VC gi­ant NEA backs an up­start’s plan to tack­le neu­rode­gen­er­a­tion

Neu­rode­gen­er­a­tive dis­eases are both one of the tough­est chal­lenges in drug de­vel­op­ment as well as po­ten­tial­ly one of the most lu­cra­tive fields in the busi­ness. If you can fig­ure out what the bad play­ers are that trig­ger dam­age, and come up with a drug to halt or even mod­est­ly slow that process, you could have a block­buster on your hands.

But then there’s a long line of com­pa­nies that have tried and failed at it, blow­ing bil­lions in the process.

An­nex­on Bio­sciences in the South San Fran­cis­co hub is one of the up­starts that is try­ing to beat the odds. To­day, it gained the sup­port of New En­ter­prise As­so­ci­ates – the be­he­moth in the glob­al biotech VC com­mu­ni­ty – for a $44 mil­lion B round that will be­gin to put their the­o­ries to a hu­man test.

The bad play­er for An­nex­on is C1q, a pro­tein that plays a nat­ur­al role in prun­ing un­need­ed synaps­es in our youth. But when brain trau­ma oc­curs, say when tox­ic pro­teins clus­ter in the brain, C1q goes rogue, run­ning amuck and cre­at­ing dam­age by re­mov­ing healthy synaps­es and in­flict­ing se­ri­ous dam­age on our cog­ni­tive abil­i­ties.

Or at least that is An­nex­on’s work­ing the­o­ry. So far, it’s been run­ning an­i­mal mod­els on safe­ty and ef­fi­ca­cy. The lead pro­gram will set out to pro­tect nerve con­nec­tions in the brain as well as an­oth­er pro­gram fo­cused on oph­thal­mol­o­gy. 

An­nex­on was co-found­ed by Stan­ford’s Ben Bar­res and the for­merC­SO at Ri­nat, Arnon Rosen­thal. 

Cor­re­la­tion Ven­tures and a group of ex­ist­ing An­nex­on in­vestors, in­clud­ing No­var­tis Ven­ture Fund, Clarus, and Sat­ter In­vest­ment Man­age­ment, all joined the round. And NEA’s Frank Tor­ti, M.D., is join­ing the board.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Bi­cy­cle Ther­a­peu­tics takes Roche's Genen­tech on an up to $2B im­muno-on­col­o­gy ride

Bicycle Therapeutics — which is developing a new class of chemically synthesized drugs designed to be pharmacologically as active as biologics, yet manufactured as small molecules —  has scored another big partner: Roche’s Genentech.

The transatlantic biotech, which was created in 2009 and made its public debut a decade later, gets $30 million upfront and a meaty $1.7 billion in potential milestone payments in an early discovery immuno-oncology collaboration with Genentech.

When drug val­ue as­sess­ment meets re­al-world ev­i­dence: ICER en­lists Ae­tion in pric­ing eval­u­a­tion

In a union of two of the hottest trends in the US biopharma world, ICER is teaming up with a high-profile company to integrate real-world evidence in their assessment of treatment value.

The drug pricing watchdog — formally the Institute for Clinical and Economic Review — said it will utilize Aetion’s evidence platform in “select upcoming assessments” and their new 24-month re-evaluations of drugs granted accelerated approval by the FDA.

First US Covid-19 tri­als set to get un­der­way in Ne­bras­ka and Wash­ing­ton, backed by NIH

The first US clinical trials on the novel coronavirus are scheduled to get underway next month at the University of Nebraska Medical Center, where American passengers were taken after being evacuated from the Diamond Princess cruise ship, and at the Kaiser Permanente Washington Health Research Institute. Both trials are sponsored by the NIH’s National Institute for Allergy and Infectious Diseases, which has led the US’s medical response to the outbreak.

Anthony Fauci (AP Images)

UP­DAT­ED: NIH-part­nered Mod­er­na ships off its PhI-ready coro­n­avirus vac­cine can­di­date to a sea of un­cer­tain­ty

Off it goes.

Moderna has shipped the first batch of its mRNA vaccine against SARS-CoV-2 from its manufacturing facility in Norwood, Massachusetts, to the National Institute of Allergy and Infectious Diseases in Bethesda, Maryland, for a pioneering Phase I study.

It’s a hectic race against time. In the 42 days since Moderna selected the sequence they would use to develop their vaccine — a record time, no less — the number of confirmed cases around the world has surged astronomically from a few dozen to over 80,000, per WHO and Johns Hopkins estimates.

The candidate that they came up with, mRNA-1273, encodes for a prefusion stabilized form of the spike protein, which gives the virus its crown shape and plays a key role in transmission. The Coalition for Epidemic Preparedness Innovations, the Oslo-based group better known as CEPI, funded the manufacture of this batch.

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Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

Mallinck­rodt, once the na­tion’s largest oxy­codone pro­duc­er, an­nounces ten­ta­tive $1.6B set­tle­ment

Three years after it first paid out fines for its role in the US opioid abuse epidemic, Mallinckrodt has announced an agreement-in-principle that will see the company pay out $1.6 billion and place its generics unit in bankruptcy.

The tentative deal would settle hundreds of lawsuits from state and local governments over Mallinckrodt’s role in the epidemic, while also helping address the company’s increasingly mountainous debt. Although Purdue Pharma has drawn the bulk of both public and legal acrimony for opioid sales, documents made public earlier this year showed that Mallinckrodt subsidiary SpecGx, along with the generic subsidiaries of Teva and Endo Pharmaceuticals, accounted for the vast majority of the 76 billion opioid pills distributed from 2006 to 2012. Mallinckrodt was at the top of that list.

UP­DAT­ED: NGM Bio takes leap for­ward in crowd­ed NASH field

South San Francisco-based NGM Bio may have underwhelmed with its interim analysis of a key cohort from a mid-stage NASH study last fall — but stellar topline data unveiled on Monday showed the compound induced significant signs of antifibrotic activity, NASH resolution and liver fat reduction, sending the company’s stock soaring.

There are an estimated 50+ companies focused on developing drugs for non-alcoholic steatohepatitis, or NASH, a common liver disease that has long flummoxed researchers. The first wave of NASH drug developers struggled with efficacy as well as safety — and companies big and small have crashed and burned.

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UP­DAT­ED: No­var­tis' plans to wres­tle Eylea mar­ket share take a hit as Beovu is linked to safe­ty con­cerns

While Regeneron’s flagship eye therapy Eylea hurtles towards a patent cliff, the sales of its main rival — Novartis’ Beovu — could be tainted by safety concerns.

On Sunday night, Chicago-based American Society of Retina Specialists (ASRS) issued a note to members about 14 cases of retinal vasculitis (including 11 cases of occlusive retinal vasculitis) — a sight-threatening inflammatory eye condition that involves the retinal vessels — across 46,000 injections administered since Beovu’s launch in November 2019, Wall Street analysts reported.

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