Af­ter a short stint at In­ter­cept, Rachel McMinn makes the leap from biotech an­a­lyst to biotech CEO

Over a 13-year ca­reer as a biotech an­a­lyst at Piper Jaf­fray, Cowen and Bank of Amer­i­ca Mer­rill Lynch, Rachel McMinn learned a lot about the in­dus­try. Enough to think that maybe she should be the one run­ning the com­pa­ny rather than just ask­ing ques­tions — and dis­cussing op­tions — re­lat­ed to its per­for­mance.

“Over time I felt I was giv­ing a lot of com­pa­nies free ad­vice,” McMinn says. “I felt that maybe I could do it.”

Rachel McMinn

But she didn’t feel pre­pared to make the leap in one jump. In­stead, she com­plet­ed a 3.5-year stint as chief busi­ness and strat­e­gy of­fi­cer at In­ter­cept, where she was able to plunge di­rect­ly in­to the nit­ty grit­ty of biotech busi­ness.  (Af­ter she once gave the com­pa­ny $ICPT an, er, bull­ish $872 price ob­jec­tive on the stock. But she al­so had plen­ty of suc­cess­es to boast about.) And to­day she’s for­mal­ly launch­ing a new biotech of her own af­ter an im­pres­sive $68.5 mil­lion Se­ries A raise.

The broad out­lines of the com­pa­ny — Neu­ro­gene, tak­ing its place in the bur­geon­ing biotech hub of New York — will sound fa­mil­iar to any­one who’s been ob­serv­ing the biotech scene over the past decade. 

McMinn and the 20 staffers at the biotech are tack­ling rare neu­ro­log­i­cal con­di­tions with a pipeline of gene ther­a­pies us­ing AAV tech. Just a few days ago the com­pa­ny out­lined mouse da­ta from a pre­clin­i­cal study of a gene ther­a­py out of UT South­west­ern for AGU, or as­partyl­glu­cosamin­uria, a neu­rode­gen­er­a­tive dis­ease trig­gered by a rogue AGA gene. An­oth­er re­searcher from The Jack­son Lab out­lined re­sults from a mouse study of Neu­ro­gene’s pro­gram for Char­cot-Marie-Tooth dis­ease, type 4J (CMT4J) syn­drome.

Those aren’t the on­ly 2 gene ther­a­pies they’re work­ing on, McMinn tells me. Oth­ers are in the works as well. And aside from an an­nounce­ment on her new chief sci­en­tif­ic of­fi­cer, the Uni­ver­si­ty of Ed­in­burgh’s Stu­art Cobb, the new­ly mint­ed CEO starts to put the brakes on the specifics.

Not un­usu­al­ly, Neu­ro­gene wants to keep the unique as­pects of their work un­der wraps for awhile. The key to mak­ing the lat­est gene ther­a­py com­pa­ny to hit the scene a suc­cess, she adds, will re­volve around some of the new tech they’re fo­cused on re­lat­ed to vec­tors and genes. Some of these healthy genes they’re try­ing to load up for de­liv­ery aren’t easy to get in­to the tiny ve­hi­cles. And get­ting the dose and de­liv­ery right will be es­sen­tial to tack­ling the rare con­di­tions on their radar.

But the de­tails will have to wait. So will time­lines. McMinn is not in­ter­est­ed at this time in broad­cast­ing sched­ules for mov­ing in­to the clin­ic. How long is their run­way? Time will tell.

Why rare neu­ro­log­i­cal con­di­tions? 

McMinn traces her in­spi­ra­tion to Avex­is and the re­mark­able da­ta they put to­geth­er for SMA, be­fore No­var­tis bought them out. And there’s a fam­i­ly tie as well.

“I re­al­ly want­ed to re­vis­it the idea of do­ing some­thing that was mean­ing­ful for me per­son­al­ly,” says McMinn, cit­ing an old­er, autis­tic broth­er. 

What’s the goal? To build a gene ther­a­py com­pa­ny that can steer one new prod­uct to the mar­ket every year, McMinn replies.

She’s set a high mark for Neu­ro­gene. Now they be­gin.

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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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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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

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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SEC calls out lit­tle Ther­a­peu­tic­sMD for its in­sid­er con­tacts with an­a­lysts to boost share price, then halt rout

Back in May 2017, following an FDA rejection, TherapeuticsMD saw its share price plummet to the lowest levels in two years. The little Florida biotech eventually found its way back to the good side of regulators, scoring a curious OK a year later for its therapy preventing vaginal pain during sex. But the SEC is now accusing it of selectively disclosing nonpublic information in attempts to manipulate its stock.

In two instances in June and July of 2017, TherapeuticsMD allegedly violated the Regulation Fair Disclosure rule by sharing material information with certain sell-side analysts and not the public, resulting in a more favorable stock move than otherwise would be expected.

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Therapists Marcela Ot'alora and Bruce Poulter are trained to conduct MDMA-assisted psychotherapy. In this reenactment, they demonstrate how they help guide and watch over a patient who is revisiting traumatic memories while under the influence of MDMA. (Photo: Multidisciplinary Association for Psychedelic Studies)

MD­MA, now in Phase III, shows promise as a PTSD treat­ment

The first time Lori Tipton tried MDMA, she was skeptical it would make a difference.

“I really was, at the beginning, very nervous,” Tipton said.

MDMA is the main ingredient in the club drug known as ecstasy or molly. But Tipton wasn’t taking pills sold on the street to get high. She was trying to treat her post-traumatic stress disorder by participating in a clinical trial.

After taking a dose of pure MDMA, Tipton lay in a quiet room with two specially trained psychotherapists. They sat next to her as she recalled some of her deepest traumas, such as discovering her mother’s body after Tipton’s mother killed two people and then herself in a murder-suicide.

Ted Ashburn. Oncorus

Cowen, Per­cep­tive lead $79.5M Se­ries B for 's­tand­out' biotech shep­herd­ing on­colyt­ic virus to clin­ic

As several Big Pharma players secure biotech partners in the oncolytic virus space for new immuno-oncology combos, Cowen and Perceptive Advisors have come out with their own bet on a startup that promises to shine.

The marquee investors are joining MPM, Deerfield, Celgene, Astellas, Arkin Bio Ventures and UBS Oncology Impact Fund in backing the drug developer, Oncorus, which will now deploy the $79.5 million in Series B cash toward clinical development of its lead program. Other new investors include Surveyor Capital, Sphera Funds, IMM Investment, QUAD Investment Management, UTC Investment, SV Investment Corp and Shinhan Investment-Private Equity, the last five of which are Korean-based funds.

Fu­til­i­ty analy­sis au­gurs de­feat in piv­otal tri­al test­ing of Nu­Cana's lead drug in metasta­t­ic pan­cre­at­ic can­cer

Nearly two years after making its public debut, UK-based NuCana’s mission to make chemotherapies more potent and safer was dealt a blow, after a pivotal study testing its lead experimental drug halted enrollment in a hard-to-treat advanced form of cancer, following a futility analysis.

The drug, Acelarin, is being evaluated for use in metastatic pancreatic cancer patients who were not considered suitable for combination chemotherapy. In the late-stage ACELARATE study — which compared the experimental drug against the chemotherapy gemcitabine — 200 patients had been enrolled by the sponsor, Clatterbridge Cancer Centre, before an analysis from an independent safety and data monitoring panel suggested the study’s main goal would not be met.