal with its cur­rent in­di­ca­tions, it can reach some 5,000 pa­tients in the US, Rhythm pre­vi­ous­ly es­ti­mat­ed. How­ev­er, with the stud­ies it has go­ing on now — in­clud­ing a Phase II tri­al in pa­tients who get treat­ed for a be­nign brain tu­mor and come out with ex­treme hunger, and a Phase III tri­al look­ing at 4 ad­di­tion­al genes — it could add up­wards of 50,000 pa­tients, Meek­er es­ti­mat­ed.

The drug does come with some side ef­fects though, and five pa­tients dis­con­tin­ued the tri­al. Most no­tably, many of the pa­tients ex­pe­ri­enced skin dark­en­ing since Im­civree al­so hits a path­way that caus­es melanin re­lease. “It’s like tan­ning when you go out in the sun … some peo­ple note it, some peo­ple don’t, and some peo­ple like it, and some peo­ple are both­ered by it,” Meek­er said, adding that a few of the pa­tients who dis­con­tin­ued did so due to their skin dark­en­ing.

Paul Hudson, Sanofi CEO (Cyril Marcilhacy/Bloomberg via Getty Images)

FDA side­lines Paul Hud­son's $3.7B MS drug af­ter es­tab­lish­ing link to liv­er dam­age

One of Sanofi CEO Paul Hudson’s top picks in the pipeline — picked up in a $3.7 billion buyout 2 years ago — has just been sidelined in the US by a safety issue.

The pharma giant put out word early Thursday that the FDA has put their Phase III studies of tolebrutinib in multiple sclerosis and myasthenia gravis on partial clinical hold, halting enrollment and suspending dosing for patients who have been on the drug for less than 60 days. Patients who have completed at least 60 days of treatment can continue therapy as researchers explore a “limited” — but unspecified in Sanofi’s statement — number of cases of liver injury.

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Phar­ma re­acts to post-Roe; Drug­mak­ers beef up cy­ber de­fense; Boehringer, Roche qui­et­ly axe drugs; 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.

As a reminder, we are off on Monday for the Fourth of July. I hope this recap will kick off your (long) weekend well and that the rest of it will be just what you need. See you next week for a shortened edition!

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Eric Hughes, incoming Teva EVP of global R&D and CMO

Te­va chief raids Ver­tex for his new glob­al head of re­search and de­vel­op­ment

Teva CEO Kåre Schultz has found his new R&D chief and CMO in Vertex’s ranks.

The global generics giant, which has some 3,500 staffers in the R&D group, has named Eric Hughes to the top research spot in the company. He’ll be replacing Hafrun Fridriksdottir, who held the role for close to five years, on Aug. 1.

Hughes hasn’t been at Vertex for long, though. He jumped from Novartis less than a year ago, after heading the immunology, hepatology & dermatology global development unit. Before that, he completed a five-year stint as head of early clinical research for the specialty discovery medicine department in the exploratory clinical & translational research group at Bristol Myers Squibb, according to his LinkedIn profile.

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#BIO22: Man­ag­ing a biotech in tur­bu­lent times. 'There's a per­fect shit­show out there'

On Tuesday, June 14, Endpoints News EIC John Carroll sat down with a group of biotech execs to discuss the bear market for industry stocks and how they were dealing with it. Here’s the conversation, which has been lightly edited for brevity.

Martin Meeson, sponsor opening:

Thank you, John. Hello everyone. My name’s Martin Meeson, I’m the CEO of Fujifilm Diosynth. For those of you who don’t know Fujifilm Diosynth, we operate in the development of clinical and commercial product scale up, we have facilities in Europe and the US, and around about 4,000 employees. We run on average about 150 programs, so when it comes to managing in turbulent times over the last two years, we’ve had quite a lot of experience of that. Not just keeping the clinical pipelines and the commercial pipelines open, but also our response to the pandemic and the molecules that we’ve had within there. One of the phrases that I coined probably about a year ago when we were talking at JP Morgan, was I talked about managing through turbulent times. Well, it’s become the fact that we are not managing and leading through these times, we are managing in them, which is why that’s really the purpose of and the topic that we’ve got today.

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Amgen's taking social media followers around the globe as it introduces the many different

From Tam­pa to Mu­nich, Am­gen’s ‘Places’ cam­paign in­tro­duces its lo­ca­tions around the world

Amgen is taking social media followers around the world with its latest corporate campaign. Called “Places of Amgen,” the twice monthly posts highlight the biopharma’s different offices and sites – and the people who work there.

Each post runs on LinkedIn, Facebook and Instagram with details about the work Amgen does in that location, when it was established, comments from people who work there and other interesting facts. The most recent one about Paris, France, for example, notes that Amgen France last year signed a French association charter committed to the inclusion of LBGT+ people in the workplace.

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Credit: Shutterstock

Bio­haven takes mi­graine cam­paign to pa­tients' Twit­ter feeds, months ahead of Pfiz­er takeover

Two weeks ago, Biohaven hit an all-time high in weekly Nurtec prescriptions. CEO Vlad Coric attributes at least some of that success to a new interactive Twitter campaign that encourages patients to free their feed of potential migraine triggers.

Earlier this month, Biohaven in partnership with Twitter launched the #RelieveYourFeed campaign that allows users to customize their app settings based on their migraine triggers.

Oncologists on Twitter are talking up ASCO studies, health equity and burnout, according to new deep dive research from Harris Poll (via Shutterstock)

What’s been on in­flu­en­tial on­col­o­gist­s' minds? Most­ly AS­CO, but al­so health eq­ui­ty, ac­cord­ing to new Har­ris Poll re­search

Over the past few months, oncologists have been talking a lot about the ASCO conference. Not surprising considering its import and the study data presented, but what may be less expected is the second most talked about topic of health equity.

That’s according to data from The Harris Poll and the new expert network the researcher is building out. While the company has been doing deep data dives into specific health issues or conditions for pharma clients, it’s now monitoring influencer networks of therapy area specialists to find out what experts are really thinking about.

Emer Cooke, ICRMA chair (AP Photo/Geert Vanden Wijngaert)

ICM­RA to launch sev­er­al reg­u­la­to­ry pi­lot pro­grams cen­tered around man­u­fac­tur­ing in­spec­tions

As regulatory agencies look to catch up on inspections amid the Covid-19 pandemic, ICMRA is unveiling several pilot programs to address industry applications and inspections.

ICMRA, which is made up of the world’s top drug regulators, is launching multiple pilot programs, including two regulatory pilots addressing facility inspections for chemistry and manufacturing controls (CMC) and post-approval change (PAC) submission assessments and related regulatory actions.

No­var­tis to re­sume the pro­duc­tion of two ra­di­oli­gand ther­a­pies af­ter re­solv­ing qual­i­ty is­sues

Earlier this year, Novartis touted its radioligand as a major piece to counter competition in the cancer space. However, the physical production of its products has had anything but a smooth ride.

In May, Novartis had to suspend production of Lutathera and Pluvicto, its two primary radiotherapies. According to the company, this was done out of an abundance of caution as a result of potential quality issues identified in its manufacturing. The production suspension impacts the commercial and clinical trial supply of the products.