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.”

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Innoforce is off to the races at its new site in the city of Hangzhou, China.

The Chinese CDMO announced last week that it has started manufacturing at the new facility, which was built to offer process development and manufacturing operations for RNA, plasmid DNA, viral vectors and other cell therapeutics. It will also serve as Innoforce’s corporate HQ.

The company said it’s investing more than $200 million in the 550,000-square-foot manufacturing base for advanced therapies. The GMP manufacturing facility features space for producing plasmids with three 30-liter bioreactors. For viral vector manufacturing, Innoforce also has 200- and 500-liter bioreactors at its disposal, along with eight suites to make cell therapies. The site also includes several labs and warehouse spaces.

As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.

A biotech centered on treating fibrosis — born out of Mass General and Brigham and Women’s Hospital — has received a financial boost.

According to an SEC filing, the company has raised $31,761,186 in its latest funding round, which includes 17 investors. The filing lists six names attached to the company, including Meredith Fisher, a partner at Mass General Brigham Ventures and Mediar’s acting CEO.