Look­ing for an ear­ly suc­cess in PhII Alzheimer’s study, Bio­gen and Ei­sai just racked up the lat­est set­back

Chalk up an­oth­er big set­back for the amy­loid be­ta the­o­ry and an­oth­er failed at­tempt at blaz­ing a clin­i­cal path to suc­cess.

In­ves­ti­ga­tors for Bio­gen $BI­IB and Ei­sai say that their an­ti-amy­loid be­ta an­ti­body BAN2401 flunked the 12-month pri­ma­ry end­point in their Phase II study. They had hoped that us­ing Bayesian analy­sis meth­ods would set them up for an ear­ly win, based on a new goal for suc­cess us­ing a com­pos­ite dis­ease score with the pa­tients en­rolled. Now they will go on to the full 18 month mark and then run a check of bio­mark­ers for the dis­ease — which has proven to be a re­mark­ably un­re­li­able as­sess­ment for clin­i­cal ef­fi­ca­cy.

Bio­gen shares dropped more than 4% this morn­ing.

Lynn Kramer, Ei­sai

Still, the biotechs aren’t giv­ing up, hold­ing out hope that they can move in­to late-stage test­ing — a mine field for com­pa­nies in the space which no one has suc­cess­ful­ly nav­i­gat­ed in the past 15 years.

The no­tion that elim­i­nat­ing the tox­ic tan­gles seen in many, though not all, pa­tients di­ag­nosed with this mem­o­ry-wast­ing dis­ease has cost bil­lions of dol­lars of re­search cash in re­cent years. Eli Lil­ly tried and failed with solanezum­ab three times, and still has work on­go­ing. Mer­ck re­cent­ly failed on the first step with their BACE ap­proach and Vivek Ra­maswamy’s Ax­o­vant tanked af­ter they took an­oth­er failed ap­proach on a dif­fer­ent strat­e­gy for amp­ing cog­ni­tion.

With the po­ten­tial earn­ings from any suc­cess gen­er­al­ly rank­ing in the bil­lions, though, don’t look for any­one in the field to slow down or stop.

Bio­gen and Ei­sai are al­lied on three dif­fer­ent ther­a­pies for Alzheimer’s, with the late-stage ad­u­canum­ab and the BACE drug E2609 in­clud­ed in the pack­age.

For it to be con­sid­ered a suc­cess at 12 months, the da­ta would need to demon­strate a greater than 80% chance of suc­cess for see­ing a greater than 25% re­duc­tion in the dis­ease score. Ever­cor­eISI’s Umer Raf­fat says that we won’t re­al­ly know about the re­sult un­til we get to 18 months, and then he ex­pects to see it move ahead in­to late-stage stud­ies.

“By us­ing Bayesian sta­tis­tics in this unique­ly-de­signed tri­al we had hoped that it would en­able us to demon­strate clin­i­cal suc­cess faster than more tra­di­tion­al study de­signs. We now await the fi­nal study analy­sis which will be con­duct­ed af­ter 18 months of treat­ment, which rep­re­sents an amount of treat­ment time that is con­sid­ered as ap­pro­pri­ate for as­sess­ing ef­fi­ca­cy in dis­ease mod­i­fy­ing agents for Alzheimer’s dis­ease,” said Lynn Kramer, Ei­sai’s R&D chief for neu­rol­o­gy.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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Blueprint CEO Jeff Albers (file photo)

Blue­print plots re­turn to FDA with new Ay­vak­it da­ta in rare con­di­tion — and the an­a­lysts cheer

Over a decade after launch, Blueprint Medicines nabbed the first approval for their first drug earlier this year. Now, as they move forward with a Roche-partnered global launch, they’re touting data that could push them into more patients.

The Jeff Albers-led Cambridge biotech released their full pivotal data for Ayvakit in patients with advanced systemic mastocytosis. In one 53-person study, they showed that 76% of patients responded to the drug, 36% had complete responses and that on average their responses lasted for just over 3 years. A smaller, 32-patient study had a 75% response rate and most were still responding after 10.4 months, the last follow-up.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

Anthony Coyle (Repertoire)

Flag­ship's merged biotech Reper­toire nets ex-Pfiz­er CSO An­tho­ny Coyle as R&D chief

Flagship is building a big-name C-suite at its new, $220 million merged biotech.

Repertoire Immune Medicines, which already boasts former Bioverativ chief John Cox as its CEO, announced yesterday that Anthony Coyle, the former Pfizer CSO and the founding CEO of Pandion, will join as their head of R&D.

“As we progress clinical trials for our multi-clonal T cell candidates in immuno-oncology, Tony’s deep expertise in cellular immunology and novel therapeutic development will help us achieve our vision of creating a new class of transformative medicines for patients,” Cox said in a statement.

President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists. HHS con­tin­ues to claim Azar “will de­fer com­plete­ly to the FDA"

President Donald Trump, who seems intent on announcing a Covid-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

#ES­MO20: Bris­tol My­ers marks Op­di­vo's sec­ond ad­ju­vant win — eye­ing a stan­dard of care gap

Moving into earlier and earlier treatment lines, Bristol Myers Squibb is reporting that adjuvant treatment with Opdivo has doubled the time that esophageal or gastroesophageal junction cancer patients stay free of disease.

With the CheckMate-577 data at ESMO, CMO Samit Hirawat said, the company believes it can change the treatment paradigm.

While a quarter to 30% of patients typically achieve a complete response following chemoradiation therapy and surgery, the rest do not, said Ronan Kelly of Baylor University Medical Center. The recurrence rate is also high within the first year, Hirawat added.