Mile­stone Phar­ma grabs an $80M round as it steams in­to piv­otal tri­al work, starts out­lin­ing com­mer­cial struc­ture

Eleven years af­ter it got start­ed, Mile­stone Phar­ma­ceu­ti­cals has gained the $80 mil­lion it needs to ex­plore in a piv­otal study whether its new car­dio drug works as ex­pect­ed. And there’s enough mon­ey in the pack­age to start lay­ing the foun­da­tion of a mar­ket­ing ef­fort.

Joseph Olive­to

Based in Mon­tre­al, Mile­stone has been pa­tient­ly work­ing on a drug called etri­pamil, a cal­ci­um chan­nel block­er that Mile­stone has for­mu­lat­ed in­to a nasal spray for quick re­lease to halt tachy­car­dias.

In the vast ma­jor­i­ty of cas­es, parox­ys­mal supraven­tric­u­lar tachy­car­dia — or PSVT — is not dan­ger­ous. But pa­tients who ex­pe­ri­ence these cas­es of­ten can’t tell that, rush­ing off to the ER com­plain­ing of heart pal­pi­ta­tions. 

In a mid-stage dose-rang­ing study, in­ves­ti­ga­tors tracked a high suc­cess rate in quelling the symp­toms of PSVT in the three high­est dose co­horts, with con­ver­sion rates of 65% to 95%. The place­bo arm reg­is­tered a 35% suc­cess rate, giv­ing them the sta­tis­ti­cal sig­nif­i­cance they were look­ing for.

“As our reg­is­tra­tion pro­gram for etri­pamil in PSVT is pro­gress­ing well, we are now in­creas­ing­ly fo­cused on our com­mer­cial pre­pared­ness and, at the same time, look­ing to ex­pand our pipeline,” said CEO Joseph Olive­to in a state­ment.

New in­vestors in­clud­ed RTW In­vest­ments, which led the round, Ven­rock Health­care Cap­i­tal Part­ners and Box­er Cap­i­tal of Tavi­s­tock Group. Ex­ist­ing in­vestors in­clud­ed No­vo Hold­ings A/S, For­bion, funds man­aged by Tekla Cap­i­tal Man­age­ment, Do­main As­so­ci­ates, BDC Cap­i­tal, Pap­pas Cap­i­tal, GO Cap­i­tal, and Fonds de sol­i­dar­ité FTQ. No­vo Hold­ings led the C round, which oc­curred in Ju­ly 2017.

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

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: 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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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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Mickey Kertesz, KidsandArtOrg via YouTube

Soft­Bank's newest, $165M biotech in­vest­ment looks for in­fec­tious traces in the blood

SoftBank has found its newest biotech investment.

The Japanese bank has invested $165 million into Karius, a company that uses blood tests to diagnose infectious diseases, as part of its new Vision Fund 2. The full scope of the new fund has yet to be announced, but the first and newly-beleaguered Vision Fund poured $100 billion into technology companies, including the biotechs Vir Biotechnology and Roivant and the sequencing company 10x Genomics.

Methicillin-resistant Staph aureus (Shutterstock)

FDA grants ‘break­through’ sta­tus to an­tibi­ot­ic al­ter­na­tive as Con­tra­Fect rush­es to join fight against su­per­bug

An experimental drug that promises to be the first anti-infective agent to prove superior to vancomycin — an antibiotic approved in 1958 — has notched the FDA’s “breakthrough” status.

ContraFect said the designation was based on Phase II data in which exebacase was tested against a superbug known as methicillin-resistant Staph aureus, or MRSA. In a subgroup analysis, the clinical responder rate at day 14 was 42.8% higher than that among those treated with standard of care, the company said (p=0.010).