Ax­o­vant races to PhI­II de­men­tia study as nelotanserin clears an ear­ly hur­dle, but ques­tions linger

Vivek Ra­maswamy

Ax­o­vant says that its drug nelotanserin has cleared an ear­ly hur­dle in a Phase II study for Lewy body de­men­tia, trig­ger­ing a de­ci­sion to ex­pand the mid-stage tri­al and hit­ting the green light for a Phase III to get start­ed lat­er this year. But in­vestors are like­ly to ques­tion a stum­ble on key sec­ondary goals.

A snap­shot of re­sults for the first tiny group of 11 pa­tients con­clud­ed that they were demon­strat­ing sta­tis­ti­cal­ly sig­nif­i­cant re­spons­es to the drug on the pri­ma­ry end­point. These pa­tients in­clud­ed peo­ple suf­fer­ing from Parkin­son’s as well as Lewy body de­men­tia. They were re­cruit­ed af­ter suf­fer­ing from fre­quent vi­su­al hal­lu­ci­na­tions.

“The pri­ma­ry out­come mea­sures of the study were ex­trapyra­mi­dal signs as as­sessed by Uni­fied Parkin­son’s Dis­ease Rat­ing Scale (UP­DRS) Parts II + III and safe­ty,” the com­pa­ny re­port­ed.

There was a big hitch, though. The tri­al did not hit sec­ondary end­points laid out by in­ves­ti­ga­tors to track the fre­quen­cy of vi­su­al hal­lu­ci­na­tions, in­clud­ing both a stan­dard SAPS as­sess­ment as well as an end­point they cre­at­ed in­ter­nal­ly at Ax­o­vant. And that may have weighed in on in­vestors’ re­sponse to the re­sults, with Ax­o­vant’s stock up on­ly 5% in pre-mar­ket trad­ing.

Ax­o­vant, the first of Vivek Ra­maswamy’s biotechs to get start­ed, plans to hus­tle in­to the ex­pand­ed Phase II for the drug — a 5HT2A an­tag­o­nist — as it wastes no time in get­ting the Phase III up and run­ning. Ra­maswamy has made no se­cret of his plans to ac­cel­er­ate R&D at his com­pa­nies. And Ax­o­vant is the most ad­vanced with a big tri­al un­der­way for Alzheimer’s.

Ax­o­vant al­ready faces an es­tab­lished mar­ket play­er. Aca­dia’s ri­val drug Nu­plazid won FDA ap­proval for Parkin­son’s de­men­tia last spring, but on­ly af­ter reg­u­la­tors ex­pressed their safe­ty con­cerns, not­ing a dis­tinct in­crease in the num­ber of deaths as well as the rate of ad­verse events among the pa­tients tak­ing the drug com­pared to the con­trol arm of the study — even if there was no ob­vi­ous clue what was trig­ger­ing those events.

Ax­o­vant’s hope is that it can come out of clin­i­cal tri­als with a bet­ter suc­cess rate and a clean­er safe­ty pro­file as both com­pa­nies tar­get a broad pa­tient pop­u­la­tion suf­fer­ing from dif­fer­ent forms of de­men­tia.

“I am in­trigued by the ben­e­fits ob­served on the UP­DRS in this study,” stat­ed Dr. James Lev­erenz, Chair, Sci­en­tif­ic Ad­vi­so­ry Coun­cil of the Lewy Body De­men­tia As­so­ci­a­tion and Di­rec­tor of the Cleve­land Lou Ru­vo Cen­ter for Brain Health at the Cleve­land Clin­ic. “If a sin­gle drug could si­mul­ta­ne­ous­ly ad­dress the mo­tor and neu­ropsy­chi­atric symp­toms of Lewy body de­men­tia, which the pub­lished lit­er­a­ture sug­gests may be the case for 5HT2A an­tag­o­nists, it would rep­re­sent a unique and im­por­tant ad­vance for the treat­ment of this con­di­tion. I look for­ward to re­view­ing the full dataset lat­er this year.”

Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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Covid-19 roundup: J&J, BAR­DA set ear­ly 2021 fin­ish line for $1B vac­cine race; FDA al­lows emer­gency drug use, ahead of piv­otal da­ta

J&J has zeroed in on a Covid-19 vaccine candidate that it hopes to begin testing in humans by September this year — with the extraordinary goal of getting it ready for emergency use in early 2021. And together with BARDA, it’s committing $1 billion to make it happen.

That kind of accelerated timeline would fall on the fast side of NIAID director Anthony Fauci’s well-publicized prediction that it would be another 12 to 18 months before a vaccine can be available for public use. A Phase I trial of Moderna’s mRNA vaccine began two weeks ago, and both the biotech and fellow mRNA player CureVac have discussed similar, if not even faster, timelines for emergency use among healthcare workers.

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As­traZeneca says its block­buster Farx­i­ga proved to be a game-chang­er in CKD — wrap­ping PhI­II ear­ly

If the FDA can still hold up its end of the bargain, AstraZeneca is already on a short path to scooping up a cutting-edge win with a likely approval for their SGLT2 drug Farxiga in cutting the risk of heart failure. Now the pharma giant says it can point to solid evidence that the drug — initially restricted to diabetes — also works for chronic kidney disease, potentially adding a blockbuster indication for the franchise.

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It is 'kind of a proven tech­nol­o­gy': Hep B vac­cine mak­er joins glob­al hunt for coro­n­avirus vac­cine

Using lab-grown proteins that are engineered to mimic the architecture of viruses to induce an immune response, VBI Vaccines is joining the hunt for a coronavirus vaccine — harnessing technology that has initially been proved safe in early trials as a prophylactic for cytomegalovirus (CMV) infection.

Unlike the raft of the companies in the Covid-19 vaccine race — including Moderna, CureVac and J&J — VBI is taking a pan-coronavirus approach, by developing a vaccine that will encompass Covid-19, severe acute respiratory syndrome (SARS), and Middle East respiratory syndrome (MERS).

Can a pair of top AveX­is alum­ni steer a new gene ther­a­py up­start to R&D glo­ry? 3 VCs bet $60M on it

VCs love few things more than a proven executive team when it comes to launching a new company. And now a group of A-listers has turned to a pair of top execs out of AveXis to steer the latest gene therapy player into the clinic.

The biotech is Waltham, MA-based Affinia and the two execs are Sean Nolan and Rick Modi — the former CEO and CBO respectively of AveXis, the gene therapy pioneer that fetched $8.7 billion in a sale to Novartis. Nolan has now taken the chairman’s role at Affinia while Modi moves up to the CEO post at the company.

Un­de­terred by a pan­dem­ic, Gilde Health­care rais­es their largest fund yet

When Pieter van der Meer started raising the capital for Gilde Healthcare’s fifth fund in the waning months of 2019, he had his eyes on a different chain of events that could change the healthcare system and perhaps even play to his firm’s advantage: The US presidential election.

Since raising their third fund in 2011, the 34-year-old Dutch firm had focused on value-based care. They chose late-stage biotechs that came up with new devices and delivery systems for de-risked established compounds, and when they chose preclinical biotechs, they spoke with potential pharma partners, payers and regulators to ask where and at what prices the drug made sense. As the Democratic primary became a contest over how to lower healthcare costs, it looked like a strategy that could pay off.

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Gilead CEO Dan O'­Day of­fers a de­tailed ex­pla­na­tion on remde­sivir ac­cess — re­as­sur­ing an­a­lysts that Covid-19 da­ta are com­ing fast

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDA’s orphan status for remdesivir — reserved to encourage the development of rare disease therapies — Gilead CEO Daniel O’Day had some explaining to do about the company’s approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program — one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

In addition to trying to reassure patients that they will once again have an avenue to pursue access, O’Day also reassured some analysts who had been fretting that China’s quick comeback from the coronavirus outbreak could derail its ultra-fast schedule for testing the drug in patients. The data are still expected in a few weeks, he says in the letter, putting the readout in April.

O’Day emphasizes that Gilead intends to pursue a pricing approach that will make this drug widely available — if it proves effective and safe. But no one is quite sure just what the longterm value would be, given the work being done on a variety of vaccines that may be rolled out as early as this fall — at least to the most heavily threatened groups.

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