Warts for the win: Aclar­is' lead drug clears piv­otal study

Aclaris Ther­a­peu­tics has found a way to get rid of the warts and all.

The com­pa­ny — which ear­li­er this month de­cid­ed to fo­cus on its ar­se­nal of ki­nase in­hibitors — on Mon­day un­veiled pos­i­tive da­ta from a piv­otal study test­ing its lead ex­per­i­men­tal drug for use in com­mon warts.

The drug, A-101, was test­ed in a 502-pa­tient study called THWART-2 — pa­tients en­rolled had one to six warts be­fore qual­i­fy­ing for the tri­al. Pa­tients ei­ther self-ad­min­is­tered A-101 top­i­cal so­lu­tion or a ve­hi­cle twice a week over a two-month pe­ri­od. A high­er pro­por­tion of pa­tients on the drug (a po­tent hy­dro­gen per­ox­ide top­i­cal so­lu­tion) saw their warts dis­ap­pear at day 60, ver­sus the ve­hi­cle (p<0.0001) — meet­ing the main goal of the study.  Each sec­ondary end­point al­so emerged in fa­vor of A-101, the com­pa­ny said.

Shares of the Wayne, Penn­syl­va­nia-based com­pa­ny $ACRS soared about 65.5% to $1.82 in pre­mar­ket trad­ing on Fri­day.

An­oth­er late-stage tri­al in com­mon warts is on­go­ing, and da­ta are ex­pect­ed in the fourth quar­ter.

“Giv­en no FDA ap­proved treat­ments for com­mon warts, which im­pacts 22M Amer­i­cans each year, it seems that there would be a rel­a­tive­ly ‘low bar’ for A-101 45% to re­ceive ap­proval, as­sum­ing the con­fir­ma­to­ry Ph3 THWART-1 da­ta al­so reads out pos­i­tive­ly,” Jef­feries an­a­lysts wrote in a note.

“With on­ly topline in­for­ma­tion, it is dif­fi­cult to com­pare to the pri­or A-101 Ph2 datasets as well as VR­CA’s re­cent­ly re­port­ed Ph2 da­ta for VP-102 in treat­ing com­mon warts.”

Ver­ri­ca Phar­ma­ceu­ti­cals $VR­CA ear­li­er this year dis­closed pos­i­tive da­ta from an open-la­bel mid-stage study test­ing its drug, VP-102, in pa­tients with up to six warts. Da­ta skewed in fa­vor of VP-102 on the pri­ma­ry end­point of com­plete clear­ance of all treat­able warts at day 84, as well as the sec­ondary goal of the per­cent­age re­duc­tion of warts. The top­i­cal ther­a­py com­pris­es a so­lu­tion of 0.7% can­tharidin — a blis­ter-in­duc­ing agent se­cret­ed by cer­tain bee­tles — which was his­tor­i­cal­ly used as an aphro­disi­ac. Top­i­cal can­tharidin has been used on warts and mol­lus­cum since the 1950s, al­though the chem­i­cal lost FDA ap­proval in 1962 af­ter man­u­fac­tur­ers failed to fur­nish ad­e­quate ev­i­dence sup­port­ing its ef­fi­ca­cy.

Weeks ago, Aclaris con­clud­ed a strate­gic re­view of its busi­ness — re­solv­ing to fo­cus its re­sources on its im­muno-in­flam­ma­to­ry pipeline (en­com­pass­ing ATI-450, ATI-1777 and oth­er pre­clin­i­cal drug can­di­dates). It is now look­ing for part­ners on its com­mer­cial busi­ness: Rosacea treat­ment Rhophade and se­b­or­rhe­ic ker­atoses ther­a­py Es­ka­ta. In ad­di­tion, the com­pa­ny is al­so seek­ing part­ners for a tri­fec­ta of ther­a­pies in de­vel­op­ment — ex­per­i­men­tal warts drug A-101, as well as the in­ves­ti­ga­tion­al alope­cia treat­ments ATI-501 (oral) and ATI-502 (top­i­cal).

As a re­sult, the com­pa­ny an­nounced 86 lay­offs. The re­struc­tur­ing is ex­pect­ed to keep the lights on un­til the third quar­ter of 2021.

“While we hold the mgt team in high re­gard as thought lead­ers in der­ma­tol­ogy, the com­pa­ny has ex­e­cut­ed poor­ly over the past year (e.g., botched Es­ka­ta launch, mixed clin­i­cal da­ta for the JAK alope­cia pro­grams)…” Jef­feries an­a­lysts said in a note dat­ed Sep­tem­ber 6.

In June, the com­pa­ny suf­fered a set­back af­ter a mid-stage study test­ing ATI-502 failed to im­prove scalp hair cov­er­age, which crushed its stock. Aclaris paid Rigel Phar­ma $8 mil­lion up­front in 2017 for glob­al li­cens­es to both ATI-501 and ATI-502.

The dis­clo­sure came days af­ter the FDA rep­ri­mand­ed the com­pa­ny for mak­ing “false or mis­lead­ing claims” in a pro­mo­tion­al video for Es­ka­ta.

