Lit­tle Aldeyra's shares rock­et high­er as its first PhI­II eye drug tri­al scores, clear­ing path to NDA

Aldeyra Ther­a­peu­tics’ first for­ay in­to Phase III is a suc­cess, the eye drug de­vel­op­er said, set­ting it up for a po­ten­tial NDA fil­ing by the end of the year.

The top-line re­sults in al­ler­gic con­junc­tivi­tis fol­low a sim­i­lar­ly up­beat an­nounce­ment last Sep­tem­ber that re­prox­alap made the cut in a Phase IIb for dry eye dis­ease — a con­nec­tion that Aldeyra made sure to high­light.

Todd Brady

“Giv­en that ap­prox­i­mate­ly half of al­ler­gic con­junc­tivi­tis pa­tients al­so suf­fer from oc­u­lar dry­ness, we be­lieve the AL­LE­VI­ATE re­sults high­light the rel­e­vance of re­prox­alap to our cur­rent Phase 3 pro­gram in dry eye dis­ease,” pres­i­dent and CEO Todd Brady said.

In­vestors seem to agree, ral­ly­ing around its stock $ALDX, which is up 52%.

Two con­cen­tra­tions of the re­prox­alap so­lu­tion — 0.25% and 0.5% — were test­ed in the 318-pa­tient study along­side place­bo, and both of them hit the pri­ma­ry end­point: “oc­u­lar itch score (0 to 4 scale) area un­der the curve from 10 to 60 min­utes af­ter al­ler­gen chal­lenge.” The sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tions came with p < 0.0001 and p = 00025, re­spec­tive­ly.

The com­pa­ny al­so high­light­ed a sta­tis­ti­cal­ly sig­nif­i­cant dif­fer­ence in two-point re­spon­der rates on the same itch score — a mea­sure meant to con­firm the clin­i­cal rel­e­vance of the drug.

David Clark

While Aldeyra didn’t re­port on con­junc­ti­val red­ness — a sec­ondary out­comes mea­sure ac­cord­ing to clin­i­cal­tri­als.gov — it stat­ed that mild and tran­sient in­stil­la­tion site ir­ri­ta­tion was the on­ly ad­verse events ob­served.

“There is a large al­ler­gic con­junc­tivi­tis pa­tient pop­u­la­tion that is un­der­served by cur­rent­ly avail­able med­ica­tions,” CMO David Clark said. “The AL­LE­VI­ATE re­sults sug­gest the po­ten­tial of top­i­cal oc­u­lar re­prox­alap as a nov­el, safe, and ef­fec­tive ther­a­peu­tic op­tion that could be used to com­ple­ment ex­ist­ing ther­a­py be­fore re­sort­ing to cor­ti­cos­teroids, which can lead to se­ri­ous oc­u­lar tox­i­c­i­ty.”

Re­prox­alap works by di­min­ish­ing alde­hyde lev­els, which can con­tribute to in­flam­ma­tion.

The goal now is to tie up all loose strings around a dif­fer­ent clin­i­cal method that in­volves en­vi­ron­men­tal ex­po­sure — rather than an al­ler­gen chal­lenge cham­ber like this cur­rent study — and push ahead with new stud­ies that might bol­ster its case in front of the FDA when Aldeyra meets with reg­u­la­tors in the sec­ond half of this year.

Aldeyra has se­cured a $60 mil­lion loan from Her­cules Cap­i­tal to fund all that — with a late-stage op­er­a­tion now go­ing at full steam fol­low­ing a re­cent $10 mil­lion buy­out of He­lios Vi­sion and its Phase III-ready drug.

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

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As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.