Tuesday, January 3, 2017

The Readout by Damian Garde & Meghana Keshavan

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Your take on 2017

What will the new year bring for biopharma? Check out our top reader predictions on STAT Plus. A sample:

‘Precision’ isn’t going anywhere

“In 2016, ‘precision medicine’ reigned supreme as the catch phrase of the year,” writes Dr. Christopher Ianelli, founder and CEO of iSpecimen. “‘Precision research,’ or the ever-more targeted research necessary to make good on the promise of precision medicine, will propel forward in 2017.... Refined research questions requiring the interrogation of highly specific biospecimens and associated data sets will form the backbone of this precision research wave.”

Data still wants to be free

“In 2017, we’re going to see a new push for data transparency in health care,” according to Ahmed Albaiti, CEO and founder of Medullan, a digital health consulting firm. “Electronic health record vendors will be asked to share data sets to better understand what better serves smaller populations, utilizing real world evidence for population health management.”

More combos are coming in cancer

“In 2017, because of favorable tolerability profiles of new drugs, combinations of multiple drugs will become even more the norm in oncology,” per Dr. Liviu Niculescu, vice president of medical affairs at Takeda Oncology. “Based on a growing body of research, companies are shifting toward developing increasingly targeted treatments or precision medicines that address specific issues at the cellular level and reduce systemic toxicity, opening the door for more combinations.”

Meet the year's first IPO contender

Last year wasn’t exactly a banner period for biotech IPOs, as a choppy market led to a drop in offerings and some disappointing returns for the companies that made it out. But the window hasn’t closed.

So Jounce Therapeutics has filed to become the first biotech IPO of 2017, seeking to raise $75 million to support its work in immuno-oncology. The company, based in Cambridge, Mass. is fresh off a $225 million partnership with Celgene and has already advanced its top cancer prospect into clinical trials.

Whether Jounce is able to get the cash it needs at the valuation it desires will be closely watched, as investors battered by 2016’s slump and bewildered by the US presidential election are looking for signs that biotech is approaching some kind of normalcy in the new year.

A window into the fraught world of rare disease

there are no approved therapies for sanfilippo syndrome. (sandy huffaker / stat)

Biotech bottom lines aren't all about IPOs and pricing margins. We can't forget that drug discovery often offers the best chance for families struggling with rare disease. It's a world fraught with risk, disappointment, and frustration, but also hope — as we see in the story of Zoe Dewaghe, a sunny five-year-old with a terminal lysosomal disorder called Sanfilippo syndrome. 

Read more.

Intarcia’s $50 million meeting

Back in June, Bill Gates swept through Boston and got something of a guided tour of the local biotech scene, including a meeting with local diabetes unicorn Intarcia.

Six months later, that sit-down blossomed into a $50 million equity deal, with the Gates Foundation promising up to $90 million more if Intarcia’s implantable drug delivery technology reaches certain milestones. 

Intarcia’s big priority is Type 2 diabetes, and it has raised more than $1 billion from investors to develop a mini-drug pump that metes out AstraZeneca’s treatment exenatide. But Gates’ interest stems from the potential of loading that same pump with a prophylactic treatment for HIV. If it works, patients in the developing world could get around-the-clock therapy that needs to be reloaded only once or twice per year.

Life science limericks: 2016 edition

elizabeth holmes is too easy a target. (hyacinth empinado / stat)

Nothing brings levity to a bleak situation like a silly rhyme. Last year wasn't the greatest — so here's a compendium of limericks to remember it by. An example:

Drug pricing? Pandora’s black box
First Shkreli, then the EpiPen shocks
It’s all a bit frightening
But also enlightening
We can expect way more price gouging talks

Read them all here.

More reads

  • FDA drug approvals plummeted last year, but 2017 is looking rosier. (STAT Plus)
  • Tinkering with DNA can have serious unintended consequences. (NPR)
  • Are scientific advances getting harder to achieve? If so, what do we do? (The Incidental Economist)

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Thanks for reading! Until tomorrow,

Damian & Meghana

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