His way: Greg Ver­dine is build­ing a new kind of drug, with back­ing from a dif­fer­ent kind of in­vestor

Af­ter help­ing spear­head the launch of a long string of biotechs over the years from his pres­ti­gious perch at Har­vard, Greg Ver­dine is now de­vot­ing the bulk of his time to lead­ing the ef­fort on grow­ing two biotechs from scratch. And he’s just chalked up a $66 mil­lion round to move one of them — Fog­Phar­ma — in­to the clin­ic next year with a new tech­nol­o­gy he’s con­vinced can break its way in­to ther­a­peu­tic ter­ri­to­ries that have re­mained off lim­its to de­vel­op­ers up to now.

Fog­Phar­ma has been en­gi­neer­ing a new kind of ther­a­peu­tic class, one that com­bines the cell-pen­e­trat­ing ca­pac­i­ty of small mol­e­cules with the tar­get-en­gag­ing tenac­i­ty of bi­o­log­ics in­to what he calls CPMPs — cell pen­e­trat­ing minipro­teins. 

The big idea here — if you strip it all down to the chas­sis — is that Ver­dine and his crew have de­vel­oped a struc­tur­al “brace” that promis­es to make their polypep­tides ef­fec­tive against tough tar­gets like β-catenin. The brace — Ver­dine’s “se­cret sauce” — locks the struc­ture in place and amps up its abil­i­ty to pen­e­trate a cell, ze­ro in on the spe­cif­ic tar­get and main­tain a high lev­el of the drug in blood for a sus­tained pe­ri­od. And he says that each suc­ces­sive it­er­a­tion of their drugs in pre­clin­i­cal test­ing has proven bet­ter at the big job they’re de­signed for: drug­ging the un­drug­gable.

Leon Chen

This new mon­ey in the B round gives Fog­Phar­ma the op­por­tu­ni­ty to take its β-catenin pro­gram — which in­volves Wnt path­way ac­ti­va­tion — in­to a Phase I/II pro­gram in the sec­ond half of next year while lin­ing up an IND on a Cbl-b in­hibitor pro­gram, with a third undis­closed ef­fort com­ing up the line. The dis­cov­ery plat­form in­cludes “three ad­di­tion­al, dis­tinct and dif­fer­en­ti­at­ed forms of cell-pen­e­trat­ing minipro­teins.” 

“The en­tire fi­nanc­ing is three-and-a-half years worth of mon­ey,” Ver­dine tells me. Add that to the $11 mil­lion in Se­ries A cash raised at three sep­a­rate points be­gin­ning in ear­ly 2016, and Ver­dine has round­ed up a to­tal of $77 mil­lion for Fog­Phar­ma.

But it could have been more if he had need­ed it.

Ge Li

Much of the seed mon­ey for Fog­Phar­ma came from Deer­field and WuXi’s cor­po­rate fund, over­seen by Ge Li, a man with ac­cess to a vast amount of cap­i­tal around the world. That kind of fi­nanc­ing al­lowed Ver­dine to cre­ate Fog­Phar­ma with­out be­ing forced to adopt the short-term tac­tics of tra­di­tion­al VCs in the busi­ness.

“We built the com­pa­ny on a dif­fer­ent mod­el,” says Ver­dine. “I want­ed to do this one from soup to nuts. For that rea­son in the Se­ries A we didn’t turn to ven­ture in­vestors who would come in and have a sig­nif­i­cant role in build­ing the com­pa­ny.”

With­out even a hint of boast­ing, Ver­dine be­lieves that with his re­la­tion­ships among elite biotech in­vestors, at this point in his ca­reer of de­vel­op­ing new drugs, he could raise a bil­lion dol­lars if need­ed. There’s an ap­petite for tru­ly pi­o­neer­ing drug de­vel­op­ment work, says Ver­dine, that looks to vault ahead on crit­i­cal, break­through ther­a­pies.

He has the re­sume to back it up. And he’s built a plat­form com­pa­ny which has the ca­pac­i­ty to find and eval­u­ate new tar­gets and prospec­tive new ther­a­pies in a mat­ter of months.

Jeff Leerink

It’s no ac­ci­dent that Ver­dine’s lead pro­gram forms a junc­tion with check­point ther­a­pies; the β-catenin/Wnt path­way dis­rupts im­mune re­spons­es and an ef­fec­tive push here holds the promise of over­com­ing ini­tial re­sis­tance to im­munother­a­py as well as the de­vel­op­ment of drug re­sis­tance to an ef­fec­tive treat­ment. And it’s the first such ther­a­py to make it to the thresh­old of a clin­i­cal tri­al.

But Ver­dine is a proud pa­pa to mul­ti­ple pro­grams, and he is just as ex­cit­ed, if not more so, by the Cbl-b im­muno-on­col­o­gy drug that’s com­ing up be­hind the lead.

“What we’ve seen is that these mol­e­cules can be tol­er­a­ble at rel­a­tive­ly high dos­es, with a sig­nif­i­cant im­pact on tu­mor growth,” he says.

Kr­ish­na Yesh­want

Ver­dine has been a cel­e­brat­ed sci­en­tist in the field of drug dis­cov­ery for more years than many of his stu­dents have lived. Along the way, he’s earned some close ties to a new breed of biotech in­vestors who have now come in to back Fog­Phar­ma and well as his sec­ond ven­ture, LifeM­ine.

The sci­en­tist and se­r­i­al en­tre­pre­neur owes much of his suc­cess in rais­ing cash to close ties with Asian groups that have be­come ac­tive play­ers in biotech.

Rick Klaus­ner

Ver­dine’s ex­pe­ri­ence in Chi­na has led him to Boyu Cap­i­tal — an in­flu­en­tial pri­vate eq­ui­ty group led by co-founder and part­ner Sean Tong — and Blue Pool. The Chi­nese ven­ture group 6 Di­men­sions Cap­i­tal came in with oth­er new in­vestors, in­clud­ing Google’s GV, Hori­zons Ven­tures, Nan Fung Group and Leerink Part­ners. Deer­field Man­age­ment came back with Boyu Cap­i­tal, WuXi AppTec Cor­po­rate Ven­tures and “a promi­nent in­ter­na­tion­al group of non-in­sti­tu­tion­al in­vestors” to com­plete the syn­di­cate.

That kind of back­ing al­lowed Ver­dine to build his board with in­di­vid­u­als who are all in on his R&D strat­e­gy, with Leon Chen from 6 Di­men­sions, Leerink’s Jeff Leerink, GV gen­er­al part­ner Kr­ish­na Yesh­want and Rick Klaus­ner, a founder at Juno Ther­a­peu­tics, join­ing the group.

The new mon­ey will al­low Fog­Phar­ma to grow from 26 staffers to­day to north of 40 by the end of the year. And it’s still ear­ly days.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll