Sage ap­pears set for a break­through FDA OK on post­par­tum de­pres­sion drug, but who’s go­ing to use it?

Sage can start this week with a fair­ly firm idea on the time­line for its ini­tial trans­for­ma­tion in­to a mar­ket­ing op­er­a­tion, with the FDA and its out­side ex­perts ap­pear­ing to bless their land­mark drug brex­anolone for post­par­tum de­pres­sion. What they don’t know is who is go­ing to buy it.

The FDA’s ex­pert pan­el vot­ed to back the drug’s risk/ben­e­fit pro­file by a wide mar­gin, 17 in fa­vor and on­ly 1 against, with sev­er­al of the votes cast with an en­thu­si­as­tic en­dorse­ment for the po­ten­tial here. Com­ing in the wake of an up­beat in­ter­nal as­sess­ment at the agency, the writ­ing ap­pears to be on the wall for an OK by the De­cem­ber 19 PDU­FA date.

But the same sources have al­so in­di­cat­ed that for a new drug like this, tied to episodes of un­con­scious­ness among the small group of pa­tients who have tak­en a drug de­signed for a very large pop­u­la­tion — some­thing that may well have scut­tled any ap­pli­ca­tion ahead of the lib­er­al reg­u­la­to­ry era we’re in now — it al­so ap­pears like­ly that the ap­proval will come with a re­quire­ment that pa­tients un­der­go the bur­den­some 72-hour in­fu­sion process un­der 24/7 med­ical care. And that’s like­ly to se­vere­ly rein in de­mand for the drug.

That sen­ti­ment may have weighed down the biotech’s share price. The stock was down 5% in Mon­day morn­ing trad­ing.

Paul Mat­teis

Stifel’s Paul Mat­teis was com­fort­able with the ap­par­ent reg­u­la­to­ry at­ti­tude on abuse po­ten­tial, leav­ing the door open to an ap­proach that works for sleep­ing pills. But the ex­perts balked at home in­fu­sion, want­i­ng a set of des­ig­nat­ed fa­cil­i­ties that can in­fuse the ther­a­py over 60 hours with a 12-hour fol­lowup on safe­ty.

Al­though it was clear that FDA (and of course Sage $SAGE) would have liked to be able to OK in-home in­fu­sions and min­i­mize the bur­den for post-par­tum moth­ers, the agency – and the pan­elists – saw (loss of con­scious­ness) risks as too salient and too un­pre­dictable to al­low for use with­out rig­or­ous mon­i­tor­ing. It re­mains to be seen how spe­cif­ic FDA will be with the type of cen­ter in which it al­lows for use, but over­all the dis­cus­sion here was con­sis­tent with what one would’ve ex­pect­ed from the brief­ing book. Pan­elists asked for a va­ri­ety of things like clear treat­ment pro­to­cols, specifics on mon­i­tor­ing, and a few even men­tioned a black box warn­ing for “falls” would be a smart add; FDA not­ed it still needs to ham­mer out the de­tails of a REMS/la­bel, but its pro­pos­al for the for­mer is to cer­ti­fy health­care fa­cil­i­ties and have a cer­tain list of cri­te­ria that a fa­cil­i­ty can at­test to (con­tin­u­ous mon­i­tor­ing through 12-hours post in­fu­sion). On what ad­di­tion­al da­ta could be col­lect­ed for home in­fu­sions the pan­el was pret­ty vague, hop­ing for a large post-mar­ket­ing safe­ty data­base and/or stud­ies that could look at the ef­fi­ca­cy of short­er du­ra­tion reg­i­mens.

Add it all up, and Mat­teis is mak­ing a pre­dic­tion of $300 mil­lion in peak sales.

The mar­ket, though, is less in­ter­est­ed in the strings put on this ap­proval than the un­der­stand­ing they can glean about the FDA’s ap­proach to their oral fol­lowup SAGE-217. That drug is the key to un­lock­ing the block­buster mar­ket avail­able here. And so far, so good, with reg­u­la­tors gear­ing up to re­view a drug that has — so far — not been tied to the same safe­ty is­sues. 

With safe­ty da­ta on 140 pa­tients, Sage is still walk­ing on an in­cred­i­bly risky tightrope as it nav­i­gates its way in­to the mar­ket. Once used in a big­ger pop­u­la­tion, the drug is ripe for con­tro­ver­sy if new is­sues arise. On the oth­er hand, if Sage mas­ters the change and gets green-light­ed for PPD, it will be well on its way to jus­ti­fy­ing its multi­bil­lion-dol­lar val­u­a­tion. 

The stakes are enor­mous.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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The Advance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

No­var­tis jumps in­to Covid-19 vac­cine hunt, as Big Phar­ma and big biotech com­mit to bil­lions of dos­es

After spending most of the pandemic on the sidelines, Novartis is offering its aid in the race to develop a Covid-19 vaccine.

AveXis, the Swiss pharma’s gene therapy subsidiary, has agreed to manufacture the vaccine being developed by Massachusetts Eye and Ear and Massachusetts General Hospital. The biotech will begin manufacturing this month, while the vaccine undergoes further preclinical testing. They’ve agreed to provide the vaccine for free for clinical trials beginning in the second half of 2020, but have not disclosed financials for after.

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David Hoey (Vaxxas)

In for the long vac­cine game, Mer­ck buys in­to patch de­liv­ery tech with pan­dem­ic po­ten­tial

When Merck dived into the R&D fray for a Covid-19 vaccine earlier this week, execs made it clear that they’re not necessarily looking to be first — with CEO Ken Frazier throwing cold water on the hotly-discussed 12- to 18-month timelines. But when it does emerge from behind, the pharma giant clearly expects to play a significant part.

Part of that will depend on next-generation delivery technology that reshapes the world’s imagination of a vaccine.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

De­nali un­veils new way of cross­ing blood brain bar­ri­er as the big neu­ro­science bet en­ter its clin­i­cal years

Five years ago, as much of pharma began leaving neuroscience, three big-name scientists from Genentech and some A-list investors, including ARCH and Flagship, made a $217 million bet that new genetic insights and a reliance on biomarkers could bring them success. They called it Denali Therapeutics.

Still, Denali faced the problem that neuroscience developers have faced for decades: How do you get a large molecule across the blood-brain barrier, a natural defense evolved precisely to keep them out? Enzyme replacement therapy, for instance, would be a great candidate to treat several neurological disorders, but enzymes can’t cross the barrier.

Bris­tol My­ers Squibb fi­nal­ly gets in the front­line NSCLC game dom­i­nat­ed by Mer­ck, adding a sec­ond Op­di­vo/Yer­voy-based op­tion

Bristol Myers Squibb may be trailing Merck and Roche in the checkpoint race to treat frontline cases of non-small cell lung cancer, but as it does, it makes sure to bring its best feet forward.

Just days after scoring a landmark NSCLC approval for Opdivo and Yervoy alone for PD-L1 positive patients, the company said the FDA has also OK’d using the two agents with a limited course of chemo regardless of the biomarker status.

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