Lit­tle Cara steers through their first pos­i­tive PhI­II with a ‘break­through’ drug and a path to the FDA

Cara Ther­a­peu­tics $CARA has suc­cess­ful­ly hit a key mile­stone in its push to get their lead drug across the fin­ish line at the FDA. The biotech says it racked up a set of clear­ly pos­i­tive da­ta for their one drug, Ko­r­su­va (CR845), in their first Phase III tri­al read­out for a se­vere itch­ing con­di­tion among pa­tients on he­modial­y­sis. And if they can do it again in a con­fir­ma­to­ry Phase III due out lat­er in the year, they’ll be ready to beat a trail to reg­u­la­tors in search of an OK.

The da­ta are solid­ly in its fa­vor this time, with slight­ly more than half of the pa­tients in the drug arm of the KALM-1 tri­al re­port­ing an im­prove­ment in symp­toms get­ting an in­jec­tion of the drug, com­pared to 28% in the con­trol arm. And there were some pos­i­tive scores in a slate of sec­on­daries that will stand in its fa­vor, in­clud­ing itch scores and a qual­i­ty of life mea­sure. And re­searchers re­port­ed no sig­nif­i­cant safe­ty is­sues that could ham­per the drug, a kap­pa opi­oid re­cep­tor ag­o­nist.

That was good for a boost on the stock price, as shares jumped 18% on Wednes­day morn­ing.

The biotech is all-in on this one, with a range of clin­i­cal tri­als in the pipeline for Ko­r­su­va. Jef­feries has pegged peak po­ten­tial sales at a step up from the $500 mil­lion mark, and Jef­feries an­a­lyst Mark How­er­ton has of­fered a few en­thu­si­as­tic thumbs up along the way, look­ing past the ini­tial IV for­mu­la­tion to oral drugs he be­lieves has greater mar­ket po­ten­tial for this in­di­ca­tion.

Derek Chalmers Cara Ther­a­peu­tics

CEO Derek Chalmers al­so said that in­ves­ti­ga­tors picked up signs of im­prove­ment af­ter the first week, then run­ning through the course of the 12-week pro­gram. In­ves­ti­ga­tors re­cruit­ed 350 pa­tients with mod­er­ate to se­vere pru­ri­tis for this study.

Cara bagged brag­ging rights to the FDA’s break­through ther­a­py des­ig­na­tion 2 years ago, just days be­fore the biotech took a hit on a failed pain tri­al they were run­ning for this drug among pa­tients with os­teoarthri­tis. But they’ve suc­cess­ful­ly re­fo­cused in­vestors on oth­er sources of pain and pru­ri­tis as they con­tin­ued to push ahead on this one drug.

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.

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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.

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As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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UP­DAT­ED: Pay­back? An­a­lysts say Sarep­ta was blind­sided by an FDA re­jec­tion dri­ven by reg­u­la­to­ry re­venge

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

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Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
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FDA de­ci­sion on Ver­tex's CF triple will come just ahead of planned CEO shake­up

Vertex has clinched a priority review for the all-important cystic fibrosis triple that will blaze the trail for treating a large group of patients unhelped by its current drugs.

FDA regulators have set a PDUFA date of March 19, 2020, just a year after the Boston biotech posted positive Phase III results showing that people with two F508del mutations experienced statistically significant improvements in lung function after a 4-week regimen of VX-445, tezacaftor and ivacaftor. After reviewing 24-week data among patients with one F508del mutation and one minimal function mutation — and thoroughly comparing the VX-445 triple with another combo featuring VX-659 on scores like safety, drug-drug interactions, and photosensitivity — Vertex ultimately went with VX-445.

An MIT spin­out kills one of its ‘liv­ing ther­a­peu­tics’ af­ter flunk­ing an ear­ly-stage study — shares rout­ed

Just a few weeks after bagging $80 million in a deal to collaborate with Gingko Bioworks on its special blend of engineered bacteria used for “living therapeutics,” little Synlogic in Boston $SYBX is tossing one of its two clinical programs after watching an early-stage study go down in defeat.

Their Phase Ib/IIa study for SYNB1020 to counter the accumulation of ammonia in the body, a condition called hyperammonemia or urea cycle disorder, floundered at the interim readout, forcing the biotech to kill it and reserve its cash for pipeline therapies with greater potential.

Elan­co to buy Bay­er's an­i­mal health busi­ness for $7.6B, as deal­mak­ing gath­ers steam in the sec­tor

Last week, Elanco explicitly dodged answering questions about its rumored interest in Bayer’s animal health business in its post-earnings call. On Tuesday, the Eli Lilly spinoff disclosed it was purchasing the German drug maker’s veterinary unit in a cash-and-stock deal worth $7.6 billion. 

Elanco $ELAN has been busy on the deal-making front. In April, it laid out plans to swallow its partner, Kansas-based pet therapeutics company Aratana $PETX. A July report by Reuters suggested a potential Bayer deal was being explored, and Bloomberg last week said the deal was imminent, citing sources.