Ad­verum Bio of­fers a late no­ti­fi­ca­tion about FDA hold on lead gene ther­a­py for wet AMD

The FDA has some ques­tions for Ad­verum Biotech­nolo­gies about the CMC work re­lat­ed to their lead gene ther­a­py for wet, age-re­lat­ed mac­u­lar de­gen­er­a­tion, and reg­u­la­tors have put a clin­i­cal hold on their clin­i­cal de­vel­op­ment pro­gram un­til they get some an­swers.

The Men­lo Park, CA-based biotech $AD­VM re­port­ed Mon­day as the mar­ket opened that the agency had placed a hold on their drug “in ear­ly April” and were now re­view­ing a re­sponse to their query, sub­mit­ted last week. This was the first chance that in­vestors got to hear about this news from the pub­lic com­pa­ny, which re­served word about the FDA ac­tion un­til af­ter they not­ed they had a green light from the in­de­pen­dent da­ta mon­i­tor­ing com­mit­tee for the re­cruit­ment of their sec­ond co­hort of pa­tients for AD­VM-022 — which they say was the chrono­log­i­cal or­der of events.

This is their on­ly clin­i­cal-stage pro­gram. But the news seems to be sit­ting well with its share­hold­ers. The stock is up 7% in mid-af­ter­noon trad­ing.

I asked in a fol­low-up query to the com­pa­ny why they didn’t file any­thing with the SEC on the clin­i­cal hold when it oc­curred, or with­in a few days of the event. I haven’t heard back.

Leone Pat­ter­son

The treat­ment in­volves an afliber­cept cod­ing se­quence which is de­liv­ered to the eye via a com­pa­ny-owned vec­tor. Afliber­cept is a drug de­liv­ered to the eye via in­jec­tion, mak­ing any re­duc­tion in treat­ments some­thing most pa­tients would wel­come.

Ed­ward Nash at Sun­Trust says he’s been in touch with the ex­ec­u­tive crew at the biotech and not­ed that un­der the best case sce­nario the pro­gram would be de­layed a month. That, ob­vi­ous­ly, could stretch out if the FDA takes more time to suss things out.

Ad­verum was formed out of Avalanche Biotech­nolo­gies’ ac­qui­si­tion of Paris-based An­na­pur­na and their merg­er in­to a new gene ther­a­py com­pa­ny fol­low­ing some trou­ble at Avalanche. Late last year the biotech aban­doned their then lead, AD­VM-043, af­ter re­searchers de­ter­mined it was hav­ing an in­suf­fi­cient ef­fect on A1AT de­fi­cien­cy.

“We are work­ing with the FDA to re­solve this mat­ter as quick­ly as pos­si­ble,” said Ad­verum CEO Leone Pat­ter­son in a state­ment. “We are deeply com­mit­ted to the de­vel­op­ment of our nov­el gene ther­a­py AD­VM-022 for pa­tients with wet AMD. In the OP­TIC tri­al, the DMC re­viewed the safe­ty da­ta and unan­i­mous­ly agreed that we could pro­ceed to dos­ing the sec­ond co­hort. No pa­tient has ex­pe­ri­enced an SAE, with a fol­low-up pe­ri­od of up to five months. We look for­ward to shar­ing 24-week pri­ma­ry and sec­ondary out­comes from the first co­hort of pa­tients at a sci­en­tif­ic meet­ing in the sec­ond half of this year.”


Im­age: Shut­ter­stock

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

CEO Pascal Soriot via Getty Images

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

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.