Sage shrugs off po­ten­tial com­pe­ti­tion in post­par­tum de­pres­sion, as­serts plan to re­main in­de­pen­dent

Post­par­tum de­pres­sion (PPD), a so-far un­tapped mar­ket, is large enough for mul­ti­ple play­ers with 1 in 7 women di­ag­nosed with the con­di­tion that has no spe­cif­ic ther­a­pies ap­proved by the FDA. But Sage $SAGE, which re­port­ed im­pres­sive late-stage da­ta on its pill in se­vere PPD pa­tients on Mon­day, is not wor­ried about po­ten­tial com­pe­ti­tion.

An­a­lysts have drawn cau­tious com­par­isons with Mar­i­nus $MRNS, whose drug ganax­olone is al­so un­der eval­u­a­tion for PPD. An IV for­mu­la­tion of ganax­olone is cur­rent­ly in a Phase II study in se­vere PPD pa­tients, while mid-stage da­ta from an oral for­mu­la­tion of ganax­olone in mod­er­ate PPD pa­tients is ex­pect­ed in the first half of this year.

Steve Kanes

Sage, mean­while, is clear­ly ahead. The FDA is ex­pect­ed to make a de­ci­sion on the ap­prov­abil­i­ty of its IV PPD drug, brex­anolone, by March 19, af­ter a set­back de­layed the de­ci­sion by three months. The com­pa­ny’s oral PPD drug — SAGE-217 — re­port­ed stel­lar Phase III da­ta on Mon­day, and Sage in­tends to wait for da­ta from a piv­otal study on the pill in pa­tients with ma­jor de­pres­sive dis­or­der (ex­pect­ed in 2020) — be­fore it sub­mits a mar­ket­ing ap­pli­ca­tion. The oral drug has se­cured the agency’s break­through ther­a­py des­ig­na­tion.

“I think we’re in two very dif­fer­ent worlds, both sci­en­tif­i­cal­ly at this point and strate­gi­cal­ly as well,” Sage chief med­ical of­fi­cer Steve Kanes said in an in­ter­view with End­points News, when asked about the Mar­i­nus com­pound.

But Can­tor Fitzger­ald an­a­lysts pre­dict the two com­pa­nies may end up with equal mar­ket share con­sid­er­ing the size of the large­ly un­tapped mar­ket — if both man­age to take their re­spec­tive drugs across the fin­ish line.

“We mod­el ~572,000 women per year in the U.S. are af­fect­ed with PPD and be­lieve both Sage and Mar­i­nus (we mod­el 30% peak mar­ket pen­e­tra­tion) could have fair mar­ket share ver­sus ex­ist­ing ther­a­pies that take too long to work and are not ef­fi­ca­cious for a ma­jor­i­ty of pa­tients,” they wrote in a note.

Sage de­clined to pro­vide de­tail on their ex­pect­ed time­line to­ward ap­proval for SAGE-217 and sug­gest­ed it was too ear­ly to talk about pric­ing.

When asked about whether the com­pa­ny would be open to takeover dis­cus­sions, Kanes re­it­er­at­ed their plan was al­ways to grow and de­vel­op and in­de­pen­dent com­pa­ny — from dis­cov­ery all the way to com­mer­cial­iza­tion.

“The kinds of op­por­tu­ni­ties we look for are ones that ac­cel­er­ate treat­ment for pa­tients — a good ex­am­ple of that is our part­ner­ship Shino­gi in Japan,” Kanes said.

Japan’s Sh­iono­gi last June agreed to an up $575 mil­lion deal to bag lim­it­ed Asian rights to the SAGE-2017 — with $90 mil­lion in as cash up­front, a day af­ter the Cam­bridge, Mass­a­chu­setts-based com­pa­ny se­cured break­through ther­a­py sta­tus from the reg­u­la­tor.

But when pressed on whether a takeover was off the ta­ble, Kanes did not di­rect­ly com­ment, elect­ing in­stead to say that op­er­at­ing as an in­de­pen­dent en­ti­ty has al­ways been their po­si­tion.

Sage’s SAGE-217 re­sults on Mon­day morn­ing cat­a­pult­ed the stock up about 44% to $140.66 in mid­day trad­ing.

“We had thought that a pos­i­tive study would fun­da­men­tal­ly add in the $15-20 range. How­ev­er, that fore­cast was way off giv­en the $50-55 pos­i­tive move this morn­ing, much of which we would at­tribute to ex­cite­ment re­gard­ing M&A in the biotech space, and since SAGE has been viewed as a po­ten­tial take out can­di­date,” Leerink an­a­lysts wrote in a note.

NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data are messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data are exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty

 

I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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Marty Duvall, Oncopeptides CEO

On­copep­tides stock craters as it pulls can­cer drug Pepax­to from the mar­ket

Shares of Oncopeptides crashed more than 70% in early Friday trading after the company said it’s pulling its multiple myeloma drug Pepaxto (melphalan flufenamide) from the US market after failing a confirmatory trial. The move will force the company to close its US and EU business units and enact significant layoffs.

The FDA had scheduled an adcomm meeting next Thursday to discuss Pepaxto, which first won accelerated approval in February and costs about $19,000 per course of treatment. The committee was to weigh in on whether the confirmatory trial demonstrated a worse overall survival in the treatment arm compared to the control arm.

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Oc­u­lar Ther­a­peu­tix ham­mered by a PhII fail­ure in dry eye dis­ease — shares tank

Ocular Therapeutix $OCUL has had its ups and downs in the 7 years since it went public. Friday was one of those down days.

The Bedford, MA-based biotech reported that its lead experimental eye drug, OTX-CSI (cyclosporine intracanalicular insert), failed a Phase II trial for dry eye disease. And the stock experienced one of its periodic meltdowns, dropping more than 30% ahead of the bell.

The therapy flat failed the primary endpoint: increased tear production at 12 weeks as measured by the Schirmer’s Test compared to the vehicle control group. And while investigators called out an improvement from baseline in “signs of dry eye disease as measured by total corneal fluorescein staining (CFS) and symptoms of dry eye disease as measured by the visual analogue scale (VAS) eye dryness in subjects treated with the OTX-CSI insert,” it wasn’t statistically significant.

Pfiz­er pitch­es its Covid-19 vac­cine for younger chil­dren ahead of ad­comm next week

Pfizer will present its case to the FDA’s vaccine adcomm next week, seeking authorization for a lower-dose version of its Covid-19 vaccine for kids ages 5 through 12, which the Biden administration said will likely begin rolling out early next month.

Two primary doses of the 10 µg vaccine (the dose for those ages 12 and up is 30 μg) given 3 weeks apart in this group of children “have shown a favorable safety and tolerability profile, robust immune responses against all variants of concern including Delta, and vaccine efficacy of 90.7% against laboratory-confirmed symptomatic COVID-19,” the company said in briefing documents ahead of next Tuesday’s meeting of the FDA’s Vaccines and Related Biological Products Advisory Committee.

David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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