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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Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA chief Stephen Hahn on Capitol Hill earlier this week (Getty Images)

As FDA’s work­load buck­les un­der the strain, Trump again ac­cus­es the agency of a po­lit­i­cal hit job

Peter Marks appeared before a virtual SVB Leerink audience yesterday and said that his staff at FDA’s CBER is on the verge of working around the clock. Manufacturing inspections, policy work and sponsor communications have all been pushed down the to-do list so that they can be responsive to Covid-related interactions. And the agency’s objective right now? “To save as many lives as we can,” Marks said, likening the mortality on the current outbreak as equivalent to “a nuclear bomb on a small city.”

David Berry (Flagship)

Flag­ship's next big tech­no­log­i­cal bet? The cloud

Earlier this month, Flagship announced their big bet on the software half the industry is talking about, launching the AI and machine learning startup. Now, they and a couple other investors are gambling $100 million on a software that much of the public generally thinks of as a cool, IT afterthought: cloud computing.

The idea, says founder and Flagship partner David Berry, is one of scale: The sheer magnitude of biological data that you can store on cloud technology is unprecedented. And that size, when leveraged properly, can allow you to ask questions and form insights that are similarly unprecedented.

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The win­dow is wide open as four more biotechs join the go-go IPO class of 2020

It’s another day of hauling cash in the biopharma world as four more IPOs priced Friday and a fifth filed its initial paperwork.

The biggest offering comes from PMV Pharma, an oncology biotech focusing on p53 mutations, which raised $211.8 million after pricing shares at $18 apiece. Prelude Therapeutics, developing PRMT5 inhibitors for rare cancers, was next with a $158 million raise, pricing shares at $19 each. Graybug Vision raised $90 million after pricing at $16 per share for its wet AMD candidates, and breast cancer biotech Greenwich Lifesciences brought up the rear with a small, $7 million raise after pricing shares at $5.75.

J&J of­fers PhI/IIa da­ta show­ing its sin­gle-dose vac­cine can stir up suf­fi­cient im­mune re­sponse

Days after J&J dosed the first participants of its Phase III ENSEMBLE trial, the pharma giant has detailed the early-stage data that gave them confidence in a single-dose regimen.

Testing two dose levels either as a single dose or in a two-dose schedule spaced by 56 days in, the scientists from Janssen, the J&J subsidiary developing its vaccine, reported that the low dose induced a similar immune response as the high dose. The interim Phase I/IIa results were posted in a preprint on medRxiv.

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Play-by-play of Gilead­'s $21B Im­munomedics buy­out de­tails a fren­zied push — and mints a new biotech bil­lion­aire

Immunomedics had not really been looking for a buyout when the year began. Excited by its BLA for Trodelvy, submitted to the FDA in late 2019, executive chairman Behzad Aghazadeh started off looking for potential licensing deals and zeroed in on four potential partners, including Gilead, following January’s JP Morgan Healthcare Conference in San Francisco. Such talks advanced throughout the year, with discussions advancing to the second round in mid-August.

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President Donald Trump reacts after signing an executive order following his remarks on his healthcare policies yesterday in Charlotte, North Carolina (Getty Images)

Op-ed: Will phar­ma re­al­ly pay for Trump’s lat­est law­less promise to 33 mil­lion Medicare ben­e­fi­cia­ries? Not like­ly

Sitting atop the executive branch, President Donald Trump is the ultimate authority at the FDA. He can fast track any vaccine to approval himself. If it came to that, of course.

What he can’t do is unilaterally order the legislative branch to loosen the Treasury’s coffers for $6.6 billion. Nor can he command pharmaceutical companies to pay for $200 vouchers sent to 33 million Medicare beneficiaries for prescription drugs before the election.

New York governor Andrew Cuomo (AP Images)

An­drew Cuo­mo says New York will un­der­take its own vac­cine re­view process, and wouldn’t rec­om­mend trust­ing the fed­er­al gov­ern­ment

The concerns keep mounting over President Donald Trump’s politicization of the FDA and other federal agencies guiding the development of a safe and effective vaccine. And today, the telegenic New York governor Andrew Cuomo appeared to introduce even more politics into the matter — latest in an ongoing series of incidents that have cast the proudly independent FDA in starkly political terms.

During his daily press conference Cuomo said that the state will review any coronavirus vaccines approved by the federal government, citing a lack of trust in the Trump administration. The announcement comes one day after Trump accused the FDA of making an “extremely political” move in proposing stricter vaccine guidance.

President Trump walks past HHS secretary Alex Azar (Getty Images)

Azar falls in line un­der Trump again. Ex­perts say he's re­in­forc­ing a dark sig­nal sent to the FDA

In the latest incident where Alex Azar has steadfastly taken the side of President Donald Trump over that of the FDA, the HHS secretary was noncommittal this morning when asked if he supports the attempt by his subordinates at the FDA to strengthen guidelines for a vaccine EUA.

Appearing on NBC’s Today Show, the HHS secretary muddied the waters, stating that the guidance that matters is the one that is “actually already out there.”