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.

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

UK re-in­ves­ti­gates Pfiz­er's eye-pop­ping price goug­ing on an epilep­sy drug

When a drugmaker raises the price of a drug in the US by more than 2,000% overnight, and without any particular reason for that increase, nothing typically happens to the company. No fines, no court orders, just business as usual.

Martin Shkreli’s decades-old anti-parasitic drug Daraprim was the perfect example — massive price spike on an old drug, lots of media attention, public outcry, Congressional committees dragging his former company through multiple hearings, and at the end of it? Nothing happened to the price or the company (until generic competition came).

Thomas Lingelbach, Valneva CEO

A small vac­cine de­vel­op­er fa­vored by the UK gov­ern­ment in Covid-19 touts a PhI­II first in chikun­gun­ya

Before Valneva garnered the favor of the UK government as a potential supplier of Covid-19 vaccines, the French biotech prided itself on being the first company to bring a chikungunya vaccine into Phase III.

It now has positive pivotal results to back up the breakthrough therapy designation the FDA granted just weeks ago.

There are currently no approved jabs to prevent chikungunya virus infection despite decades of R&D efforts, a fact that underscores just how arduous traditional vaccine development can be, particularly for neglected tropical disease. In a absence of a major commercial market, the US government and NGOs such as CEPI have deployed various grants and incentives to spur on a small crew of academics and industry players, with Merck, via its acquisition of Themis, claiming a spot in that race.

Marianne De Backer (L) and Jeff Hatfield

Bay­er nabs star biotech Vi­vid­ion with a $2B buy­out and an ‘arms-length’ pact, pulling a part­ner out of the IPO con­ga line

Vividion is canceling that IPO it filed. Instead of following the industry-wide migration to Nasdaq, the biotech that has captured considerable attention for its still-preclinical work finding cryptic pockets to bind to on proteins is going to work for Bayer now.

The pharma giant is putting out word today that it has bought out Vividion for $1.5 billion in cash and another half-billion dollars in milestones.

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Josh Hoffman, outgoing Zymergen CEO (Zymergen)

UP­DAT­ED: Syn­bio uni­corn Zymer­gen jet­ti­sons found­ing CEO, cuts guid­ance as cus­tomers re­port lead prod­uct does­n't work

Zymergen, just months off a $500 million IPO that put the synthetic bio firm in rarified air, has now ejected its founding CEO and downgraded its revenue forecasts after customers reported its lead film product doesn’t work as advertised, the company said Tuesday afternoon.

CEO Josh Hoffman will leave his role and sacrifice his board seat immediately in favor of Jay Flatley, the former CEO of Illumina who will take the lead role on an interim basis as the company conducts a search for its next leader.

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Tadataka Yamada (Photographer: Kiyoshi Ota/Bloomberg via Getty Images)

Sci­ence pi­o­neer, phar­ma re­search chief, glob­al health ad­vo­cate and biotech en­tre­pre­neur Tadata­ka ‘Tachi’ Ya­ma­da has died

Tadataka Yamada, a towering physician-scientist who made his name in academia before transforming drug development at GlaxoSmithKline and developing vaccines for malaria and meningitis at the Gates Foundation, died unexpectedly of natural causes at his home in Seattle Wednesday morning.

He was 76. Frazier Healthcare Partners’ David Socks confirmed his death.

Known widely by the mononym “Tachi,” Yamada had a globetrotting career and arrived in industry relatively late in life. A 2004 Independent article noted GSK had asked Yamada to stay on beyond his approaching 60th birthday, the company’s usual retirement age. Yamada would continue working for the next 17 years, steering the Gates Foundation’s global health division for 6 years, funding Jim Wilson’s gene therapy work when few would touch it, launching Takeda Vaccines and co-founding a series of high-profile biotechs.

Zymergen co-founders Zach Serber, Josh Hoffman, and Jed Dean (Zymergen via website)

Zymer­gen's sud­den im­plo­sion shocked biotech. A lin­ger­ing loan could make things even worse

As former synbio unicorn Zymergen picks up the pieces from its spectacular implosion Tuesday, an outstanding loan from Perceptive Advisors — the only blue-chip biotech crossover investor to touch Zymergen’s fundraising efforts — could make the situation worse, according to public documents.

In December 2019, more than a year before Zymergen filed for what would eventually become a $500 million IPO, the “biofacturing” firm signed a $100 million credit facility with Perceptive to help supplement the nearly $700 million the company had raised across four VC rounds.

Lan Huang, BeyondSpring CEO

Shares of Be­yond­Spring sky­rock­et on new, pos­i­tive can­cer drug tri­al re­sults

Sometimes results come along that shock even a biotech’s believers.

On Wednesday, BeyondSpring, a small New York biotech with an offbeat approach to immunotherapy, announced its lead drug significantly extended non-small cell lung cancer patients’ lives in a large trial. Although the company did not release the exact survival data, it said that nearly twice as many patients were alive after two years on the drug arm than on the standard-of-care arm.

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Pfiz­er puts the pres­sure on Eli Lil­ly's JAK in­hibitor Olu­mi­ant with new da­ta in alope­cia area­ta

As Eli Lilly looks to secure a win for its blockbuster Olumiant in alopecia areata, going where no JAK inhibitor has gone before, Pfizer is coming up from behind with Phase IIb/III results suggesting its own candidate can help regrow hair lost due to the autoimmune disease.

On Wednesday, Pfizer unveiled topline results from the ALLEGRO trial, which enrolled 718 patients 12 years and older with alopecia areata, a condition that can cause sudden, severe and patchy hair loss. While the patients’ episodes of alopecia areata varied in length, they all had one thing in common: They had lost at least half the hair on their scalps.