Here's why Su­cam­po paid $200M for its PhI­II Nie­mann-Pick drug

In­ves­ti­ga­tors have out­lined the re­sults of a small, sin­gle-arm study of a new drug for Nie­mann-Pick type C dis­ease which in­spired a $200 mil­lion biotech buy­out ear­li­er this year.

The drug is cy­clodex­trin, or at least a unique mix­ture of 2-hy­drox­ypropyl-ß- cy­clodex­trins (HPßCD) that Vtesse had moved in­to a piv­otal study af­ter glean­ing pos­i­tive da­ta from a study with 14 pa­tients. Su­cam­po bagged the biotech and its drug for $200 mil­lion in April, tak­ing over con­trol of the Phase III.

Daniel Ory

Ac­cord­ing to Daniel Ory of Wash­ing­ton Uni­ver­si­ty School of Med­i­cine in St Louis — the first au­thor of a new pa­per pub­lished in The Lancet — re­searchers re­cruit­ed 14 NPC pa­tients, who typ­i­cal­ly die of the dis­ease 10 to 15 years af­ter it’s di­ag­nosed.

De­liv­ered in the spinal col­umn every two weeks for 18 months, the symp­toms of 7 of the 14 pa­tients im­proved, with some re­gain­ing an abil­i­ty to speak as re­searchers tracked scores on gait, cog­ni­tion and speech.

To be sure, this is no cure. The re­searchers say that the scores of pa­tients for the point sys­tem they used wors­ened by 1.2 per year on av­er­age. But that com­pares to the his­tor­i­cal da­ta they used from an ear­li­er study, as there was no place­bo arm, with an ex­pect­ed 2.9 point de­cline. And in­ves­ti­ga­tors say that they tracked hear­ing loss as a se­ri­ous ad­verse event in the study.

“Some of the pa­tients be­gan this tri­al with­out the abil­i­ty to speak, and now they speak,” Ory said. “There is a slow­ing of the de­cline, but we were sur­prised to see trends to­ward im­prove­ment in a few cat­e­gories. Com­pared with the his­tor­i­cal da­ta, half of the pa­tients in this study saw an im­prove­ment or no wors­en­ing in the neu­ro­log­i­cal sever­i­ty score.”

Added Ory: “A ther­a­py that caus­es hear­ing loss is not ide­al. But since the dis­ease it­self caus­es hear­ing loss, we felt that this side ef­fect may be a rea­son­able trade-off, giv­en the al­ter­na­tive de­cline and death that the dis­ease al­so caus­es.”

Re­searchers at the NIH’s Na­tion­al In­sti­tute of Child Health and Hu­man De­vel­op­ment de­signed and con­duct­ed the study. And they say that they were al­so able to track a pos­i­tive in­flu­ence on sev­er­al key bio­mark­ers for the dis­ease, which is caused by the buildup of cho­les­terol in brain cells.

RWE chal­lenges for to­day's bio­phar­ma

The rapid development of technology — and the resulting avalanche of data — are catalysts for significant change in the biopharmaceutical industry. This translates into urgent pressures for today’s biopharma, including a need to quickly and affordably develop products with proven therapeutic efficacy and value. This urgency is expedited by the growth of value-based contracting, where access to reimbursement and profit depends on these abilities.

UP­DAT­ED: In a stun­ning turn­around, Bio­gen says that ad­u­canum­ab does work for Alzheimer's — but da­ta min­ing in­cites con­tro­ver­sy and ques­tions

Biogen has confounded the biotech world one more time.

In a stunning about-face, the company and its partners at Eisai say that a new analysis of a larger dataset on aducanumab has restored its faith in the drug as a game-changer for Alzheimer’s and, after talking it over with the FDA, they’ll now be filing for an approval of a drug that had been given up for dead.

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As shares suf­fer from a lin­ger­ing slump, a bruised Alk­er­mes slash­es 160 jobs in R&D re­struc­tur­ing

With its share price in a deep slump after suffering through a regulatory debacle over their depression drug ALKS 5461, Alkermes CEO Richard Pops is taking the ax to its R&D organization in a restructuring aimed at cutting costs ahead of its next attempt at a rollout in a tough field.

Richard Pops, Endpoints via Youtube

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Acor­da's Ron Co­hen brings the ax back out as new drug sales on­ly trick­le in while cash cow is led to the slaugh­ter

With its new drug earning meager sums and its one-time cash cow reduced to a bony shadow of its former self, Acorda Therapeutics today is rolling out a new restructuring aimed at slashing the staff and cutting costs to get through the hard times ahead.

