Positive PhIII results in hand, Concert Pharma forges ahead with NDA and commercialization plans for hair loss drug – Endpoints News

Con­cert Phar­ma­ceu­ti­cals has been wait­ing to tout Phase III da­ta amidst a sell-off of the bulk of its pipeline to Ter­ran Bio­sciences and sev­er­al oth­er events for the biotech. And on Mon­day, the com­pa­ny rolled out the red car­pet.
Con­cert un­veiled topline re­sults from its sec­ond Phase III clin­i­cal tri­al, dubbed THRIVE-AA2, eval­u­at­ing its oral can­di­date cur­rent­ly called CTP-543 in adult pa­tients with mod­er­ate to se­vere alope­cia area­ta. The can­di­date is an in­hibitor of Janus ki­nas­es JAK1 and JAK2.
The pri­ma­ry ef­fi­ca­cy end­point, the per­cent­age of pa­tients achiev­ing a cer­tain score on an alope­cia sever­i­ty scale af­ter 24 weeks, was met with sta­tis­ti­cal sig­nif­i­cance in both the 8 mg twice-dai­ly and 12 mg twice-dai­ly dose groups rel­a­tive to place­bo. Treat­ment of the can­di­date was al­so gen­er­al­ly well-tol­er­at­ed.
Ac­cord­ing to the com­pa­ny, those en­rolled were re­quired to have at least 50 per­cent scalp hair loss as mea­sured by the SALT scale (with a score of 100 rep­re­sent­ing com­plete scalp hair loss and ze­ro as no loss). The av­er­age base­line SALT score across all pa­tients was ap­prox­i­mate­ly 87.9.
Sta­tis­ti­cal­ly sig­nif­i­cant pro­por­tions of pa­tients treat­ed with ei­ther dose lev­els achieved a SALT score of 20 or less. Con­cert said 38.3% hit the score in the 12 mg twice-dai­ly dose group and 33% in the 8 mg twice-dai­ly dose group. The study ul­ti­mate­ly net­ted a p-val­ue of p<0.0001 for both dos­es.
The pos­i­tive da­ta were wel­come news to in­vestors, as the Mass­a­chu­setts biotech’s stock price $CNCE rose over 6% since open­ing.
“Based on the re­sults from our Phase 3 pro­gram, we be­lieve CTP-543, if ap­proved, could be a best-in-class treat­ment op­tion for alope­cia area­ta and have a dis­tinct ther­a­peu­tic pro­file with fea­tures that are im­por­tant to pa­tients, in­clud­ing the per­cent­age of pa­tients with clin­i­cal­ly-mean­ing­ful hair re­growth, pa­tient sat­is­fac­tion lev­els, and hair re­growth rates,” said Con­cert’s chief de­vel­op­ment of­fi­cer James Cas­sel­la.
On the back of the re­sults, Cas­sel­la said the com­pa­ny is now look­ing to pre­pare an NDA sub­mis­sion to the FDA in the first half of next year as well as plans to com­mer­cial­ize the drug.
Con­cert’s path to the FDA comes as Eli Lil­ly was the first across the line in June, as Olu­mi­ant (baric­i­tinib) net­ted FDA ap­proval in a once-dai­ly pill in three dose lev­els, putting pres­sure on the biotech now to get ap­proval for its can­di­date.
Sec­ondary end­points in­clud­ed 47% of pa­tients in the 8 mg twice-dai­ly group and 52% of pa­tients in the 12 mg twice-dai­ly group say­ing they were “sat­is­fied” or “very sat­is­fied” with the treat­ment, com­pared to 2% of pa­tients in the place­bo group.
The can­di­date and the com­pa­ny it­self have been on a wild ride. In 2018, the drug’s da­ta ini­tial­ly did not hold up against Pfiz­er’s can­di­date, but ex­ecs de­cid­ed to forge ahead and got break­through ther­a­py des­ig­na­tion in 2020. CTP-543 is one of the com­pa­ny’s ma­jor hopes as the com­pa­ny hit a sig­nif­i­cant speed bump last year when a Phase II tri­al for its schiz­o­phre­nia pro­gram flopped, caus­ing the com­pa­ny to ax the pro­gram and its stock to tank by 40%.
Amgen CEO Bob Bradway is bellying up to the M&A table today, scooping up the newly anointed commercial biotech ChemoCentryx $CCXI and its recently approved rare disease drug for $3.7 billion out of the cash stockpile. The deal comes in at $52 a share — a hefty increase over the $24.11 close yesterday.
Bradway and the Amgen team get a drug called Tavneos (avacopan) in the deal, a complement factor C5a inhibitor OK’d to treat anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis, an autoimmune disease which can be lethal.
