The End­points 11: A group of dis­rup­tive up­starts on a do-or-die mis­sion to launch new meds

Over the last 15 years I’ve had the chance to help select about 200 private biotechs for up-and-coming awards like this. Looking over the rank and file of this disruptive crowd, I had my standouts, my OK borderline selections, and some truly dreadful, cringe-worthy choices.

Such is the game of judging private biotechs, where you always wind up making hunches based on an incomplete picture. But then that’s also much of the fun, right?

Everyone who does this sort of thing likes to pretend that they can pick which of these fledglings can shoot the rapids of drug development and come out of the white water doing high fives. But the reality is that we all have our good and bad ideas.

And you learn along the way.

With my first selection of the Endpoints 11 (complete with a neat logo conceptualized by our creative assistant editor Amber Tong), I’m getting started on generation 2.0 of my idea of top companies that just may be headed for greatness.

There are several key attributes that characterize each of the E11, and help me hedge my bets. Each represents an important trend in biotech creation.

Most have top teams that are well recognized for earlier successes. Experienced biotech execs these days can generally have their pick of the litter when it comes to new companies angling for a launch. So when you see a prominent biotech exec make the transition out of incubation and onto the stage — often alongside close associates that they have known and worked with for years — it may not guarantee a winner, but it sure is comforting when smart, successful people love the science behind a startup.

That will serve as my segue into technology. Me-too drugs have been discredited for years now. Payers may use them to pick the lock on lower prices, but it’s a woeful development strategy. Every company in this year’s maiden E11 is swinging for the fences, looking to drug the undruggable or race with ambitious rivals to achieve something remarkable.

So scientific ambition is key.

Enough money to get through to the next stage of human data is critical.

There is a healthy debate going on right now whether the 4-year tidal wave of investor cash coursing through the industry is essentially causing risky behavior that will squander cash. Given the inherent risks associated with drug development, and the groundbreaking nature of what they’re trying to achieve, a good chunk of that investment money is going up in flames — under the best of circumstances.

If anyone in the E11 fail, it likely won’t be because they were starved for cash. And this business isn’t cheap. Also, if any of these companies below go belly up, you will hear the explosion from halfway around the world.

The right partner can be everything in this business, helping make all the difference in picking up speed in the clinic and providing the kind of commercial clout needed to move markets. That’s another big factor in the list.

If there’s one overarching theme I’d like to highlight most, it’s that drug development is a global pursuit. The US may be where the money is in terms of windfall profits, and it may still drive the lion’s share of the development work as the industry feels the full flush of cash coursing through labs, but the science is international. So is much of the clinical work. These companies span three continents, from North America to Europe and Asia.

And Asia is coming on strong, with major implications for the industry as a whole.

Finally, just because this is the first E11 doesn’t mean I’m starting over. If you’ve already been highlighted in another annual award I once managed, your chances of a repeat here were reduced to nil. We need to share the spotlight.

I’ll be back in the fall with my picks of 2018. And if you have any recommendations along the way, send them my way as I manage the next short list. — John Carroll 

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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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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive PhI­II for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma. 

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

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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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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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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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

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.