Armed with promis­ing alope­cia PhII, Con­cert edges for­ward with mod­i­fied JAK in­hibitor — but safe­ty will still be key

Con­cert Phar­ma has scored a fresh round of pos­i­tive Phase II da­ta for its JAK in­hibitor amid an on­go­ing ri­val­ry with Pfiz­er, paving the way for a late-stage show­down in alope­cia area­ta. It’s al­so cau­tious­ly stak­ing a claim about safe­ty at a time the whole class is un­der a cloud of un­cer­tain­ty.

The glow­ing re­sults — in which both the 8 mg and 12 mg twice-dai­ly dos­es of CTP-543 hit the pri­ma­ry end­point of re­liev­ing hair loss — re­couped some en­thu­si­asm for the drug lost in its in­ter­im read­out. In­ves­ti­ga­tors re­port­ed that 58% of pa­tients in the 12 mg co­hort achieved at least 50% rel­a­tive re­duc­tion in the over­all Sever­i­ty of Alope­cia Tool (SALT) while 47% in the 8 mg group saw that im­prove­ment, com­pared to 9% for place­bo (p <0.001 in both cas­es).

Shares $CNCE climbed 2.48% to $10.32.

Con­cert is drop­ping the 4 mg dose from the up­com­ing Phase III study af­ter con­clud­ing its dif­fer­ence with place­bo is not sta­tis­ti­cal­ly sig­nif­i­cant.

The biotech is go­ing up against two Pfiz­er drugs for alope­cia area­ta, an au­toim­mune dis­or­der that can cause hair loss not just on the scalp but the beard, eye­brows, and oth­er ar­eas of the body for both men and women. CTP-543 in­hibits JAK1 and JAK2, while PF-06651600 tar­gets JAK3 and PF-06700841 blocks TYK2 and JAK1.

While JAK, or Janus ki­nase, are be­lieved to play an im­por­tant role in in­flam­ma­to­ry process­es via sig­nal­ing for over 50 cy­tokines and growth fac­tors, drugs in the class have re­cent­ly been im­pli­cat­ed for se­ri­ous side ef­fects. These con­cerns were in part ex­em­pli­fied by Pfiz­er’s own block­buster Xel­janz af­ter FDA and EMA reg­u­la­tors re­strict­ed its use due to a high­er risk of blood clots and death. And Ab­b­Vie’s FDA ap­proval for upadac­i­tinib (Rin­voq) days ago came with a black box warn­ing about in­fec­tions and throm­bo­sis.

In its press re­lease Con­cert high­light­ed that “no throm­boem­bol­ic events were re­port­ed dur­ing the tri­al,” and the on­ly se­ri­ous ad­verse event pos­si­bly re­lat­ed to treat­ment was a bac­te­r­i­al in­fec­tion on the face (fa­cial cel­luli­tis).

Oth­er side ef­fects in­clude headache, na­sopharyn­gi­tis, up­per res­pi­ra­to­ry tract in­fec­tion and ac­ne. The break­down:

James Cas­sel­la Con­cert

Sim­i­lar­ly, Pfiz­er has ob­served in­fec­tions, gas­troin­testi­nal and skin/sub­cu­ta­neous tis­sue is­sues in their Phase II alope­cia area­ta study.

“We are high­ly fo­cused on the need for an ef­fec­tive and safe treat­ment for alope­cia area­ta, and we plan to ad­vance CTP-543 in­to Phase 3 test­ing next year,” Con­cert chief de­vel­op­ment of­fi­cer James Cas­sel­la said in a state­ment.

But Con­cert will have more than one bat­tle to fight. Since CTP-543 is a deuter­at­ed ver­sion of Jakafi, In­cyte has filed — and won — a patent chal­lenge that it is now ap­peal­ing.

So­cial im­age: Alope­cia, Shut­ter­stock

 

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Biohaven CEO Vlad Coric (Photo Credit: Andrew Venditti)

Pssst: That big Bio­haven Alzheimer's study? It was a bust. Even the sub­group analy­sis ex­ecs tout­ed was a flop

You know it’s bad when a biopharma player plucks out a subgroup analysis for a positive take — even though it was way off the statistical mark for success, like everything else.

