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.

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.

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.”