The top­i­cal treat­ment for alope­cia area­ta that Aclaris Ther­a­peu­tics li­censed from Rigel Phar­ma has failed a mid-stage study for alope­cia area­ta, leav­ing the com­pa­ny with a drug that seems to have caused no harm but no ben­e­fit ei­ther.

Neal Walk­er Aclaris

While ex­ecs vowed to find a pos­si­ble way for­ward with sub­group analy­sis, the re­sults didn’t leave much room for hope. Nei­ther the 0.46% or 0.12% con­cen­tra­tions of ATI-502 proved su­pe­ri­or to place­bo in im­prov­ing scalp hair cov­er­age — at least not in a sta­tis­ti­cal­ly sig­nif­i­cant way. In fact, an un­ex­pect­ed­ly ef­fec­tive ve­hi­cle out­per­formed the low­er dose on sev­er­al end­points, which were al­ter­na­tive mea­sure­ments of hair loss sever­i­ty.

“We are sur­prised and ex­treme­ly dis­ap­point­ed by the re­sults of this Phase 2 tri­al,” pres­i­dent and CEO Neal Walk­er said in a state­ment.

So were in­vestors. Aclaris’ stock $ACRS has tum­bled 32.58% since the an­nounce­ment Wednes­day at mar­ket close and now sits at $3.00.

It con­tin­ues a down­ward tra­jec­to­ry trig­gered by the FDA’s re­quest for Aclaris to pull a pro­mo­tion­al video on a dif­fer­ent treat­ment sev­er­al days ago, stat­ing that the ad “makes false or mis­lead­ing claims” re­gard­ing their hy­dro­gen per­ox­ide top­i­cal so­lu­tion, Es­ka­ta, for raised se­b­or­rhe­ic ker­atoses.

Aclaris is now pin­ning its hopes on the up­com­ing Phase II read­out of ATI-501, an oral JAK1/JAK3 in­hibitor that goes af­ter the same dis­ease. The ba­sic mech­a­nism re­mains the same — stop­ping T cells from launch­ing an at­tack on fol­li­cle cells — but the hope is it will work for more se­vere pa­tients.

Both ATI-501 and ATI-502 were sub­jects of a 2017 li­cense agree­ment, where Aclaris paid Rigel $8 mil­lion up­front for world­wide rights.

“From a hair loss per­spec­tive we still re­main fo­cused on the cat­e­go­ry broad­ly and look for­ward to the re­sults of the oral work lat­er this year,” a sub­dued Walk­er told an an­a­lyst on a con­fer­ence call.

While the biotech al­so counts a “soft top­i­cal” JAK in its ar­se­nal — ob­tained from a $20 mil­lion buy­out of St. Louis-based Con­flu­ence Life Sci­ences — Walk­er said they are more in­ter­est­ed in de­ploy­ing it for oth­er types of hair loss as well as atopic der­mati­tis.

Aclaris is al­so test­ing ATI-502 for vi­tili­go, an­dro­ge­net­ic alope­cia and atopic der­mati­tis while await­ing Phase III re­sults for A-101, a top­i­cal ther­a­py for com­mon warts.

So­cial im­age: Shut­ter­stock

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Roche’s Kadcyla has become the first antibody-drug conjugate to enter the Chinese market, marking a dramatic advance for both the Swiss pharma giant and the therapeutic class.

The local arm of Roche announced the approval late Tuesday, which covers the therapy’s use in the adjuvant setting in patients with early HER-2 positive breast cancer who still have residual invasive disease after receiving paclitaxel and Herceptin as neoadjuvant treatment.

Pascal Soriot has spent more than 20 years at the top of an industry recently found to emit more carbon than the automotive industry.

He called himself a “global citizen,” and traveled often across three-plus continents. While CEO of AstraZeneca, he commissioned a flight service — media-dubbed AstraZeneca airlines — from Cambridge to the company’s other European hub in Gothenburg. He made few, if any, public statements on the environment or his companies’ impact on it.

Every few years, a public health crisis (think Ebola, Zika) spurred by a rogue pathogen triggers a small-biotech rally, as drugmakers emerge from the woodwork with ambitious plans to treat the mounting outbreak. In most cases, that enthusiasm never quite delivers.

Things are no different, as the coronavirus outbreak in Wuhan, China takes hold. There have been close to 300 confirmed human infections in China, and at least four deaths. Coronaviruses are a large family of viruses, which include MERS and SARS. On Tuesday, the CDC reported the virus was detected in a US traveler returning from Wuhan.

With an FDA decision date looming, Roche on Wednesday unveiled positive pivotal data on its blockbuster-bound oral spinal muscular atrophy (SMA) drug in patients with the most severe form of the muscle-wasting disease.

The FDA is set to make its decision on the therapy, risdiplam, by May 24. It is expected to compete with Biogen’s Spinraza and Novartis’ Zolgensma.

Partnered with PTC Therapeutics, the Roche drug was tested in 41 patients aged 1-7 months with type 1 SMA, a rare genetic muscle-wasting disease. The trial, dubbed FIREFISH, measured efficacy via the proportion of infants sitting without support after 12 months of treatment, and longer.