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

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data is messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data is exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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Leen Kawas (L) has resigned as CEO of Athira and will be replaced by COO Mark Litton

Ex­clu­sive: Athi­ra CEO Leen Kawas re­signs af­ter in­ves­ti­ga­tion finds she ma­nip­u­lat­ed da­ta

Leen Kawas, CEO and founder of the Alzheimer’s upstart Athira Pharma, has resigned after an internal investigation found she altered images in her doctoral thesis and four other papers that were foundational to establishing the company.

Mark Litton, the company’s COO since June 2019 and a longtime biotech executive, has been named full-time CEO. Kawas, meanwhile, will no longer have ties to the company except for owning a few hundred thousand shares.

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Sen. Richard Durbin (D-IL, foreground) and Sen. Richard Blumenthal (D-CT) (Patrick Semansky/AP Images)

Sen­a­tors back FDA's plan to re­quire manda­to­ry pre­scriber ed­u­ca­tion for opi­oids

Three Senate Democrats are backing an FDA plan to require mandatory prescriber education for opioids as overdose deaths have risen sharply over the past decade, with almost 97,000 American opioid-related overdose deaths in the past year alone.

While acknowledging a decline in overall opioid analgesic dispensing in recent years, the FDA said it’s reconsidering the need for mandatory prescriber training through a REMS given the current situation with overdoses, and is seeking input on the aspects of the opioid crisis that mandatory training could potentially mitigate.

Bris­tol My­ers pledges to sell its Ac­celeron shares as ac­tivist in­vestors cir­cle Mer­ck­'s $11.5B buy­out — re­port

Just as Avoro Capital’s campaign to derail Merck’s proposed $11.5 billion buyout of Acceleron gains steam, Bristol Myers Squibb is leaning in with some hefty counterweight.

The pharma giant is planning to tender its Acceleron shares, Bloomberg reported, which add up to a sizable 11.5% stake. Based on the offer price, the sale would net Bristol Myers around $1.3 billion.

To complete its deal, Merck needs a majority of shareholders to agree to sell their shares.

Ep­i­darex, Sofinno­va dou­ble down on a par­al­lel take on 3rd-gen CAR-T — aim­ing straight at ovar­i­an can­cer

When John Maher treated the first head and neck cancer patient at Guy’s Hospital in London with his pan-ErbB CAR-T back in 2015, he was among a small club of researchers convinced they had an answer to the challenges that had kept those engineered T cells — wildly successful in hematological cancers — either too dangerous or out of reach for patients with solid tumors.

The field has blossomed since then, with a proliferation of technologies that promise to address any number of challenges identified as unique to solid tumors. And Maher himself has rethought his approach and come up with a new CAR-T platform to generate the next slate of candidates.

Boost­er bo­nan­za: FDA en­dors­es 'mix-and-match' scheme, and Mod­er­na and J&J too

The FDA late Wednesday signed off on authorizing the use of heterologous — or what FDA calls a “mix and match” of a primary vaccine series and different booster doses — for all currently available Covid-19 vaccines, in addition to separately authorizing Moderna and J&J boosters.

On the mix-and-match approach, which FDA officials insisted isn’t too confusing in a press conference, the agency offered the example of an 18-year-old who received the J&J shot at least two months ago and may now receive a single booster of the J&J, a half dose of the Moderna, or the Pfizer-BioNTech booster.

David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.