Ipsen bets up to $363M on an­oth­er shot at a de­bil­i­tat­ing con­di­tion re­lat­ed to Parkin­son's dis­ease

Ipsen is in a deal-mak­ing mood. On Thurs­day, the com­pa­ny put down $28 mil­lion up­front and an­oth­er $335 mil­lion in biobucks for the rights to IR­LAB’s mid-stage can­di­date for lev­odopa-in­duced dysk­i­ne­sia (LID) — a con­di­tion re­lat­ed to Parkin­son’s dis­ease that tripped up the French biotech about a decade ago.

“This is some­thing that fits nice­ly with our strat­e­gy in neu­ro­science, with move­ment dis­or­ders, and we’re al­so ob­vi­ous­ly look­ing more broad­ly in oth­er ther­a­peu­tic ar­eas, in our core ther­a­peu­tic ar­eas, in­clud­ing rare dis­eases and on­col­o­gy,” ex­ec­u­tive VP and head of R&D Howard May­er told End­points News. 

Howard May­er

IR­LAB’s mes­dopetam is cur­rent­ly in a Phase IIb study in LID that’s set for a read­out in 2022. Re­searchers be­lieve the drug al­so has po­ten­tial to treat psy­chosis in Parkin­son’s dis­ease, and May­er says it could en­ter the clin­ic for that in­di­ca­tion the same year.

Parkin­son’s dis­ease is com­mon­ly treat­ed with a drug de­vel­oped back in the 1960s called lev­odopa, which works by re­plen­ish­ing the brain’s sup­ply of dopamine. But by the ear­ly 70s, sci­en­tists be­gan look­ing in­to whether the drug was caus­ing dysk­i­ne­sias, or im­pair­ments of vol­un­tary move­ment.

It’s es­ti­mat­ed that 40% to 50% of pa­tients who use lev­odopa for at least five years de­vel­op LID — a side ef­fect that caus­es in­vol­un­tary or er­rat­ic move­ments, or slow and ex­tend­ed mus­cle spasms, ac­cord­ing to Ipsen. In se­vere cas­es, the con­di­tion can be in­ca­pac­i­tat­ing.

“It’s very, very de­bil­i­tat­ing, af­fect­ing ac­tiv­i­ties of dai­ly liv­ing, and ob­vi­ous­ly com­pli­cates their Parkin­son­ian symp­toms,” May­er said.

Mes­dopetam works by block­ing a dopamine re­cep­tor called D3, which has been ge­net­i­cal­ly linked to an in­creased risk of in­vol­un­tary move­ments. Parkin­son’s pa­tients with LIDs have been found to have high­er amounts of D3 re­cep­tors in parts of the brain es­sen­tial to the con­trol of move­ment.

In a Phase IIa study, pa­tients who took mes­dopetam with the stan­dard of care saw a dose-de­pen­dent in­crease in dai­ly time with­out dysk­i­ne­sias, which IR­LAB calls “Good ON” time, IR­LAB said. At the best-per­form­ing dose, 7.5 mg, Good ON time in­creased by 5.6 hours dai­ly in the treat­ment group, com­pared to one hour in the place­bo group (p<0.002). Pri­or to treat­ment with mes­dopetam, these pa­tients on­ly had about 6.3 hours of Good ON time per day, ac­cord­ing to IR­LAB.

“When mes­dopetam was giv­en in ad­di­tion to stan­dard Parkin­son med­ica­tion, pa­tients ex­pe­ri­enced con­sid­er­ably longer pe­ri­ods of good dai­ly mo­tor func­tion with­out ag­gra­vat­ed in­vol­un­tary move­ments,” IRL CMO Joakim Tedroff said at the time.

IR­LAB will be re­spon­si­ble for the on­go­ing Phase IIb tri­al, while Ipsen is on the hook for all oth­er clin­i­cal de­vel­op­ment and world­wide com­mer­cial­iza­tion, in­clud­ing Phase III prepara­to­ry work.

This isn’t Ipsen’s first shot at treat­ing LIDs. Back in 2010, Ipsen gave San­thera Phar­ma­ceu­ti­cals about $15.3 mil­lion up­front and promised an­oth­er $151 mil­lion in biobucks for the rights to its LID can­di­date fi­pame­zole out­side North Amer­i­ca and Japan. But a short two years lat­er, the com­pa­ny re­turned the rights to San­thera, which lat­er dropped it from the pipeline.

George Scangos (L) and Marianne De Backer

Pi­o­neer­ing biotech icon George Scan­gos hands in his re­tire­ment pa­pers — and this time it’s for re­al

George Scangos, one of the all-time great biotech CEOs, says the time has come to turn over the reins one last time.

