Al­ler­gan bags an ear­ly-stage gene ther­a­py in $60M Ret­roSense buy­out

While Al­ler­gan’s ac­quis­i­tive CEO Brent Saun­ders was map­ping out a new com­mit­ment to shun preda­to­ry pric­ing, he si­mul­ta­ne­ous­ly threw in the lat­est in a se­ries of bolt-on buy­outs.

David Nichol­son, Al­ler­gan

This time, Saun­ders has de­cid­ed to pay $60 mil­lion up front to ac­quire a small gene ther­a­py play­er based in Ann Ar­bor, MI. Ret­roSense Ther­a­peu­tics has been at­tract­ing at­ten­tion for its work us­ing a gene ther­a­py to re­store light sen­si­tiv­i­ty to the reti­na in an at­tempt to re­store the sight of peo­ple who have been blind­ed by re­tini­tis pig­men­tosa.

Ret­roSense hasn’t been in the in­dus­try spot­light much, but its op­to­ge­net­ics tech­nol­o­gy did at­tract the at­ten­tion of MIT Tech­nol­o­gy Re­view, which just tapped the biotech as one of the smartest com­pa­nies of 2016. Al­ler­gan can now ben­e­fit from the rep.

Al­ler­gan is reap­ing a lead treat­ment dubbed RST-001, which start­ed a Phase I/IIa clin­i­cal tri­al in the spring. The treat­ment im­plants a pho­to­sen­si­tiv­i­ty gene, chan­nel­rhodopsin-2, to cre­ate new pho­to­sen­sors in reti­nal gan­glion cells. The add-on beefs up Al­ler­gan’s pipeline of eye drugs and Ret­roSense in­vestors al­so get a slate of un­spec­i­fied mile­stones for a suc­cess­ful pro­gram.

Al­ler­gan has a pipeline that bal­ances a rel­a­tive­ly low-risk cos­met­ic R&D ap­proach with high-risk, high-re­ward ef­forts for de­pres­sion and oth­er CNS con­di­tions. Af­ter its deal to merge with Pfiz­er fell through ear­li­er in the year, Saun­ders im­me­di­ate­ly re­turned to his strat­e­gy of buy­ing up new com­pa­nies, large­ly stick­ing with late-stage ef­forts. The deal for Ret­roSense sig­nals that Saun­ders al­so is in­creas­ing­ly will­ing to step in ear­ly on po­ten­tial­ly game-chang­ing pro­grams.

Gene ther­a­pies have been ex­pe­ri­enc­ing some­thing of a re­nais­sance in re­cent years, with com­pa­nies like Spark Ther­a­peu­tics mak­ing some big gains in the clin­ic. Still, each com­pa­ny in the field has some big work to do prov­ing just how durable their treat­ments will be and whether they stack up as re­al gains for pa­tients. UniQure gained a pi­o­neer­ing ap­proval in Eu­rope, but its treat­ment has re­port­ed­ly on­ly been used once. Biotechs here will al­so have to come up with some way to price gene ther­a­pies, which may be of­fered as an un­ver­i­fied cure.

David Nichol­son, the chief re­search and de­vel­op­ment of­fi­cer at  Al­ler­gan, had this to say:

“The team at Al­ler­gan is ex­cit­ed by the prospect of ad­vanc­ing an en­tire­ly new ap­proach in the treat­ment of reti­nal dis­eases, and this tech­nol­o­gy is high­ly com­ple­men­tary to our on­go­ing de­vel­op­ment pro­grams in this crit­i­cal treat­ment area.”

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

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 72,900+ biopharma pros reading Endpoints daily — and it's free.

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.

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.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 72,900+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 72,900+ biopharma pros reading Endpoints daily — and it's free.

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.

Anthony Fauci, AP Images

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.

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.