Equi­l­li­um's im­muno-in­flam­ma­to­ry drug wins FDA's or­phan drug sta­tus; Van­da sues FDA for man­dat­ing lengthy dog study

→ San Diego-based Equi­l­li­um has nabbed an or­phan drug des­ig­na­tion for EQ001 on the verge of be­gin­ning a Phase Ib/II for the im­muno-in­flam­ma­to­ry drug. Al­so known as itolizum­ab, the drug was in-li­censed from In­dia’s Bio­con, which sells it in the coun­try as a pso­ri­a­sis treat­ment. Equi­l­li­um $EQ, how­ev­er, is fo­cus­ing its US clin­i­cal work on the pre­ven­tion and treat­ment of acute graft-ver­sus-host dis­ease — a strat­e­gy that has al­lowed it to skip fi­nanc­ing rounds and jump straight in­to an IPO.

→ Ear­li­er this week, Pain Ther­a­peu­tics $PTIE di­rect­ed its ire against the “FDA’s pur­port­ed sham­bol­ic reg­u­la­tions.” On the same day Van­da Phar­ma­ceu­ti­cals $VN­DA al­so fumed at the agency, say­ing the US health reg­u­la­tor had im­posed a non-bind­ing re­quire­ment that a nine-month non-ro­dent tox­i­c­i­ty study be con­duct­ed be­fore its drug, tradip­i­tant, can be stud­ied in hu­mans. The com­pa­ny is test­ing the drug in a 12-week dog study, but the agency is in­sist­ing on a 9-month study. When Van­da re­fused to con­duct this study, which usu­al­ly in­volves young bea­gles as the test sub­jects that must be sac­ri­ficed to per­mit eval­u­a­tion of the an­i­mal’s tis­sues, the FDA slapped a par­tial clin­i­cal hold on Van­da tri­als test­ing tradip­i­tant for longer than 12 weeks and did not pro­vide a jus­ti­fi­ca­tion for its de­ci­sion, the com­pa­ny said. Con­se­quent­ly, Van­da has sued the agency.

“The FDA is ig­nor­ing a large body of pub­lished sci­en­tif­ic ev­i­dence which con­cludes that these chron­ic dog stud­ies do not of­fer any ad­di­tion­al use­ful in­for­ma­tion. That pol­i­cy is based on old, out­dat­ed sci­ence and re­quires the killing of too many dogs with­out any sci­en­tif­i­cal­ly jus­ti­fied pur­pose. Yet, com­pa­nies have been ret­i­cent to stand up to the FDA and de­mand that it change its pol­i­cy. Van­da is un­will­ing to ac­cept the sta­tus quo,” com­pa­ny chief Mi­hael Poly­meropou­los said in a state­ment. The al­le­ga­tions by Van­da — which al­so is­sued an open let­ter to the FDA — come months af­ter the agency said it was work­ing on new reg­u­la­tions to re­duce dog test­ing in drug de­vel­op­ment.

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 help@endpointsnews.com with any issues.

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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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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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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. 

As­traZeneca's di­a­betes drug Farx­i­ga helps pa­tients with heart dis­ease and with­out di­a­betes in land­mark tri­al

Months ago, data on J&J’s $JNJ Invokana indicated the diabetes drug conferred cardiovascular (CV) benefit in patients who do and do not have preexisting CV disease. On Tuesday, AstraZeneca’s $AZN rival treatment, Farxiga, was shown to cut the risk of CV death or the worsening of heart failure in patients with heart disease, in a landmark trial.

The treatments, in addition to Jardiance from Eli Lilly $LLY, belong to a class of diabetes drugs called sodium-glucose co-transporter 2 (SGLT2) inhibitors, which work by curbing the absorption of glucose via the kidneys so that surplus glucose is excreted through urination.

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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