Tien Lee, Aardvark Therapeutics CEO

Emerg­ing from stealth mode, Aard­vark rounds up enough cash to put its lead drug through Prad­er-Willi PhII

When Aard­vark Ther­a­peu­tics CEO Tien Lee start­ed his work on the biotech’s lead can­di­date, ap­petite sup­pres­sion was the goal for the small mol­e­cule.  Soon af­ter, his team start­ed to see added ben­e­fits with low­er blood glu­cose lev­els and an­ti-in­flam­ma­to­ry ac­tiv­i­ty. On the tail end of that, the com­pa­ny has emerged from stealth mode and an­nounced to­day that they’ve raised enough cash in the B round to cov­er mid-stage de­vel­op­ment work.

San Diego-based Aard­vark has se­cured $29 mil­lion in Se­ries B fi­nanc­ing. The mon­ey will be used to com­plete three Phase II tri­als of its lead com­pound ARD-101, a small mol­e­cule bit­ter taste re­cep­tor pan-ag­o­nist.  The funds will al­so be used to ad­vance ad­di­tion­al for­mu­la­tions for the can­di­date.

The fundrais­ing was led by Sor­ren­to Ther­a­peu­tics and fea­tured par­tic­i­pa­tion from Vick­ers Ven­ture Part­ners, Pre­mier Part­ners, BNH In­vest­ment, and Ko­rea Omega. The Foun­da­tion for Prad­er-Willi Re­search par­tic­i­pat­ed as well, which is key be­cause a Phase II tri­al in pa­tients with Prad­er-Willi Syn­drome is set to kick off lat­er this year. PWS is a ge­net­ic dis­or­der that leads pa­tients to be­come con­stant­ly hun­gry. That can of­ten lead to obe­si­ty and type 2 di­a­betes.

“I think we’re re­al­ly on to some­thing,” Lee said in a call with End­points News Wednes­day. “There are a few com­pa­nies that have looked in­to this space be­fore but no one has re­al­ly delved in and tried to ex­ploit this gut-brain path­way us­ing bit­ter taste re­cep­tors.”

ARD-101 has shown pos­i­tive ef­fects against obe­si­ty, hy­per­pha­gia, di­a­betes, hy­per­lipi­demia and in­flam­ma­tion in an­i­mal mod­els so far.

The drug uti­lizes bit­ter taste re­cep­tors, which Lee says are not on­ly in the mouth, but all over the body, and act as na­ture’s way of pro­tect­ing you against tox­ins. Over 99% of the drug is re­tained in the gut, which nor­mal­ly could be a bad thing for small mol­e­cules, but in this case, it ac­ti­vates en­teroen­docrine cells in the gut. When giv­en through an IV, the can­di­date doesn’t work the same, Lee said.

Aard­vark raised $10 mil­lion dur­ing its Se­ries A fundrais­ing two years ago, and that mon­ey was used to bring the can­di­date to in-hu­man tri­als. While the $29 mil­lion will more than cov­er the com­ple­tion of three Phase II tri­al, there is a lot more op­por­tu­ni­ty on the hori­zon, Lee said. The in­tent is to de­vel­op be­yond PWS in the long term, but for now it’s fo­cused on tak­ing a shot at an ac­cel­er­at­ed ap­proval for the ge­net­ic dis­ease — a goal that has elud­ed oth­ers.

“The main qual­i­ty of life im­pair­ment for Prad­er-Willi Syn­drome is an un­abat­ed ap­petite, says the CEO. “These kids, if giv­en un­re­strict­ed food ac­cess, they eat to the point of stom­ach rup­ture some­times. We have a lot of di­a­betes and obe­si­ty re­lat­ed com­pli­ca­tions in young adult­hood, and the fact that we’re hit­ting a va­ri­ety of hor­mones, we think we’re able to shut down ap­petite in a very unique way. We’ll see. Bi­ol­o­gy is hard and we’re not mak­ing any promis­es, but we’re def­i­nite­ly very hope­ful that we can see a dif­fer­ence for these kids.”

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; and more

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For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

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A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

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Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

Rob Califf ad­vances as Biden's FDA nom­i­nee, with a close com­mit­tee vote

Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

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CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

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‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

A patient in Alaska receiving an antibody infusion to prevent Covid hospitalizations in September. All but one of these treatments has been rendered useless by Omicron (Rick Bowmer/AP Images)

How a tiny Swiss lab and two old blood sam­ples cre­at­ed one of the on­ly ef­fec­tive drugs against Omi­cron (and why we have so lit­tle of it)

Exactly a decade before a novel coronavirus broke out in Wuhan, Davide Corti — a newly-minted immunologist with frameless glasses and a quick laugh — walked into a cramped lab on the top floor of an office building two hours outside Zurich. He had only enough money for two technicians and the ceiling was so low in parts that short stature was a job requirement, but Corti believed it’d be enough to test an idea he thought could change medicine.

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