FDA ap­proves trail­blaz­ing post­par­tum de­pres­sion ther­a­py, in cru­cial mile­stone for wom­en's health

In a land­mark de­ci­sion, the US health reg­u­la­tor has ap­proved the first treat­ment for moth­ers suf­fer­ing from post­par­tum de­pres­sion (PPD), a com­mon but of­ten over­looked and stig­ma­tized com­pli­ca­tion of child­birth that af­fects an es­ti­mat­ed 1 in 7 women.

The man­u­fac­tur­er of the in­jectable treat­ment — Sage Ther­a­peu­tics $SAGE — has been busy, rais­ing more than half a bil­lion dol­lars through the sale of its stock to sup­port the com­mer­cial roll­out of the drug, hir­ing a 180-strong sales­force and set­ting up se­lect cen­ters for women to get ad­min­is­tered with the one-time treat­ment un­der med­ical su­per­vi­sion.

The drug brex­anolone — to be sold as Zul­res­so — was orig­i­nal­ly de­signed for at-home in­fu­sions. But that ex­pec­ta­tion was scut­tled last year, af­ter the agency and ex­perts un­der­scored con­cerns about faint­ing episodes, which oc­curred in 6 of 140 women ex­posed to brex­anolone dur­ing in­fu­sion. Still, the drug won a ring­ing en­dorse­ment by the FDA ad­vi­so­ry pan­el on the ba­sis that des­ig­nat­ed fa­cil­i­ties to mon­i­tor ad­min­is­tra­tion (en­com­pass­ing a 12-hour fol­low-up to the 60-hour in­fu­sion) would be set up. Sage ac­com­mo­dat­ed this de­mand by sub­mit­ting a fresh risk eval­u­a­tion and mit­i­ga­tion strate­gies (REMS) plan, which pushed the FDA de­ci­sion date by three months to March 19.

Mike Cloo­nan

The treat­ment — which car­ries a boxed warn­ing high­light­ing the risk of sud­den loss of con­scious­ness — is ex­pect­ed to launch in late June, af­ter the DEA sched­ules the drug, con­sis­tent with oth­er ap­proved GABAer­gic ther­a­pies — agents that di­rect­ly mod­u­late the GA­BA sys­tem in the body or the brain.

Sage has as­signed a list price of $7,450 per Zul­res­so vial, re­sult­ing in a pro­ject­ed av­er­age course of ther­a­py cost of $34,000 per pa­tient be­fore dis­counts, a com­pa­ny spokesper­son said on Tues­day.

The drug de­vel­op­er has been con­sult­ing with “hun­dreds of pay­ers” to se­cure ac­cess, Sage’s chief busi­ness of­fi­cer Mike Cloo­nan said in an in­ter­view with End­points News ahead of the de­ci­sion.

In dis­cus­sions with phar­ma­cy ben­e­fit man­agers, the com­pa­ny feels “com­fort­able that we won’t have to re­bate a sig­nif­i­cant por­tion here, be­cause of the in­no­va­tion here we’re build­ing with Zul­res­so,” he said.

As part of its REMS strat­e­gy, the com­pa­ny is al­so work­ing on es­tab­lish­ing des­ig­nat­ed cen­ters where women can ac­cess the ther­a­py un­der med­ical su­per­vi­sion, Cloo­nan added, not­ing that “it can take months to get a site cer­ti­fied, reg­is­tered, and to es­tab­lish re­im­burse­ment path­ways.”

PPD is con­sid­ered a life-threat­en­ing con­di­tion be­cause pa­tients car­ry a risk of sui­cide, but aware­ness of the dis­or­der is patchy com­pared to oth­er ma­jor de­pres­sive con­di­tions, part­ly due to the so­cial stig­ma of be­ing la­beled an “un­hap­py moth­er” — an is­sue a grow­ing num­ber of celebri­ty moth­ers such as mod­el and cook­book au­thor Chris­sy Teigen have brought in­to the cul­tur­al zeit­geist.

PPD, which is an um­brel­la term for sev­er­al mood dis­or­ders, has pro­found neg­a­tive ef­fects on the ma­ter­nal-in­fant bond and lat­er in­fant de­vel­op­ment. Al­though a num­ber of an­ti­de­pres­sants ex­ist in the mar­ket, there is lit­tle ev­i­dence of their ef­fi­ca­cy in PPD, they usu­al­ly take 6 to 8 weeks to kick in and none are specif­i­cal­ly ap­proved for PPD.