“That said, we see val­ue in the cur­rent cap­i­tal­iza­tion. Look­ing ahead, with a sub $40M mar­ket val­ue (trad­ing at ~0.45x net cash), ACRS shares could re­bound if mgt can ex­e­cute on its re­vamped strat­e­gy,” the an­a­lysts added.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA chief Stephen Hahn on Capitol Hill earlier this week (Getty Images)

As FDA buck­les un­der the strain of a pan­dem­ic work­load, Trump again ac­cus­es the agency of a po­lit­i­cal hit job

Peter Marks appeared before a virtual SVB Leerink audience yesterday and said that his staff at FDA’s CBER is on the verge of working around the clock. Manufacturing inspections, policy work and sponsor communications have all been pushed down the to-do list so that they can be responsive to Covid-related interactions. And the agency’s objective right now? “To save as many lives as we can,” Marks said, likening the mortality on the current outbreak as equivalent to “a nuclear bomb on a small city.”

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Play-by-play of Gilead­'s $21B Im­munomedics buy­out de­tails a fren­zied push — and mints a new biotech bil­lion­aire

Immunomedics had not really been looking for a buyout when the year began. Excited by its BLA for Trodelvy, submitted to the FDA in late 2019, executive chairman Behzad Aghazadeh started off looking for potential licensing deals and zeroed in on four potential partners, including Gilead, following January’s JP Morgan Healthcare Conference in San Francisco. Such talks advanced throughout the year, with discussions advancing to the second round in mid-August.

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The win­dow is wide open as four more biotechs join the go-go IPO class of 2020

It’s another day of hauling cash in the biopharma world as four more IPOs priced Friday and a fifth filed its initial paperwork.

The biggest offering comes from PMV Pharma, an oncology biotech focusing on p53 mutations, which raised $211.8 million after pricing shares at $18 apiece. Prelude Therapeutics, developing PRMT5 inhibitors for rare cancers, was next with a $158 million raise, pricing shares at $19 each. Graybug Vision raised $90 million after pricing at $16 per share for its wet AMD candidates, and breast cancer biotech Greenwich Lifesciences brought up the rear with a small, $7 million raise after pricing shares at $5.75.

J&J of­fers PhI/IIa da­ta show­ing its sin­gle-dose vac­cine can stir up suf­fi­cient im­mune re­sponse

Days after J&J dosed the first participants of its Phase III ENSEMBLE trial, the pharma giant has detailed the early-stage data that gave them confidence in a single-dose regimen.

Testing two dose levels either as a single dose or in a two-dose schedule spaced by 56 days in, the scientists from Janssen, the J&J subsidiary developing its vaccine, reported that the low dose induced a similar immune response as the high dose. The interim Phase I/IIa results were posted in a preprint on medRxiv.

President Donald Trump reacts after signing an executive order following his remarks on his healthcare policies yesterday in Charlotte, North Carolina (Getty Images)

Op-ed: Will phar­ma re­al­ly pay for Trump’s lat­est law­less promise to 33 mil­lion Medicare ben­e­fi­cia­ries? Not like­ly

Sitting atop the executive branch, President Donald Trump is the ultimate authority at the FDA. He can fast track any vaccine to approval himself. If it came to that, of course.

What he can’t do is unilaterally order the legislative branch to loosen the Treasury’s coffers for $6.6 billion. Nor can he command pharmaceutical companies to pay for $200 vouchers sent to 33 million Medicare beneficiaries for prescription drugs before the election.

Pal­la­dio bags $20 mil­lion Se­ries B to top­ple a prob­lem­at­ic kid­ney dis­ease drug

Palladio Biosciences just took one step further in its quest to topple Otsuka’s Samsca with its own — it hopes safer — autosomal dominant polycystic kidney disease (ADPKD) drug.

The Pennsylvania-based biotech announced a $20 million Series B on Friday, which will fund a 10-person Phase III trial of its vasopressin V2 receptor agonist, lixivaptan. CEO Alex Martin expects to read out data in the first half of next year, then launch straight into a larger pivotal Phase III study with about 1,200 participants.

New York governor Andrew Cuomo (AP Images)

An­drew Cuo­mo says New York will un­der­take its own vac­cine re­view process, and wouldn’t rec­om­mend trust­ing the fed­er­al gov­ern­ment

The concerns keep mounting over President Donald Trump’s politicization of the FDA and other federal agencies guiding the development of a safe and effective vaccine. And today, the telegenic New York governor Andrew Cuomo appeared to introduce even more politics into the matter — latest in an ongoing series of incidents that have cast the proudly independent FDA in starkly political terms.

During his daily press conference Cuomo said that the state will review any coronavirus vaccines approved by the federal government, citing a lack of trust in the Trump administration. The announcement comes one day after Trump accused the FDA of making an “extremely political” move in proposing stricter vaccine guidance.

Laura Shawver (Silverback Therapeutics)

Fol­low­ing a hefty Se­ries B, Sil­ver­back Ther­a­peu­tics quick­ly pulls in $85M for 'an im­por­tant growth phase'

Months after reeling in a $78 million Series B round, Silverback Therapeutics has hooked an even larger Series C.

The Seattle-based company announced Wednesday that it netted $85 million from a slate of new and previous investors. The quick boost could be a sign that an IPO is on the way.

In an email, Silverback CEO Laura Shawver told me she was “not able to provide any additional comments about Silverback” beyond what was shared in the company’s news release. In the prepared statement, she said the company is at “an important growth phase.”