Bay­er boost­ed as FDA OKs its PI3K drug Aliqopa for re­lapsed fol­lic­u­lar lym­phoma pa­tients

A lit­tle more than a year ago, Gilead ran in­to some se­ri­ous prob­lems when its PI3K can­cer pill Zy­delig was linked with a rash of deaths in front­line stud­ies, crip­pling what was once con­sid­ered a po­ten­tial block­buster sales ef­fort. Now Bay­er is com­ing along with an in­fused ri­val that will try and start to fol­low up on the suc­cess en­joyed by John­son & John­son $JNJ and Ab­b­Vie’s $AB­BV Im­bru­vi­ca, a po­ten­tial mega-block­buster play­er which has been scor­ing some big gains in leukemia and lym­phoma.

The FDA has ap­proved Bay­er’s co­pan­lis­ib, with will now be mar­ket­ed as Aliqopa for re­lapsed fol­lic­u­lar lym­phoma. Reg­u­la­tors were quick to fol­low up af­ter pro­vid­ing Bay­er’s can­cer prospect with a pri­or­i­ty re­view, lop­ping 4 months off the reg­u­la­to­ry process.

Bay­er has some lim­it­ed am­bi­tions here, say­ing ear­li­er that it ex­pects to rack up about $600 mil­lion a year, in line with an­a­lysts es­ti­mates for its peak sales po­ten­tial.

Richard Padzur

The pri­ma­ry end­point in Phase II, which is be­ing used for the ac­cel­er­at­ed ap­proval, was over­all re­sponse among treat­ment re­sis­tant pa­tients. And in all groups the ORR of 59.2%, with a 12% com­plete re­sponse rate and a me­di­an du­ra­tion of re­sponse of more than 98 weeks, or 687 days. But pulling out the 104 fol­lic­u­lar lym­phoma pa­tients in CHRONOS-1, co­pan­lis­ib scored an ORR of 58.7%, in­clud­ing a com­plete re­sponse of 14.4% and a me­di­an DOR of more than 52 weeks, or 370 days.

“For pa­tients with re­lapsed fol­lic­u­lar lym­phoma, the can­cer of­ten comes back even af­ter mul­ti­ple treat­ments,” said Richard Paz­dur, di­rec­tor of the FDA’s On­col­o­gy Cen­ter of Ex­cel­lence. “Op­tions are lim­it­ed for these pa­tients and to­day’s ap­proval pro­vides an ad­di­tion­al choice for treat­ment, fill­ing an un­met need for them.”

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,300+ biopharma pros reading Endpoints daily — and it's free.

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.

Some can­cer pa­tients now have to find oth­er op­tions as Bris­tol My­er­s' Abrax­ane falls in­to short­age from man­u­fac­tur­ing woes

When Beth Hogan, a metastatic pancreatic cancer patient, showed up for her infusion at Yale’s Smilow Cancer Hospital in New Haven, CT on Oct. 11, she said she was informed that day that she would not be receiving Bristol Myers Squibb’s Abraxane, part of her combo treatment, because of a shortage.

“I was told we don’t know when you can have it,” she told Endpoints News via email, adding that she doesn’t expect to receive any Abraxane this coming Monday at her treatment appointment either, and she doesn’t know when things will change.

Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Valeriano Di Domenico)

Up­dat­ed: Bio­gen sells just $300K worth of Aduhelm in Q3, as ques­tions on long-term vi­a­bil­i­ty re­main

Barely anyone is accessing Biogen’s controversial Alzheimer’s treatment, with the company reporting just $0.3 million in Aduhelm sales in the third quarter. Although investors will be looking to the longer term, when CMS may decide to cover the drug and open the floodgates for more reimbursement, use of the drug is currently stalled.

Since June, when the FDA first signed off on the drug under its accelerated pathway, Biogen said Wednesday that it’s sold a total of $2 million worth of Aduhelm. That’s a far cry from the peak Wall Street sales estimate of about $9 billion in annual sales, and even a ways away from the sell-side consensus of about $17 million in Q3 sales.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,300+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Eli Lil­ly or­dered to pay roy­al­ties on block­buster di­a­betes drugs, though ex­act dam­ages are un­clear

A federal court found Eli Lilly in breach of a royalty agreement with an Arizona company, likely sending the case — which deals with Lilly’s blockbuster diabetes drugs — to a trial.

The Arizona District Court ordered Lilly to pay the royalties to Tucson, AZ-based Research Corporation Technologies, per an opinion delivered Tuesday, stemming from a 1990 agreement involving materials used in manufacturing Lilly’s insulin products. Lilly had agreed to pay a 2% royalty on worldwide sales, and the exact amount of damages will be determined in a trial, Judge Scott Rash wrote.

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.