A biotech start­up breaks a 3-year si­lence, dis­play­ing a $65M raise and a next-gen ap­proach to PARP

Vic­to­ria Ri­chon’s team at Ri­bon Ther­a­peu­tics has been cruis­ing un­der the biotech radar for the past 3 years. But with a $65 mil­lion B round to dis­cuss with plans to move in­to the clin­ic, it’s time for a pub­lic dis­play on just what they’ve been work­ing on.

Over the past few years a whole group of play­ers has been ad­vanc­ing PARPs in­to the mar­ket for can­cer, throw­ing a mon­key wrench in a mech­a­nism cells use to re­pair dam­age. But while ri­vals stack up be­hind Lyn­parza from As­traZeneca and Mer­ck, Ri­bon is tak­ing it all one big step down the road.

Those pi­o­neer­ing PARPs on the mar­ket tar­get PARP1, but that’s just one of a broad fam­i­ly of 17 en­zymes. Ri­bon’s lead pro­gram goes af­ter PARP7, a dif­fer­ent pro­tein and a dif­fer­ent tar­get in this world, sim­i­lar­ly ac­ti­vat­ed by stress and cel­lu­lar re­sponse mech­a­nisms. And they have oth­er pro­grams com­ing up from be­hind.

“We look at our­selves as pi­o­neers,” Ri­chon, a Sanofi vet, tells me on be­half of the team of 26. Their PARP7 pro­gram is now be­ing steered to the clin­ic in search of proof-of-con­cept da­ta to back up their lab work. And as the tech­nol­o­gy has been sharp­ened, the CEO adds that they can move faster now in iden­ti­fy­ing which PARPs to use on par­tic­u­lar dis­ease tar­gets.

That pro­file has at­tract­ed a mix of cor­po­rate and strate­gic in­vestors to the B round, which brings their to­tal raised to $108 mil­lion, fol­low­ing the $43 mil­lion in Se­ries A cash gar­nered in 2015.

No­var­tis Ven­ture Fund led the round, with J&J In­no­va­tion and Cel­gene Cor­po­ra­tion jump­ing in along­side The Col­umn Group, Deer­field Man­age­ment, U.S. Ven­ture Part­ners, Os­age Uni­ver­si­ty Part­ners, Take­da Ven­tures and Eu­clid­ean Cap­i­tal.


Im­age: Vic­to­ria Ri­chon. HOTSPOT

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

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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.

Bio­gen touts new ev­i­dence from the gene ther­a­py com­pa­ny it wa­gered $800M on

A year ago, Biogen made a big bet on a small gene therapy company. Now they have new evidence one of their therapies could work.

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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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In fi­nal re­port, ICER ap­pears to have a change of heart on new acute mi­graine ther­a­pies

ICER appears to have reversed course on the fresh crop of acute migraine therapies.

The cost-effectiveness watchdog in November issued a draft report suggesting that existing generic medicines are more effective and cheaper than Allergan’s December-approved CGRP ubrogepant, Biohaven rival molecule, rimegepant (which is under FDA review), and Lilly’s October-sanctioned lasmiditan, which binds to 5-HT1F receptors.

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.

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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.

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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.

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.