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The field of gene therapy has been diligently moving forward over the past several decades to bring potentially life-saving treatments to patients with genetic diseases. In addition to two approved adeno-associated viral (AAV) gene therapies, there are more than 250 AAV gene therapies in various clinical trial stages.1 AAV vectors remain the most frequently used vector for delivering therapeutic transgenes to target tissues due to their demonstrated and lasting clinical efficacy and extensive safety track record. As AAV therapies advance through clinical trials and into commercialization, many biotech companies are turning to contract development and manufacturing organizations (CDMOs) to prepare their programs for late-stage clinical and commercial scale manufacturing. Given the scope and scale of the manufacturing needs that will accompany regulatory approvals for these assets, CDMOs continue to expand their capacity to meet the needs of increasing prevalent patient populations. However, despite rapid growth, projected gene therapy manufacturing demands still outpace the collective capacity of the CDMO industry.
Alnylam $ALNY has laid claim to a major success in Phase III, with its RNAi drug patisiran hitting the primary endpoint in its APOLLO-B study for ATTR amyloidosis with cardiomyopathy, one of the most important pivotal trials to read out this year.
Shares of the big biotech soared 46% ahead of the bell, after starting the day with a market cap of $17 billion.
This is a topline readout only, with no details on the precise data comparison for the primary endpoint of change from baseline in the 6-Minute Walk Test at 12 months compared to the placebo arm. That’s still some weeks away, reserved for a conference. But the p-value hit 0.0162, while a key secondary on the quality of life also weighed in on the positive side of the stat boundary at 0.0397. And that sets the stage for a quick march to the FDA in search of a 2023 market launch.
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While Pfizer’s $11.4 billion acquisition of Array BioPharma in the summer of 2019 was mainly focused on oncology, namely Braftovi and Mektovi, there were a few non-cancer assets, including a Phase III drug being tested in a rare cardiovascular disease.
The late-stage trial is now being axed, alongside any further development of the oral small molecule, the pharma giant disclosed after the closing bell on Wednesday. Based on an interim futility analysis of the global Phase III REALM-DCM trial, Pfizer determined a path forward was not in its best interest. Pfizer no longer expected the study would meet its primary goal.
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When you’ve got an alliance as hefty as the one Arcus and Gilead have, every update counts.
That’s why the companies found analysts frowning on the latest disclosure around quemliclustat, their small molecule inhibitor of CD73. Although an interim analysis from a triplet combo trial in pancreatic cancer looks “promising,” it didn’t seem that much different from chemotherapy alone — triggering a decision to wait for “more mature” progression-free survival and overall survival data before making a call.
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Two of the most outspoken — and successful — drug developers in biotech say they’ve collected early-stage clinical data that are pointing them down the trail to the holy grail in cancer immunotherapy R&D.
While analysts largely busied themselves today with chronicling the ongoing success of Regeneron’s two big cash cows — Dupixent and Eylea — chief scientist George Yancopoulos and CEO Len Schleifer used the Q2 call to spotlight their early success with a combination of the “homegrown” PSMAxCD28 costimulatory bispecific antibody REGN5678 in combination with their PD-1 checkpoint Libtayo. The presentation comes just weeks after Regeneron completed a deal to gather all rights to the PD-1 that had been in Sanofi’s hands. And the two top execs are unstinting in their praise of the potential of a whole set of costimulatory pipeline projects which they say may finally deliver the long-awaited next-level approach to broadening the immunotherapy field of drugs.
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As the old saying goes, ‘If at first you don’t succeed, try, try again.’ The origin of this mantra is disputed but has been widely attributed to Robert the Bruce, the first king of Scotland, as he and his army did battle with invading English forces in the early 14th century.
But what happens if you try a second time and again don’t succeed?
Travere Therapeutics will be asking itself that question after a second attempt to gun for accelerated approval was rejected by the FDA, the company announced Wednesday afternoon. The former Martin Shkreli biotech had attempted to push through its lead drug, sparsentan, to a speedy OK in a rare kidney disease, but regulators prefer the more methodical, full approval route.
With the rising number of monkeypox cases, leading researchers at the CDC, FDA and NIH are calling on a randomized clinical trial to see if an approved smallpox drug is effective at treating monkeypox.
No monkeypox treatments are approved in the US, so patients looking to get relief for their lesions and other symptoms from the virus must go through a set of hurdles to get the smallpox drug through a government expanded access program. Approved for smallpox in 2018, the drug is marketed as TPOXX by the biotech SIGA. The European Union approved it for monkeypox in addition to smallpox earlier this year and the UK followed suit in July.
One of Horizon Therapeutics’ top sellers isn’t performing as well as expected, CEO Tim Walbert revealed on Wednesday’s Q2 call, leading the pharma company to switch up its strategy.
While Walbert noted a 13% increase in sales last quarter, totaling $876.4 million, the chief executive lowered his full-year expectations for the thyroid eye disease (TED) drug Tepezza. While the drug was initially expected to see a percentage growth in the mid-30s, Walbert now anticipates a sales growth in the high-teens.
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