Gaurav Shah, Rocket Pharmaceuticals CEO

FDA lifts clin­i­cal hold on Rock­et's gene ther­a­py for heart fail­ure af­ter 3-month de­lay

New Jer­sey’s Rock­et Phar­ma­ceu­ti­cals hit a ma­jor road­block for its AAV-de­liv­ered gene ther­a­py for heart fail­ure in May af­ter the FDA came ask­ing for more de­tails on tar­get pa­tients. Three months down the road, Rock­et is clear for liftoff once again.

The FDA on Mon­day lift­ed its clin­i­cal hold on Rock­et’s Phase I tri­al for gene ther­a­py can­di­date RP-A501 for Danon dis­ease, an ul­tra-rare ge­net­ic dis­or­der that can lead to heart fail­ure in chil­dren and young adults, the com­pa­ny said in a re­lease.

It’s been a lit­tle more than three-month wait for Rock­et af­ter the agency ground­ed the tri­al, ask­ing for a re­vised pro­to­col on “pa­tient se­lec­tion and man­age­ment.”

On Mon­day, Rock­et said it had ad­dressed those is­sues and was ready to move for­ward, cit­ing no new safe­ty red flags churned up dur­ing the agency’s re­view. The com­pa­ny ex­pects to re­sume dos­ing in the adult co­hort “as soon as pos­si­ble” and is gear­ing up to be­gin en­roll­ment for a pe­di­atric co­hort as well, Rock­et said.

Un­like the rest of its pipeline, which us­es lentivi­ral vec­tors to trans­port its gene ther­a­pies, RP-A501 is Rock­et’s on­ly can­di­date that us­es AAV9 for de­liv­ery, a vec­tor that has re­ceived in­tense scruti­ny af­ter a wave of safe­ty flags in re­cent years. The ther­a­py us­es that vec­tor to trans­port a func­tion­al ver­sion of lyso­some-as­so­ci­at­ed mem­brane pro­tein 2 (LAMP-2) in­to pa­tients’ cells — the mu­tat­ed form of the pro­tein caus­es a buildup of au­tophago­somes and glyco­gen in the heart and oth­er tis­sues that can cause heart fail­ure and death, par­tic­u­lar­ly in boys and young men.

Rock­et be­lieves there are some­where be­tween 15,000 and 30,000 pa­tients with Danon in the US and Eu­rope. The drug would be­come the first gene ther­a­py ap­proved for mono­genic heart fail­ure, Rock­et said.

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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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.

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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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.