Remi Barbier, Cassava CEO

Up­dat­ed: DOJ opens crim­i­nal probe in­to Cas­sa­va over al­le­ga­tions of Alzheimer's drug da­ta ma­nip­u­la­tion — re­port

Months af­ter re­ports of al­le­ga­tions re­gard­ing Cas­sa­va Sci­ences’ lead drug can­di­date for Alzheimer’s emerged, the DOJ may now be rais­ing its head to in­ves­ti­gate the com­pa­ny.

A re­port from Reuters pub­lished this morn­ing said that two sources had con­firmed the US De­part­ment of Jus­tice opened an in­ves­ti­ga­tion in­to Cas­sa­va over al­le­ga­tions that the biotech had ma­nip­u­lat­ed search re­sults re­lat­ed to simu­fil­am, the com­pa­ny’s ex­per­i­men­tal Alzheimer’s drug.

The sources fur­ther told the out­let that the DOJ per­son­nel in­ves­ti­gat­ing Cas­sa­va “spe­cial­ize in ex­am­in­ing whether com­pa­nies or in­di­vid­u­als have mis­led or de­fraud­ed in­vestors, gov­ern­ment agen­cies or con­sumers.” The sources did not de­tail the fo­cus of the probe and if any­one spe­cif­ic was be­ing looked in­to.

Shares of $SA­VA were down ap­prox­i­mate­ly 30% in pre-mar­ket trad­ing af­ter clos­ing Tues­day at over $21 a share.

The SEC had start­ed in­ves­ti­gat­ing the com­pa­ny last No­vem­ber af­ter Cas­sa­va re­vealed that cer­tain un­named gov­ern­ment agen­cies had asked the com­pa­ny for doc­u­men­ta­tion, go­ing out of its way to say that the re­quests were not ac­cu­sa­tions of wrong­do­ing.

This saga got its start last Au­gust, af­ter law firm La­ba­ton Sucharow filed a cit­i­zen’s pe­ti­tion with the FDA on be­half of two doc­tors ask­ing the reg­u­la­to­ry agency to halt all the com­pa­ny’s clin­i­cal tri­als un­til the com­pa­ny’s da­ta had been au­dit­ed. The two doc­tors are neu­ro­sci­en­tist and Janssen’s for­mer chief of neu­ro­science dis­cov­ery David Bredt, and car­di­ol­o­gist Ge­of­frey Pitt, di­rec­tor of Weill Cor­nell Med­i­cine’s Car­dio­vas­cu­lar Re­search In­sti­tute.

The two doc­tors told the Wall Street Jour­nal in a sep­a­rate re­port last year that they had short­ed Cas­sa­va’s stock, bet­ting that the share price would fall once in­vestors rec­og­nized the prob­lems the pair found.

Hoau-yan Wang

Ac­cord­ing to the re­port, the firm start­ed in­ves­ti­gat­ing Cas­sa­va af­ter find­ing re­sults “most un­ex­pect­ed and are prob­a­bly unique” to two sci­en­tists: re­searcher Hoau-Yan Wang at City Uni­ver­si­ty of New York and Cas­sa­va’s VP of neu­ro­sciences Lind­say Burns. The sci­en­tists had pub­lished mul­ti­ple pa­pers de­tail­ing the com­pa­ny’s hy­poth­e­sis about a con­nec­tion of fil­amin A pro­tein with Alzheimer’s dis­ease — and that simu­fil­am could ad­dress it.

How­ev­er, the law firm didn’t buy it. The re­port not­ed,

This ini­tial analy­sis sug­gests a pat­tern of clear er­rors and anom­alies that are con­sis­tent with da­ta ma­nip­u­la­tion and mis­rep­re­sen­ta­tion. These find­ings un­der­cut the foun­da­tion­al sci­ence on which simu­fil­am ther­a­py is based.

La­ba­ton Sucharow then point­ed to three types of da­ta it thought might be fal­si­fied: clin­i­cal bio­mark­er da­ta ap­par­ent­ly an­a­lyzed by Wang’s lab; West­ern blot analy­sis that al­leged­ly bore marks of al­ter­ation; and re­search in­volv­ing hu­man brain tis­sue us­ing a method­ol­o­gy that La­ba­ton Sucharow said “de­fies log­ic.”

CEO Re­mi Bar­bi­er de­nied the ac­cu­sa­tions this week, say­ing in a state­ment re­spond­ing to me­dia re­ports that “I have said from the on­set that al­le­ga­tions of re­search mis­con­duct are false. No gov­ern­ment agency has in­formed us that it has found sup­port­ing ev­i­dence of re­search mis­con­duct or any oth­er wrong-do­ing.”

In con­trast, short sell­ers, some of whom have been pur­su­ing an un­prece­dent­ed at­tack on Cas­sa­va Sci­ences, have re­port­ed­ly made over $100,000,000 short­ing the com­pa­ny’s $SA­VA stock.

