The End­points 11: Here are some of the most promis­ing star­tups in biotech. And Covid-19 is­n't go­ing to stop them

Quite often when I highlight a private, typically early-stage biotech company for these annual awards, I’m asked what I look for in a company. How do you define an Endpoints 11 company?

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There’s a simple enough answer. First, I look for big ideas among the private biotechs which qualify for review, a swing for the fences. I’m drawn to people who are willing to gamble a chunk of their lives on new drug ideas, and in that regard, the stakes here are huge. Drug development is long and hard, and you could easily be wagering 10 or more years of your life on something. So go big or go home if you aren’t planning to do something transformative.

I want a team directing these projects that have the talent, vigor and knowhow to make it happen. Track records are key and earlier failures can be a big plus — depending on how that played out.

People are always more important than money. That’s a mantra of mine that always earns quick nods in any biotech group. But making sure that a company has enough lucre to make it through to proof of concept, and beyond, is also key to the selection process.

Still, you’ll find companies below that raised a few million to get started, and biotechs that appear to have found the key to Fort Knox. So it’s variable.

And then there’s the noise factor. I want to choose companies that can earn widespread recognition for success — or make a hell of a noise if they fail. None of these companies below will slink off to an anonymous midnight burial if they fall short of victory. They’ll make a noise heard throughout the global industry, and we can all learn from it.

This year, with a pandemic raging around the world, I wanted to focus more on culture than particular assets. Each of these biotechs is sorting out the R&D work, but I want to let the execs reflect more on the most important points of starting a company than the specific program goals in mind. Building companies these days takes some newfound ingenuity, and it’s important to share these points with everyone.

There are some other trends you’ll see reflected in the stories below. The intersection of biotech and Big Pharma is taking on a different tone at times, with an eye to assembling large networks of experts to tackle complex problems. And the inflow of billions of dollars of fresh investment capital allows for a new breed of startup conspiring to sire transformative therapies. That’s not cheap, and success is not guaranteed. But it is always fascinating.

Let me sum up here by noting that the biggest healthcare crisis in 100 years has helped make biotech the place to be, fortunately for all of us. These companies aren’t squandering the opportunity. And that deserves a special shout out all on its own.

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Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Tom Barnes (Orna)

The mR­NA era is here. MPM be­lieves the fu­ture be­longs to oR­NA — and Big Phar­ma wants a seat at the ta­ble

If the ultra-fast clinical development of Covid-19 vaccines opened the world’s eyes to the promises of messenger RNA, the subsequent delays in supply offered a crash course on the ultra-complex process of producing them. Even before the formulation and fill-finish steps, mRNA is the precious end product from an arduous journey involving enzyme-aided transcription, modification and purification.

For Bristol Myers Squibb, Novartis Institutes for Biomedical Research, Gilead’s Kite and Astellas, it’s time to rethink the way therapeutic RNA is engineered.

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Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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Photo: Shutterstock

Bio­phar­ma's suc­cess rate in bring­ing drugs to mar­ket has long been abysmal. Can new tools help rewrite that trou­bled past?

In 2011, a team of researchers at British drugmaker AstraZeneca had a problem they were looking to solve.

For years, drug discovery and development were a wasteland for innovation. Novel drugs largely fell into one of two categories — monoclonal antibodies and small molecules — and new therapeutic modalities were hard to come by. After a rush of promising approvals in the late 1990s — including then-Biogen’s CD20 targeting antibody breakthrough Rituxan — the field stagnated and attrition rates stayed sky-high. What exactly is the industry doing wrong? AstraZeneca asked itself.

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S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

CMO Tal Zaks bids Mod­er­na a sur­prise adieu as biotech projects $18.4B in rev­enue, plots post-Covid ex­pan­sion

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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Joe Nolan, Jaguar

Sean Nolan gath­ers AveX­is alum­ni to launch an­oth­er biotech. Is it an­oth­er race-to-IPO?

The old AveXis crew is launching another company. And they may once again look to cash in quick.

Armed with patents and a steady stream of cash from Deerfield Management, Sean Nolan and five ex-Avexis executives have launched Jaguar Gene Therapy to develop AAV9 gene therapies for a small constellation of disparate disorders, from autism to diabetes. Jaguar remains over a year from the clinic, but CEO Joe Nolan hinted they could look to follow a pace set by Tasyha, the biotech Sean Nolan and other AveXis alumni launched last year and took from Series A to IPO in less than 6 months.