Roger Tung, Concert Pharmaceuticals CEO (Concert)

Con­cert gets BTD for alope­cia drug, set­ting up a late-stage show­down with gi­ant ri­val Pfiz­er

Con­cert Phar­ma­ceu­ti­cals’ path to de­vel­op­ing a drug that treats alope­cia area­ta has been bumpy, but the phar­ma com­pa­ny scored a win Wednes­day.

The FDA grant­ed Con­cert a Break­through Ther­a­py Des­ig­na­tion (BTD) for its oral Janus ki­nase in­hibitor, named CTP-543, paving the way for a Phase III study of the drug to be­gin in the fourth quar­ter of 2020. The news fol­lows pos­i­tive Phase II re­sults from last Sep­tem­ber, which saw the drug meet its pri­ma­ry end­point in both 8 mg and 12 mg twice-dai­ly dos­es.

The news spurred a 14% spike in the biotech’s share $CNCE price.

There are cur­rent­ly no drugs on the mar­ket to treat alope­cia area­ta and Con­cert, whose drug blocks JAK1 and JAK2, has been com­pet­ing with Pfiz­er to see who can reach shelves first. CTP-543 is go­ing up against PF-06651600, which in­hibits JAK3, and PF-06700841, which tar­gets TYK2 and JAK1.

Though the FDA has of­fi­cial­ly en­dorsed CTP-543’s po­ten­tial with the BTD, the drug’s suc­cess is far from guar­an­teed.

JAK in­hibitors, which can al­so be used to treat rheuma­toid arthri­tis and ul­cer­a­tive col­i­tis, have come un­der heavy scruti­ny in re­cent years. Both the FDA and EMA have slapped warn­ings on and re­strict­ed larg­er dos­es of such in­hibitors made by Pfiz­er (Xel­janz) and Ab­b­Vie (Rin­voq) af­ter ob­serv­ing high­er rates of blood clots and death. Pfiz­er end­ed up drop­ping its high­er-dose treat­ment of Xel­janz al­to­geth­er, and Eli Lil­ly avoid­ed sell­ing high dos­es of its Olu­mi­ant drug as it be­came avail­able.

Con­cert it­self ran in­to some trou­ble a few years ago when the FDA placed a clin­i­cal hold on the Phase IIa study for CTP-543, even though the agency did not men­tion safe­ty con­cerns in its no­tice. Af­ter quick­ly fix­ing the is­sue, the Phase IIa study was com­plet­ed in late 2018, but showed Con­cert’s in­hibitors, de­spite hit­ting their main goals, did not stim­u­late hair growth as quick­ly as Pfiz­er’s in da­ta re­leased in late 2018. The fi­nal da­ta, re­leased the next year, showed the drug to be more com­pet­i­tive and sug­gest­ed it might even out­per­form Pfiz­er.

Pfiz­er is al­so re­search­ing JAK in­hibitors in teens and young adults, test­ing their ef­fi­ca­cy in treat­ments for atopic der­mati­tis. Al­though these stud­ies hit their pri­ma­ry goals, they too re­port­ed high­er rates of side ef­fects than the place­bo.

Alope­cia area­ta is an au­toim­mune dis­ease that af­fects around 700,000 peo­ple in the Unit­ed States. The dis­ease tricks the im­mune sys­tem in­to at­tack­ing a body’s own hair fol­li­cles, re­sult­ing in par­tial or com­plete loss of hair. Though the scalp is the most com­mon­ly af­fect­ed area, hair loss can oc­cur on any part of the body.

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.

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.

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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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 to be act­ing FDA com­mis­sion­er while Biden team fi­nal­izes nom­i­nee — re­ports

Janet Woodcock is set to be the most powerful person at the FDA in less than a week.

The veteran regulator and longtime director of the Center for Drug Evaluation and Research has been tapped as acting commissioner of the FDA, according to reports by BioCentury’s Steve Usdin and Pink Sheet’s Sarah Karlin-Smith.

The appointment was requested by the incoming Biden team, Karlin-Smith added, as they sort out the nomination of a permanent successor to Stephen Hahn — whose one-year tenure has been defined by Covid-19.

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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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