Ahead of ad­u­canum­ab fil­ing, Bio­gen nabs Pfiz­er castoff for $75M to ad­dress Alzheimer's, Parkin­son's symp­toms

Bio­gen be­lieves (not every­one agrees) it has a dis­ease-mod­i­fy­ing ther­a­py for Alzheimer’s on the cusp of an FDA ap­proval. Now it’s bring­ing in an ear­ly drug to al­so treat one of its symp­toms.

Al San­drock

PF-05251749 is an in­hibitor of ca­sein ki­nase 1, a fam­i­ly of en­zymes key to the reg­u­la­tion of cir­ca­di­an rhythm, that Pfiz­er had tak­en through Phase Ia be­fore culling its en­tire neu­ro­sciences unit. While some as­sets were sal­vaged in the form of a spin­out, this ap­par­ent­ly went up for sale and it’s go­ing for $75 mil­lion up­front. Bio­gen has com­mit­ted to $635 mil­lion in mile­stones.

In ad­di­tion to sun­down­ing in Alzheimer’s — a state of con­fu­sion, anx­i­ety, ag­gres­sion, ag­i­ta­tion or rest­less­ness be­set­ting about 20% of all pa­tients in the evenings — Bio­gen al­so sees po­ten­tial in a non-mo­tor symp­tom of Parkin­son’s dubbed ir­reg­u­lar sleep wake rhythm dis­or­der. Pa­tients with ISWRD have trou­ble sleep­ing at night and ex­pe­ri­ence sub­se­quent day­time sleepi­ness and se­vere fa­tigue, dis­rupt­ing their dai­ly lives.

At Pfiz­er, PF-05251749 had been test­ed in around 100 healthy vol­un­teers (in one case in con­junc­tion with mela­tonin). Bio­gen will now ini­ti­ate its own Phase Ib stud­ies for each of the two in­di­ca­tions it high­light­ed

For Baird an­a­lysts, it’s all a bit rem­i­nis­cent of the BMS-986168 (go­suranemab) deal from 2017, wrote Bri­an Sko­r­ney.

Back then Bio­gen paid $300 mil­lion up­front — siz­able, as with this new deal — to ac­quire go­suranemab from Bris­tol-My­ers Squibb — an­oth­er big phar­ma part­ner.

“Re­call, we re­cent­ly not­ed that go­suranemab flopped in the piv­otal Phase 2 PASS­PORT tri­al in pa­tients with pro­gres­sive supranu­clear pal­sy (PSP),” he added. “With re­spect to go­suranemab, it seems that Bio­gen got the short end of the stick, how­ev­er, on­ly time will tell as to whether or not the case will be the same with re­spect to PF-05251749.”

Al San­drock, Bio­gen head of R&D and big ad­u­canum­ab cheer­leader, re­mains op­ti­mistic about the po­ten­tial to ad­dress be­hav­ioral and neu­ro­log­i­cal symp­toms us­ing a CNS-pen­e­trant drug.

“This as­set is high­ly com­ple­men­tary to our ex­ist­ing pipeline of po­ten­tial dis­ease-mod­i­fy­ing ther­a­pies in Alzheimer’s and Parkin­son’s dis­eases,” San­drock, who’s al­so held on­to the CMO ti­tle, for now, said in a state­ment.

The deal kicks off a year when Alzheimer’s will loom ever larg­er at Bio­gen, as the com­pa­ny be­gins to find out whether its de­ci­sion to res­ur­rect the ad­u­canum­ab pro­gram fol­low­ing post hoc analy­sis of two Phase III stud­ies (one of which still failed re­gard­less) will fly with FDA reg­u­la­tors. The biotech has said it’d file for ap­proval in ear­ly 2020.

Their the­o­ry is that pa­tients need­ed to be con­sis­tent­ly ex­posed to a high-dose — 14 dos­es at 10mg/kg — as well as a bet­ter titra­tion reg­i­men to reap the full cog­ni­tive ben­e­fits of ad­u­canum­ab. But crit­ics slammed the ar­gu­ment, con­tend­ing that even if that were true, it would have to be borne out by a third, con­fir­ma­to­ry study be­fore the be­ta-amy­loid bind­ing an­ti­body can be ap­proved.

“We don’t file willy nil­ly,” San­drock pre­vi­ous­ly said in de­fense of the de­ci­sion to shoot for an OK with what they have. “We on­ly go to fil­ing when we be­lieve that there is a ben­e­fit-risk ar­gu­ment based on sci­ence, based on da­ta. And if you look at our his­to­ry, we haven’t done fil­ings right and left with­out good rea­son.”

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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UP­DAT­ED: Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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Ku­ra co-founder heads to Asian mul­ti-na­tion­al as biotech eyes the goal posts for lead drug

Six years after Kura Oncology snagged a farnesyl transferase inhibitor from J&J and leapt straight into clinical development, one of the biotech’s founders is leaving to start a new chapter in his career.

CMO and development chief Antonio Gualberto is exiting the company, and Kura — led by longtime biotech entrepreneur Troy Wilson — is on the hunt for a replacement. Wilson credited the CMO for some key biomarker work, including the discovery of the CXCL12 pathway as a target of their lead drug tipifarnib. Those biomarkers are being relied on to define the patient population most likely to benefit from the drug.

FDA waves Epizyme's $186K rare can­cer drug through to mar­ket — now get ready for the sec­ond act

After winning the hearts of the expert panel convened by the FDA despite a bleak in-house review and a checkered development history, Robert Bazemore has steered Epizyme to its first-ever OK for a rare cancer drug.

The approval in epithelioid sarcoma sets tazemetostat, now Tazverik, up nicely for a quick expansion to follicular lymphoma — a much bigger indication for which the biotech has just submitted an NDA.

2019 a 'trans­for­ma­tive year' for phar­ma M&A. Is that a good thing?

Big Pharma keeps getting bigger.

Fueled by the mega-mergers between Bristol-Myers Squibb and Celgene and between Allergan and AbbVie, the industry last year saw $350 billion worth of M&A, according to the new year-end report from the consultants at PwC.  That’s a more than 50% increase on 2018.

“I kind of look at 2019 as a transformational year,” report author Glen Hunzinger told Endpoints News. 

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Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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