Named for the Ital­ian term in­di­cat­ing a piece of mu­sic should be played at an up­beat and live­ly tem­po, Cal­i­for­nia-based Vi­vace Ther­a­peu­tics seems to have lived up to its name­sake.

In the mere five years the small-mol­e­cule drug dis­cov­ery and de­vel­op­ment com­pa­ny has been in busi­ness, its sci­en­tists and re­searchers have de­vot­ed sig­nif­i­cant amounts of time and mon­ey to­ward ther­a­peu­tics that tar­get a path­way in the body that can cause can­cer­ous tu­mors to de­vel­op if cer­tain pro­teins are ac­ti­vat­ed. Now, with $30 mil­lion in Se­ries C fi­nanc­ing, Vi­vace is aim­ing to test one of its can­di­dates against that path­way start­ing in ear­ly 2021.

The pro­teins, called YAP, in­vade cells through what’s called the Hip­po path­way — a sig­nal­ing sys­tem in the body that con­trols tis­sue re­gen­er­a­tion and tells or­gans when it’s time to stop grow­ing. If ac­ti­vat­ed, YAP can dis­rupt the path­way in a way that caus­es the can­cer­ous tu­mors to form.

CEO Sofie Qiao told End­points News that ex­am­in­ing the re­la­tion­ship be­tween YAP and the Hip­po path­way was the rea­son she found­ed Vi­vace in 2015 — it’s a nov­el re­la­tion­ship, es­pe­cial­ly in terms of its con­nec­tion to can­cer. For the first two years, Qiao said, the com­pa­ny op­er­at­ed in stealth mode, but in May 2017, Vi­vace raised a $25 mil­lion Se­ries B to be­come a com­mer­cial-fo­cused on­col­o­gy com­pa­ny.

Now, the work to un­der­stand and po­ten­tial­ly treat a pro­tein mu­ta­tion that may cause lung, gas­tric, colon, cer­vi­cal, ovar­i­an, breast, melanoma, he­pa­to­cel­lu­lar car­ci­no­ma and squa­mous cell car­ci­no­ma can­cers has paid off in the biggest way yet for Qiao and Vi­vace with the lat­est $30 mil­lion round.

“Most of the on­col­o­gy play­ers in our in­dus­try have been watch­ing this path­way for quite a few years, and many of them have proac­tive­ly ap­proached us for dis­cus­sion since we do have a pub­lic pres­ence,” Qiao said. “To the best of our knowl­edge, no one has sent any mol­e­cule to the clin­ic, so we are po­si­tioned to be the first com­pa­ny to test a mol­e­cule tar­get­ing this path­way in hu­man set­ting.”

No­table in­vestors for this round in­clude: Canaan Part­ners, WuXi Health­care Ven­tures, Cen­o­va Cap­i­tal, Se­quoia Cap­i­tal Chi­na, Box­er Cap­i­tal and RA Cap­i­tal Man­age­ment.

Qiao said near­ly all of the $30 mil­lion would be put in­to the hu­man clin­i­cal de­vel­op­ment process of Vi­vace’s drug can­di­date. The ini­tial process­es will tar­get tu­mors that are known to be en­tire­ly de­pen­dent on ac­ti­vat­ed YAP, and Vi­vace said in a news re­lease that pre-clin­i­cal R&D showed that its clin­i­cal can­di­date is ac­tive as a monother­a­py and in com­bi­na­tion with oth­er an­ti-can­cer ther­a­pies against tu­mors that re­ly up­on dys­func­tion of the Hip­po path­way.

Len Post, Vi­vace’s chief sci­en­tif­ic of­fi­cer, said in an in­ter­view that un­til now, no sci­en­tif­ic progress had re­al­ly been made on how to dis­rupt the Hip­po path­way if YAP pro­teins were ac­ti­vat­ed. Vi­vace’s work, he said, dis­cov­ered how cer­tain com­pounds work in the con­text of the path­way and what has to hap­pen to in­hib­it the path­way from form­ing can­cer­ous tu­mors.

For YAP pro­teins to ac­ti­vate, they have to find an­oth­er pro­tein called a tran­scrip­tion­al en­hanced as­so­ciate do­main. Vi­vace’s com­pounds block those TEAD pro­teins by pre­vent­ing the ad­di­tion of a fat­ty acid that would al­low the two pro­teins to bond.

The $30 mil­lion round brings Vi­vace’s to­tal in­vest­ment haul just since 2017 to rough­ly $70 mil­lion. Qiao said that those fig­ures in­di­cate a clear in­ter­est from the biotech in­vest­ment com­mu­ni­ty in the Hip­po path­way and its po­ten­tial im­pact on the field of on­col­o­gy.

“That shows how much en­thu­si­asm and in­ter­est the in­vestor com­mu­ni­ty has for this path­way,” she said. “I think they re­al­ly like our da­ta and our team and the po­ten­tial of our mol­e­cule re­al­ly gen­er­at­ing clin­i­cal ef­fi­ca­cy.”

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.

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.