“For a $100 mil­lion pay­day, some in­vestors may be in­cen­tivized to make false al­le­ga­tions,” said Re­mi Bar­bi­er.

The FDA re­ject­ed the pe­ti­tion sev­er­al months lat­er in Feb­ru­ary ear­li­er this year. How­ev­er, the fed­er­al reg­u­la­to­ry agency de­nied the pe­ti­tion on pro­ce­dur­al grounds, re­ply­ing that the FDA is not per­mit­ted to ini­ti­ate in­ves­ti­ga­tions based on cit­i­zen pe­ti­tions.

CDER di­rec­tor Pa­trizia Cavaz­zoni not­ed at the time that the FDA was con­cerned about the is­sues raised by the short sell­er.

“We take the is­sues you raise se­ri­ous­ly. Please note that your Pe­ti­tions are be­ing de­nied sole­ly on the grounds that your re­quests are not the ap­pro­pri­ate sub­ject of a cit­i­zen pe­ti­tion,” Cavaz­zoni wrote in her re­sponse to the firm. “This re­sponse does not rep­re­sent a de­ci­sion by the Agency to take or re­frain from tak­ing any ac­tion re­lat­ing to the sub­ject mat­ter of your Pe­ti­tions.”

Ed­i­tor’s note: this sto­ry has been up­dat­ed with com­ment from Cas­sa­va Sci­ences.

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FDA commissioner Rob Califf (Tom Williams/CQ Roll Call via AP Images)

With drug pric­ing al­most done, Con­gress looks to wrap up FDA user fee leg­is­la­tion

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

Uğur Şahin, BioNTech CEO (Kay Nietfeld/picture-alliance/dpa/AP Images)

De­spite falling Covid-19 sales, BioN­Tech main­tains '22 sales guid­ance

While Pfizer raked in almost $28 billion last quarter, its Covid-19 vaccine partner BioNTech reported a rise in total dose orders but a drop in sales.

The German biotech reported over $3.2 billion in revenue in Q2 on Monday, down from more than $6.7 billion in Q1, in part due to falling Covid sales. While management said last quarter that they anticipated a Covid sales drop — CEO Uğur Şahin said at the time that “the pandemic situation is still very much uncertain” — Q2 sales still missed consensus by 14%.

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Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

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Pascal Soriot, AstraZeneca CEO (David Zorrakino/Europa Press via AP Images)

As­traZeneca and Dai­ichi Sankyo sprint to mar­ket af­ter FDA clears En­her­tu in just two weeks

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, just two weeks after submitting a supplemental BLA. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.

Sen­ate Dems cling to a sim­ple ma­jor­i­ty to pass some of the biggest drug pric­ing re­forms ever

The Pharmaceutical Research and Manufacturers of America — and their fleet of drug industry lobbyists on Capitol Hill — are known for never losing.

Whenever a big drug pricing bill comes up, an army of the industry group’s lobbyists descend onto the Hill and either smash it outright or dismantle it piece by piece.

But for perhaps the largest drug pricing reforms ever enacted, after more than a decade of Congress trying and failing to allow Medicare to negotiate prescription drug prices, those same lobbyists and their biopharma clients were dealt a stunning blow on Sunday afternoon.

David Reese, Amgen R&D chief

UP­DAT­ED: In a fresh dis­ap­point­ment, Am­gen spot­lights a ma­jor safe­ty is­sue with KRAS com­bo

Amgen had hoped that its latest study matching its landmark KRAS G12C drug Lumakras with checkpoint inhibitors would open up its treatment horizons and expand its commercial potential. Instead, the combo spurred safety issues that blunted efficacy and forced the pharma giant to alter course on its treatment strategy, once again disappointing analysts who have been tracking the drug’s faltering sales and limited therapeutic reach.

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GSK and IQVIA launch plat­form of US vac­ci­na­tion da­ta, show­ing drop in adult rates

Throughout the Covid-19 pandemic, the issue of vaccine uptake has been a point of contention, but a new platform from GSK and IQVIA is hoping to shed more light on vaccine data, via new transparency and general awareness.

The two companies have launched Vaccine Track, a platform intended to be used by public health officials, medical professionals and others to strengthen data transparency and display vaccination trends. According to the companies, the platform is intended to aid in increasing vaccine rates and will provide data on trends to assist public health efforts.

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Ab­b­Vie sur­veys emo­tion­al im­pact of chron­ic leukemia con­di­tion, finds 'roller coast­er' of emo­tions

Rare diseases often have more than just physical effects on patients — especially when it comes to chronic conditions. In the case of the rare slow-growing blood cancer chronic lymphocytic leukemia (CLL), AbbVie wanted to try to assess the mental and emotional toll on patients.

So it surveyed more than 300 CLL patients, caregivers and physicians. While each group differed in how they felt — caregivers overwhelmingly (81%) felt positive about their role, for instance — patients described a “roller coaster” of emotions traversing diagnosis to treatment to remission and even relapse for some.