Ac­cord­ing to Sage, rough­ly 400,000 women in the Unit­ed States suf­fer from PPD any giv­en year, al­though on­ly about half are di­ag­nosed. With Zul­res­so, the com­pa­ny ini­tial­ly plans to tar­get the se­vere PPD pop­u­la­tion, rep­re­sent­ed by about 20-30% of those 200,000 iden­ti­fied pa­tients.

Since pa­tients must be con­tin­u­ous­ly mon­i­tored by a health­care pro­fes­sion­al and ac­com­pa­nied dur­ing in­ter­ac­tions with their chil­dren when be­ing in­fused with the ther­a­py, Stifel an­a­lyst Paul Mat­teis’ fore­cast was com­par­a­tive­ly mod­est. He pro­ject­ed about $270 mil­lion in peak US sales in 2023, based on 10% pen­e­tra­tion in the se­vere PPD set­ting, with no use in mod­er­ate/mild pa­tients.

Mean­while, the com­pa­ny’s keen­ly watched oral PPD ther­a­py — SAGE-217 — is the one with big tick­et block­buster po­ten­tial, hav­ing re­cent­ly cleared a Phase III study with fly­ing col­ors. The pill — al­so be­ing eval­u­at­ed for ma­jor de­pres­sive dis­or­der (MDD) and oth­er mood dis­or­ders — ap­pears to be an im­prove­ment over brex­anolone as it is not prone to in­duc­ing the loss of con­scious­ness side ef­fect seen with the use of the in­jectable. Sage in­tends to wait for da­ta from a piv­otal study on the pill in pa­tients with ma­jor de­pres­sive dis­or­der (ex­pect­ed in 2020) be­fore sub­mit­ting a mar­ket­ing ap­pli­ca­tion.

Sage’s main ri­val is 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 are ex­pect­ed in the first half of this year.

Zul­res­sa’s ap­proval bodes well for Mar­i­nus $MRNS, ar­gued Jef­feries’ An­drew Tsai in a re­cent note. “We think ap­proval would have a neu­tral im­pact on Mar­i­nus, de­spite Sage be­ing 2-3 years ahead, giv­en: 1) FDA ap­proval should de-risk the class/mech­a­nism broad­ly, and 2) the FDA has re­quired Sage to in­tro­duce a REMS pro­gram that lim­its its use case to a cer­ti­fied health­care fa­cil­i­ty (e.g. hos­pi­tal), which may open up an op­por­tu­ni­ty for Mar­i­nus. Sage‘s next-gen oral drug (SAGE-217) has al­so shown com­pelling ef­fi­ca­cy in Phase II/III PPD and MDD stud­ies and so far does not cause faint­ing (syn­cope) or loss of con­scious­ness (e.g. al­low­ing for home use), but our base case as­sump­tion is for the play­ers to share parts of the PPD mar­kets.”

Gilead CEO Dan O'­Day of­fers a de­tailed ex­pla­na­tion on remde­sivir ac­cess — re­as­sur­ing an­a­lysts that Covid-19 da­ta are com­ing fast

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDA’s orphan status for remdesivir — reserved to encourage the development of rare disease therapies — Gilead CEO Daniel O’Day had some explaining to do about the company’s approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program — one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

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Ven­ture Cap­i­tal as a Strate­gic Part­ner: Fu­el­ing In­no­va­tion be­yond Fi­nance

The average level of investment required for a biotech start-up to succeed is increasing every year, elevating the pressure even further on venture capital to make smart financial investments. Financial investment alone, however, does not always guarantee that exciting innovations can be transformed into real businesses that make a meaningful difference to patients.

Beyond just capital

At Astellas Venture Management (AVM) – a wholly-owned venture capital organization within Astellas, headquartered in the San Francisco Bay Area – capital is just one of the ingredients we offer to add value to our biotechnology investments and partnerships. We generally take a strategic investor approach for companies in our invested portfolio, providing access to expertise, technology and/or resources in addition to the injection of finance. An equity investment from AVM can include access to Astellas’ research and development (R&D) capabilities and expertise, and a global network of partner academic institutions and biotechnology companies, to help advance and accelerate the start-up’s innovation.

