Sage trig­gers an­oth­er burst of in­vestor en­thu­si­asm as oral de­pres­sion drug scores in PhII

Sage Ther­a­peu­tics shares are once again rock­et­ing up this morn­ing, this time based on pos­i­tive re­sults from a small Phase II study of an oral ver­sion of their de­pres­sion ther­a­py.

Jeff Jonas

Long one of the most volatile stocks in biotech, Sage says that SAGE-217 clear­ly hit the goal post for sta­tis­ti­cal sig­nif­i­cance in ma­jor de­pres­sive dis­or­der, achiev­ing a 64% suc­cess rate in achiev­ing clin­i­cal re­mis­sion on a com­mon­ly used score for de­pres­sion against 23% on a place­bo.

Start­ing wth a mar­ket cap of $3.8 bil­lion this morn­ing, Sage saw its shares soar 67% af­ter the lat­est round of re­sults. And now they plan to take it in­to late-stage de­vel­op­ment.

Just a few weeks ago Sage’s shares al­so ral­lied on the news that SAGE-547 — their in­fused drug — pro­duced pos­i­tive re­sults in post­par­tum de­pres­sion. The re­sults waned from their mid-stage re­sults, but in­vestors backed the no­tion that Sage has some­thing im­por­tant in a field marked by re­peat­ed set­backs.

Sage al­so has been mak­ing big gains on da­ta from small stud­ies, which have to go a long way be­fore they are ful­ly eval­u­at­ed by reg­u­la­tors. That chal­lenge still re­mains.

Paul Mat­teis at Leerink wasn’t too wor­ried about that as he jumped in­to the cheer­ing sec­tion. He not­ed the po­ten­tial of a fast, ef­fec­tive de­pres­sion drug in a field where treat­ment fail­ure is com­mon:

For the drug arm, SAGE notes that sta­tis­ti­cal sep­a­ra­tion from base­line be­gan at day 2. By com­par­i­son, al­most all oth­er de­pres­sion tri­als for oral med­i­cines run for 4-6 weeks, with the as­sump­tion that it should take this long to show an ef­fect. It’s hard to un­der­state how mean­ing­ful these da­ta are in the back­drop of the very sig­nif­i­cant un­met med­ical need in de­pres­sion: even if the SAGE da­ta on a place­bo-ad­just­ed ba­sis weren’t dif­fer­en­ti­at­ed via cross-tri­al com­par­isons, we still think ‘217 would be an im­por­tant drug as it of­fers a new mech­a­nism (ex­tra-synap­tic GA­BA-mod­u­la­tion) in an area where the vast ma­jor­i­ty of op­tions gen­er­al­ly mod­u­late the same path­ways (sero­tonin, dopamine, nor­ep­i­neph­rine).

Sage’s shares crum­bled in Sep­tem­ber when its lead drug failed to out­per­form a place­bo in treat­ing a rare form of seizures. But back-to-back suc­cess­es have since then more than dou­bled the share price, adding bil­lions in mar­ket cap.

“These very en­cour­ag­ing da­ta sug­gest the po­ten­tial of SAGE-217 in the treat­ment of MDD as well as oth­er mood-re­lat­ed dis­or­ders that we may pur­sue,” said Jeff Jonas, chief ex­ec­u­tive of­fi­cer of Sage Ther­a­peu­tics. “There has been lit­tle in­no­va­tion in the dis­cov­ery and de­vel­op­ment of treat­ments for de­pres­sion in the last two decades. Cou­pled with our re­cent pos­i­tive Phase 3 da­ta read-out eval­u­at­ing brex­anolone for the treat­ment of post­par­tum de­pres­sion, the find­ings in this study sug­gest our pipeline of pro­pri­etary GABAA mod­u­la­tors may im­pact nov­el and fun­da­men­tal brain mech­a­nisms, of­fer­ing po­ten­tial de­vel­op­ment op­por­tu­ni­ties in a va­ri­ety of in­di­ca­tions.”

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.

Ver­sant-backed Chi­nook gets a $65M launch round for its dis­cov­ery quest in a resur­gent kid­ney field

Versant is once again stepping off the beaten track in biotech to see if they can blaze a trail of their own in a field that has looked too thorny to many investors for years.

The venture group and their partners at Apple Tree are bringing their latest creation out of stealth mode today. Born in Versant’s Inception Sciences’ Chinook Therapeutics is betting that its preclinical take on kidney disease can get an early lead among the companies starting up in the field.

Sir An­drew Dil­lon, NICE's first — and on­ly — chief ex­ec­u­tive to step down next year

Using a laptop borrowed from his former employer, South London’s St George’s Hospital, Sir Andrew Dillon set about establishing NICE — launched by the then health secretary Frank Dobson — in 1999.  On Thursday, the UK cost-effectiveness watchdog said its first and only chief executive — Dillon — is stepping down in March 2020.

Back in the day, decisions about which drugs and interventions were funded by the National Health Service (NHS) were made at the local level, but this ‘postcode prescribing’ system was fraught with skewed healthcare deployment making the structure unsustainable. A national system was deemed necessary — and NICE was formed to bridge that gap.