Treve­na forges plan to take once-re­ject­ed opi­oid across fin­ish line, af­ter 'pro­duc­tive' FDA meet­ing

Tiny Treve­na may have a new lease on life for its con­tro­ver­sial opi­oid painkiller — re­ject­ed by the FDA last No­vem­ber cit­ing the dearth of drug safe­ty da­ta — fol­low­ing a scathing in­ter­nal staff cri­tique and lop­sided ex­pert pan­el re­view.

The com­pa­ny’s shares $TRVN swelled near­ly 120% on Mon­day af­ter Treve­na said the agency had agreed that the drug’s ex­ist­ing safe­ty data­base would suf­fice for a par­tic­u­lar dose of the drug — help­ing forge a path for­ward for the biotech to re­sub­mit its mar­ket­ing ap­pli­ca­tion.

The IV in­jec­tion, oliceri­dine, was de­vel­oped to help adult pa­tients man­age mod­er­ate to se­vere acute pain. It is de­signed to in­duce a sim­i­lar anal­gesic ef­fect as mor­phine, but work faster and re­duce the messy side ef­fects of se­da­tion, res­pi­ra­to­ry de­pres­sion and slow­ing gas­troin­testi­nal motil­i­ty. But mixed tri­al re­sults and gaps in safe­ty da­ta in­clud­ing QT in­ter­val da­ta — the time the heart mus­cle takes to recharge be­tween beats — prompt­ed the reg­u­la­tor to even­tu­al­ly re­ject the opi­oid in ear­ly No­vem­ber. At the time, the agency asked for a big­ger safe­ty data­base for the drug, as well as “cer­tain ad­di­tion­al non­clin­i­cal da­ta and val­i­da­tion re­ports”.

Car­rie Bour­dow

On Mon­day, Treve­na said its cur­rent safe­ty da­ta would sup­port la­bel­ing at a max­i­mum dai­ly dose of 27 mg, and that the FDA had agreed that the com­pa­ny con­duct a study (in­clud­ing place­bo- and pos­i­tive-con­trol arms) in healthy vol­un­teers to amass the re­quest­ed QT in­ter­val da­ta.

The Chester­brook, Penn­syl­va­nia-based drug de­vel­op­er in­tends to sub­mit a pro­to­col and analy­sis plan to the FDA short­ly and, up­on re­ceipt of reg­u­la­to­ry feed­back, ex­pects to ini­ti­ate the study in the first half of 2019. The com­pa­ny added it does not need any more ef­fi­ca­cy da­ta to re­sub­mit its mar­ket­ing ap­pli­ca­tion for the drug, but did not pro­vide any de­tails on when that re­sub­mis­sion could be ex­pect­ed.

CEO Car­rie Bour­dow said she was “en­cour­aged by the pro­duc­tive dis­cus­sion” with the FDA in a state­ment.

Max­ine Gowen

Back in 2016, un­der the be­hest of CEO Max­ine Gowen, Treve­na had sug­gest­ed an end-of-Phase II meet­ing with the FDA had cul­mi­nat­ed in a gen­er­al agree­ment about the Phase III de­sign for oliceri­dine, and that Gowen was “very pleased” with the dis­cus­sion. But it was on­ly last year that it was re­vealed that the FDA had in fact in­di­cat­ed oth­er­wise — the agency had dis­agreed with pro­posed dos­ing and the pri­ma­ry end­point in the late-stage pro­gram. This rev­e­la­tion prompt­ed at­tor­neys at Bern­stein Lieb­hard to ac­cuse Treve­na ex­ecs, par­tic­u­lar­ly for­mer CEO Max­ine Gowen, of mis­lead­ing in­vestors for rough­ly two years.

FDA pol­i­cy dic­tates it must not re­lease any in­for­ma­tion re­lat­ed to dis­cus­sions with drug spon­sors un­til a drug is up for re­view. Com­mis­sion­er Scott Got­tlieb had ini­tial­ly made promis­es to arm the agency to re­veal more in­for­ma­tion, for in­stance the reg­u­la­tor’s rea­son­ing be­hind is­sued CRLs, but that has not come to fruition.

Mean­while, the FDA’s ap­proval of an­oth­er opi­oid amidst a na­tion­al cri­sis of opi­oid abuse, mis­use and ad­dic­tion that kills about 130 Amer­i­cans each day will like­ly cause more fric­tion. Acel­Rx’s $ACRX ap­proval for its opi­oid Dsu­via last year sparked a flur­ry of in­tense crit­i­cism, even prompt­ing a mem­ber of the FDA’s own ex­pert pan­el to call out the agency’s at­ti­tude to­ward opi­oids as “will­ful blind­ness that bor­ders on the crim­i­nal.

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

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.

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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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, and could help shore up public trust in the agency and any eventual vaccine.

Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the biotech.

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News brief­ing: PureTech plans Nas­daq de­but with sec­ondary list­ing; GoodRx prices $725M IPO

London Stock Exchange-listed PureTech Health announced Wednesday that it’s looking to extend to Nasdaq. But due to its “strong cash position,” the biotech says it isn’t issuing any new shares in the potential secondary listing.

The company’s shares closed at £256.50 Tuesday on the London Stock Exchange. Its candidate LYT-100 is currently in Phase I development for various indications, including lymphatic flow disorders and fibrotic and inflammatory disorders. PureTech is expecting a Phase Ib readout in lymphatic flow disorders later this year, and is also planning to launch a Phase II study for the drug to treat respiratory conditions experienced after Covid-19.

Rep. Andy Harris (R-MD) (Tasos Katopodis/Pool via AP Images)

The mi­cro-cap that tapped a mask-skep­tic con­gress­man for their Covid DSMB is ap­ply­ing for an EUA. Their ev­i­dence? 21 pa­tients

NeuroRx, the tiny biotech that came under fire last week after Politico reported they selected a congressman and two other acquaintances of the CEO to supervise their Covid-19 drug trial, announced today that they will ask the FDA to authorize their drug based on the results of just 21 patients.

Such an application would test the agency’s standards of evidence for an EUA, which have already come under scrutiny after controversial authorizations for convalescent plasma and hydroxychloroquine. The only other company to announce their intention to file for an EUA, Eli Lilly, did so after results came back from a randomized control study testing their antibody in over 450 patients.

J&J re­leas­es PhI­II safe­ty blue­print for Covid-19 vac­cine tri­al. How does it stack up to Mod­er­na, Pfiz­er and As­traZeneca?

Along with the initiation of its Phase III Covid-19 vaccine study announced Wednesday morning, Johnson & Johnson also released its trial protocol, giving an inside look at how the company is conducting its late-stage research.

The move comes after the other three companies conducting Phase III’s in the US — Moderna, Pfizer and AstraZeneca — each disclosed their own trial blueprints within the last week. Though the release of such protocols is typically done after trials have been completed, drug developers had come under intense pressure after a brief safety scare in an AstraZeneca trial and amid growing concern of a politically motivated vaccine authorization.

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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