Covid-19 roundup: In­ter­cept, blue­bird and a grow­ing list of biotechs feel the pain as pan­dem­ic man­gles FDA, R&D sched­ules

Around 100 staffers at Boston area hospitals have now tested positive for Covid-19, spotlighting the growing risk that the pandemic will sideline many of the most essential workers in healthcare as caseloads peak in the US and around the globe. With more than 3,400 deaths, Spain has become the latest country to surpass the official death count attributed to the new coronavirus in China, where the outbreak originated. As of Thursday morning, confirmed global cases had crossed 470,000 and the death count eclipsed 21,000.

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Af­ter crit­ics lam­bast­ed Gilead for grab­bing the FDA's spe­cial rare drug sta­tus on remde­sivir, they're giv­ing it back

Two days after Gilead won orphan drug status for remdesivir as a potential treatment for Covid-19, they’re handing it back.

The company was slammed from several sides after Gilead reported that the FDA had come through with the special status, which comes with 7 years of market exclusivity, the waiver of FDA fees and some tax credits as well. Typically, everyone who can get orphan status lands it without much of a fuss, but Democratic presidential candidate Bernie Sanders, Public Citizen and other consumer groups were outraged.

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Mod­er­na CEO Stéphane Ban­cel out­lines a short path for emer­gency use of a coro­n­avirus vac­cine

NIAID director Anthony Fauci has left no doubts that it takes 12 to 18 months to get a new vaccine tested and in commercial use, in the best of circumstances. But in times of a global emergency — like these — maybe there’s another, faster route to follow.

In an SEC filing on Tuesday, Moderna $MRNA staked out a record-setting pathway to getting their mRNA vaccine into the frontline of the healthcare response as early as this fall. The SEC filing notes that CEO Stéphane Bancel told Goldman Sachs that an emergency use approval could allow the vaccine to go to healthcare workers and certain individuals in a matter of months — presumably provided the NIH sees the safety and efficacy data they would need from the Phase I.

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Caught in a Covid-19 mael­strom, Eli Lil­ly locks down clin­i­cal tri­als as multi­bil­lion-dol­lar R&D ops de­rail

The Covid-19 pandemic has derailed Eli Lilly’s $6 billion R&D operations.

The pharma giant reported Monday morning that it has decided to hit the brakes on most new study starts and pause enrollment for most ongoing studies. Lilly adds that it is continuing dosing for ongoing studies, “but with study-by-study consideration.”

The pandemic has severely disrupted healthcare systems around the globe, says Lilly, making it difficult or impossible to conduct studies at many research sites. And there’s no timeline for when it expects to get back on track.

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UP­DAT­ED: Ver­tex joins Mer­ck, Pfiz­er — re­vamp­ing multi­bil­lion-dol­lar tri­al strat­e­gy as biotech R&D crum­bles

You can add Pfizer, Merck and — as we found out Friday morning — Vertex to the growing list of pharma giants hitting the pause button on a range of clinical trials. But not everyone in R&D is getting a red light.

Vertex says that it’s doing its best to keep working its pipeline strategy, coming up with a plan “to enable virtual clinic visits and home delivery of study drug to ensure study continuity and medical monitoring, and to facilitate study procedures.”

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Covid-19 roundup: Bris­tol My­ers sus­pends clin­i­cal tri­als, grounds field team; Vir ush­ers an­ti­body can­di­dates to hu­man test­ing

The global nature of the Covid-19 pandemic is manifesting more profoundly every day. With Spain’s death toll now surpassing China’s and India on full lockdown, the number of confirmed cases around the world has exceeded 436,000 while recoveries edged close to 112,000.

While the outbreak derails R&D at another pharma giant, several drugmakers have some encouraging updates on both experimental and repurposed molecules. Philanthropic campaigns in anticipation of the economic fallout continue. An Australian biotech is taking extreme measures to hunker down. There’s also an alternative epidemiology model emerging out of the UK, stirring up more discussion regarding the true extent of the infections in the country.

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As share buy­backs come un­der scruti­ny, what's in store for the bio­phar­ma in­dus­try?

Stock buybacks are not to be permitted for companies that will be bailed out in the coronavirus stimulus package, Congressional leaders have signaled. To what degree the biopharma industry has relied on buybacks for earnings growth in recent years, and if the trend continues, are the big questions as scrutiny into the practice heightens and balance sheets weaken with the coronavirus pandemic wreaking havoc on global economies.

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