Thomas Perlmann (far right), Secretary of the Nobel Assembly, announces the 2020 Nobel laureates in Physiology or Medicine to (left on screen) Harvey Alter, Michael Houghton, center, and Charles Rice (Claudio Bresciani/TT via AP Images)

Three sci­en­tists win No­bel Prize in Med­i­cine for work in dis­cov­er­ing he­pati­tis C virus

Mon­day marks the start of No­bel Prize week, and the Swedish com­mit­tee kicked things off by hand­ing out the first award to three vi­rol­o­gists.

Har­vey Al­ter, Michael Houghton and Charles Rice have joint­ly won the No­bel Prize in Phys­i­ol­o­gy or Med­i­cine for their work on the dis­cov­ery of the he­pati­tis C virus. Their re­search pro­vid­ed the foun­da­tion for test­ing and an­tivi­rals that can help those in­fect­ed re­ceive treat­ment, as well as a po­ten­tial vac­cine for the liv­er dis­ease.

“For the first time in his­to­ry, the dis­ease can now be cured, rais­ing hopes of erad­i­cat­ing He­pati­tis C virus from the world pop­u­la­tion,” the No­bel com­mit­tee said in a state­ment.

Al­ter, the el­der states­man of the group, be­gan his work in the he­pati­tis field back in the 1960s and 1970s, around the time when the he­pati­tis B virus was first dis­cov­ered. Work­ing at the NIH, he had been study­ing the oc­cur­rence of the dis­ease in pa­tients who had re­ceived blood trans­fu­sions when he no­ticed that not all of the cas­es could be ex­plained by the new­ly iden­ti­fied virus.

Near­ly a decade lat­er, Houghton and his co-work­ers at the phar­ma­ceu­ti­cal com­pa­ny Ch­i­ron iso­lat­ed the ge­net­ic se­quence of the virus through ex­per­i­ments with chim­panzees. By study­ing the blood of the in­fect­ed an­i­mals, Houghton was able to pre­dict which of the DNA frag­ments with­in the genome came from the chim­panzee and which came from the virus.

The Ch­i­ron team was then able to clone the vi­ral DNA. While search­ing for an­ti­body re­spons­es, Houghton dis­cov­ered an RNA virus de­rived from the Fla­vivirus fam­i­ly that was ul­ti­mate­ly de­ter­mined to be he­pati­tis C.

Houghton’s work proved piv­otal, but one ma­jor ques­tion re­mained — could the virus alone cause he­pati­tis C? Rice, then a re­searcher at Wash­ing­ton Uni­ver­si­ty in St. Louis, be­gan in­ves­ti­gat­ing if the cloned virus could repli­cate and cause dis­ease. He de­ter­mined that a pre­vi­ous­ly un­char­ac­ter­ized re­gion in the virus genome could be in­flu­en­tial in repli­ca­tion, and en­gi­neered an RNA vari­ant to de­ter­mine this re­gion’s ef­fect.

Af­ter in­ject­ing the vari­ant in­to chim­panzees, Rice de­tect­ed the virus in their blood, con­firm­ing that he­pati­tis C could cause dis­ease and repli­cate on its own.

Al­ter con­tin­ues to work at the NIH as the chief of in­fec­tious dis­ease at its Clin­i­cal Cen­ter. Houghton, the on­ly non-Amer­i­can in the group be­ing from the UK, is cur­rent­ly a pro­fes­sor at the Uni­ver­si­ty of Al­ber­ta in Cana­da. Rice now works at Rock­e­feller Uni­ver­si­ty in New York.

The No­bel Com­mit­tee drew a par­al­lel be­tween the work in he­pati­tis C and re­search be­ing cur­rent­ly un­der­tak­en to try to iden­ti­fy and cure the coro­n­avirus at the cen­ter of the Covid-19 pan­dem­ic, per an As­so­ci­at­ed Press re­port. Com­mit­tee mem­ber Pa­trik Ern­fors called the dis­cov­ery of a virus a “crit­i­cal mo­ment.”

“The first thing you need to do is to iden­ti­fy the caus­ing virus,” he told re­porters. “And once that has been done, that is in it­self the start­ing point for de­vel­op­ment of drugs to treat the dis­ease and al­so to de­vel­op vac­cines against the dis­or­der.”

Ac­cord­ing to num­bers from the WHO, there are over 70 mil­lion cas­es of he­pati­tis C around the world with ap­prox­i­mate­ly 400,000 deaths per year.

Charles Baum, Mirati CEO

UP­DAT­ED: Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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David Hung (file photo)

Mas­ter deal­mak­er David Hung re­tools a SPAC sedan in­to a fi­nanc­ing mus­cle ve­hi­cle that leaves his can­cer start­up with $850M and a place on Wall Street

It’s only right that one of the industry’s top dealmakers just completed one of the biggest SPAC-related deals in the pipeline.

David Hung, of Medivation fame, has completed a back flip into the market, merging with EcoR1 Capital’s SPAC Panacea and landing neatly on Wall Street with an $NUVB stock ticker after filling out the blank check in his name. In addition to the $144 million held in the SPAC — provided none of the investors opt out — Hung is getting ahold of $500 million more being chipped in by a slate of institutional investors who feel that Hung could have the keys to another Medivation-style success.

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Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

A top drug pro­gram at Bay­er clears a high bar for CKD — open­ing the door to an FDA pitch

Over the past 4 years, Bayer has been steering a major trial through a pivotal program to see if their drug finerenone could slow down the pace of chronic kidney disease in patients suffering from both CKD as well as Type 2 diabetes.

Today, their team jumped on a virtual meeting hosted by the American Society of Nephrology to offer a solid set of pivotal data to demonstrate that the drug can delay dialysis or a kidney replacement as well as cardio disease, while also adding some worrying signs of hyperkalemia among the patients taking the drug. And they’re hustling it straight to regulators in search of an approval for kidney disease and cardio patients — one of the toughest challenges in the book, as demonstrated by repeated past failures.

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Adam Koppel and Jeffrey Schwartz, Bain

Bain ex­ecs Adam Kop­pel and Jef­frey Schwartz line up $125M for their first blank check deal as Wall Street con­tin­ues to em­brace biotech

Adam Koppel and Jeffrey Schwartz have jumped into the blank check game, raising $125 million for a stock listing in search of a company.

Their SPAC, BCLS Acquisition Corp, raised $125 million this week, with a line on $25 million more as it scouts for a biotech in search of money and a place on Wall Street.

The two principals at Bain Life Sciences have been on a romp since they set up the Bain operation 4 years ago. Their S-1 spells out a track record of 22 deals totaling $650 million for the life sciences group, which led to 9 IPOs.