The biotech is chopping a quarter of its staff today, carving back R&D as well as SG&A expenses. And CEO Ron Cohen is cutting deep.

Under the new austerity budget, Acorda’s R&D expenses for the full year 2019 are expected to be $55 – $60 million, reduced from $70 – $80 million. SG&A expenses for the full year 2019 are expected to be $185 – $190 million, reduced from $200 – $210 million. R&D expenses for the full year 2020 are expected to be $20 – $25 million and SG&A
expenses for the full year 2020 are expected to be $160 – $165 million.

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RAPT Ther­a­peu­tics re­turns to Wall Street to re­vive IPO bid

On May 24, FLX Bio, a small cancer and inflammation biotech with backing from GV, changed its name to RAPT Therapeutics and filed confidentially for an IPO. On July 5th, they filed to raise up to $86 million. On July 22, they announced the IPO with a $75 million goal.  And on August 1, they abruptly and without explanation called it all off.

Now, without explanation, they’re reviving the bid, filing again for a $75 million IPO, this time with a new bookrunner and a new drug candidate in the clinic. The terms will be the same: 5 million shares at $14-$16 per share. It would give them a diluted market value of $351 million.

EY vet set to re­place re­tir­ing Am­gen CFO Meline

Ahead of its third-quarter results next week, Amgen on Tuesday disclosed the planned retirement of David Meline, who has served as the company’s chief financial officer since 2014.

Meline will be replaced by Ernst & Young vet, Peter Griffith, as CFO come January 1, 2020 — but until then Griffith will serve as executive vice president, finance.

“Over the last 5 years at Amgen, Meline instituted many major changes that led to operational efficiencies and margin expansion while successfully returning cash to shareholders. Now that Amgen is on solid footing, it was a good time to step away,” Cowen’s Yaron Werber wrote in a note. “We do not anticipate any major changes to strategy or operations immediately due to this transition as Amgen is on solid footing.”

Eli Lil­ly’s USA, di­a­betes chief En­rique Con­ter­no is head­ing out af­ter 27 years, and he’s be­ing re­placed by a com­pa­ny in­sid­er

Close to 3 years after Eli Lilly CEO Dave Ricks added the title of president of the US operations to Enrique Conterno’s resume, which included his helmsmanship of the diabetes franchise, the Peruvian born exec is set to retire after a 27-year run at the pharma giant.

Lilly put out the news just as it was posting Q3 results, with a mix of upbeat and downbeat results in the latest set of numbers from Lilly.
Conterno — a grizzled, deeply experienced and sometimes gruff veteran of the pharma world — was a high-profile figure at Lilly, stepping up to expanded duties as the company was forced to deal with intense pricing pressure on the diabetes side of the business. He had replaced outgoing US president Alex Azar, who later popped up as head of Health and Human Services in the Trump administration.
As head of the diabetes unit, Conterno had to deal with an extraordinarily competitive field as payers demanded bigger discounts. Trulicity’s success helped generate new revenue for the company, but Q3’s miss on revenue had a lot to do with the need for discounting the drug ahead of Novo Nordisk’s rival therapy, Rybelsus, which was priced on the wholesale level at an almost identical rate.

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No­var­tis hands off $80M in cash to part­ner up with a top biotech play­er in the fi­bro­sis sec­tor

Never underestimate the power of a good showing at a scientific conference.
In a presentation late last year, the researchers at Pliant Therapeutics launched a series of discussions about the preclinical data they were pulling together around their work on their small-molecule integrin inhibitor aimed at transforming growth factor beta, or TGF-β, a key pathway involved in fibrosis.
And they got some serious attention for the work.
“We got interest from pharma partners and at the end Novartis basically made it,” says Pliant CEO Bernard Coulie.

Vas Narasimhan. Getty Images

UP­DAT­ED: Failed PhI­II fe­vip­iprant tri­als pour more cold wa­ter on No­var­tis' block­buster R&D en­gine — and briefly spread the chill to a high-pro­file biotech

Back in July, during an investor call where Novartis execs ran through an upbeat assessment of their Q2 performance, CEO Vas Narasimhan and development chief John Tsai were pressed to predict which of the two looming Phase III readouts — involving cardio drug Entresto and asthma therapy fevipiprant, respectively — had a higher likelihood of success. Tsai gave the PARAGON-HF study with Entresto minimally better odds, but Narasimhan emphasized that their strategy of giving fevipiprant to more severe patients gave them confidence.

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