So it was for Biohaven $BHVN on MLK Monday, as the biotech reported on the holiday that their Phase II/III Alzheimer’s study for troriluzole flunked both co-primary endpoints as well as a key biomarker analysis.

The drug — a revised version of the ALS drug riluzole designed to regulate glutamate — did not “statistically differentiate” from placebo on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB).  The “hippocampal volume” assessment by MRI also failed to distinguish itself from placebo for all patients fitting the mild-to-moderate disease profile they had established for the study.

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Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

Dan Skovronsky, Eli Lilly CSO (Lilly via Facebook)

Eli Lil­ly tees up dis­cov­ery pact worth more than $1.6B with Merus for T cell-fo­cused bis­pe­cif­ic an­ti­bod­ies

Under science chief Dan Skovronsky, Eli Lilly has taken some big swings at next-gen therapies, including trying to find the next big thing in oncology. Now, after one early failure in the field, Lilly is going back to the bispecific antibody well with a new deal with a Dutch biotech.

Lilly will pay $40 million upfront with an additional $20 million equity stake in Merus NV to identify and develop three bispecific antibodies looking to engage the CD3 antigen on T cells and redirect immune cells, the Indianapolis pharma giant said Tuesday.

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Andrew Allen (Gritstone)

As coro­n­avirus vari­ants trig­ger new alarms, the NIH is putting an un­der-the-radar ‘next-gen’ vac­cine in­to PhI

Over the past year, the world has been transfixed by the development of new vaccines to fight SARS-CoV-2. In a frenzy of activity, the new mRNA approach has delivered pioneering emergency approvals in record time. And with some setbacks, the more traditional big players are coming along with added jabs as the most affluent nations in the world begin to vaccinate large portions of their populations.

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Janet Woodcock (AP Images)

Janet Wood­cock is in the run­ning for FDA com­mis­sion­er — what does that mean for the agen­cy's fu­ture?

Just a day after reports emerged that Janet Woodcock will serve as interim chief of the FDA, word has gotten out that she is also in the running for the permanent job.

The decision, as the initial wave of reactions suggest, could have dramatic implications for where the agency is headed in the next four years — if not beyond.

Woodcock, the longtime CDER director, is being vetted alongside former FDA principal deputy commissioner Joshua Sharfstein, Bloomberg reported. Already tapped as acting head of the agency, she’s set to take over from Stephen Hahn right after Biden’s inauguration next week.

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The IPO queue adds 5 more biotechs hop­ing to ring in 2021 by blitz­ing Nas­daq

Following a record year for IPOs — in terms of both proceeds and count — there’s already a long lineup of biotechs ready to jump onto Nasdaq in the new year. The companies are likely looking for much higher raises than they initially projected on their S-1s. Now it’s time to see if investors are still hungry for another round of biotech stocks.

Sana helped set the pace early on, as its founders look to divvy up a fortune from their IPO. And late last week 5 more biotechs crowded in, looking to pick up the pace where 2020 left off. Here they are:

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News brief­ing: Beam bags a $260M pri­vate place­ment; mi­Ra­gen re­brands to Virid­i­an Ther­a­peu­tics

Agios vet John Evans has demonstrated how to raise big money for a little biotech.

The Beam Therapeutics CEO — and ARCH partner — has pieced together a $260 million private placement from a group of backers that includes Perceptive Advisors, Farallon Capital, Casdin Capital, Redmile Group and Cormorant Asset Management. And there are 3 main goals they’ll pursue with it: clinical development, strategic partnerships and general corporate purposes.

As­traZeneca keeps the ball rolling on Dai­ichi-part­nered En­her­tu, pick­ing up 2nd in­di­ca­tion in gas­tric can­cer

AstraZeneca’s big gamble on Daiichi Sankyo’s antibody-drug conjugate Enhertu has already paid off with a big approval in breast cancer more than a year ago. But the partners have big plans for their blockbuster in the making, and a new nod in gastric cancer will raise their spirits even higher.

The FDA on Friday approved Enhertu to treat locally advanced or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma in patients who have previously undergone at least one round of treatment with a Herceptin-based regimen, AstraZeneca said in a release.