The 74-year-old biotech legend spent close to three decades in a CEO post. The first was at Exelixis — which is still heavily focused on a drug Scangos advanced in the clinic. The second “retirement” was at Biogen, where he and his team were credited with a big turnaround with the now fading MS blockbuster Tecfidera. And the third comes at Vir, where he traded in his Big Biotech credentials for a marquee founder’s role back on the West Coast, hammering out a Covid-19 alliance with Hal Barron — then R&D chief at GSK — and breaking new ground on infectious diseases with some high-powered venture players.

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Jeanne Loring, director of the Center for Regenerative Medicine (Credit: Jamie Scott Lytle)

A stem cell pi­o­neer sent an ex­per­i­ment in­to space. Pa­tients are the next fron­tier

Last July, Jeanne Loring stood on a dirt road surrounded by Florida swampland and watched as a nearby SpaceX rocket blasted into the sky. The payload included a very personal belonging: cell clusters mimicking parts of her brain.

For more than two decades, Loring has been at the forefront of a stem cell field that always seems on the brink of becoming the next thing in medicine, but has been slow to lift off.

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FDA re­ports ini­tial 'no sig­nal' for stroke risk with Pfiz­er boost­ers, launch­es con­comi­tant flu shot study

The FDA hasn’t detected any potential safety signals, including for stroke, in people aged 65 years and older who have received Pfizer’s bivalent Covid booster, one senior official told members of the agency’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Thursday.

The update comes as the FDA and CDC investigate a “preliminary signal” that may indicate an increased risk of ischemic stroke in older Americans who received Pfizer’s updated shot.

FDA cuts off use for As­traZeneca’s Covid-19 ther­a­py Evusheld

The FDA has stopped use of another drug as a result of the new coronavirus variants. On Thursday, the agency announced that AstraZeneca’s antibody combo Evusheld, which was an important prevention option for many immunocompromised people and others, is no longer authorized.

The FDA said it made its decision based on the fact that Evusheld works on fewer than 10% of circulating variants.

Evusheld was initially given emergency authorization at the end of 2021. However, as Omicron emerged, so did studies that showed Evusheld might not work against the dominant Omicron strain. In October, the FDA warned healthcare providers that Evusheld was useless against the Omicron subvariant BA.4.6. It followed that up with another announcement earlier this month that it did not think Evusheld would work against the latest Omicron subvariant XBB.1.5.

In­vestor 'misalign­men­t' leads to tR­NA biotech's shut­ter­ing

A small biotech looking to carve a lane in the tRNA field has folded, an investor and a co-founder confirmed to Endpoints News.

Similar to Flagship’s Alltrna and other upstarts like Takeda-backed hC Bioscience, the now-shuttered Theonys was attempting to go after transfer RNA, seen as a potential Swiss Army knife in the broader RNA therapeutics space. The idea is that one tRNA drug could be used across a galaxy of disorders and diseases.

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#JPM23: What's re­al­ly dri­ving the cost of health­care and drugs in 2023?

Executive Editor Drew Armstrong spoke with PhRMA CEO Steve Ubl, EmsanaRx CEO Greg Baker and ICER President Steve Pearson about how the debate over drug costs has changed (or not) in the last decade, the shifting payer landscape and why there seems to be so little movement on drug rebates. This transcript has been edited for brevity and clarity.

Drew Armstrong:

So first of all, thank you to everybody for being here and for our panel for being here. Incredibly excited to have this discussion on the cost of healthcare and drugs and what’s driving that. We’re here with Steve Ubl, the head of PhRMA. Thank you so much. Steve Pearson from ICER, and Greg Baker from EmsanaRx. I want to start this conversation with a little bit of a personal reminiscence. So about almost 10 years exactly. I was a reporter back in my previous job and I was covering drug pricing and Gilead had just launched their hepatitis C drug and I was having a conversation with another Steve over at Express Scripts and he made some comments essentially about how they intended to launch a price war over hepatitis C therapies.

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Pa­tient death spurs tri­al halt for Ma­gen­ta Ther­a­peu­tics

Magenta Therapeutics is pausing an early-stage clinical trial after a patient died. The death was deemed to be possibly related to its drug, MGTA-117.

The biotech said the pause of the Phase I/II trial is voluntary and gives it time to review all available data before deciding what to do next. It’s also reported the known information to the FDA.

The dose-escalation trial was designed to test whether MGTA-117, an antibody-drug conjugate, could serve as a more targeted alternative to high-intensity chemotherapy as a conditioning agent for cancer patients who are set to receive a stem cell transplant. It recruited patients with relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome.

In a win for Re­gen­eron, No­var­tis' sy­ringe for AMD drug de­clared 'un­patentable'

Regeneron has won a patent case against Swiss pharma giant Novartis over the delivery system for its eye drug Eylea.

The US Patent Trial and Appeal Board ruled that Novartis’ pre-filled syringe for injecting its eye medication Lucentis was “unpatentable” and handed the victory to Regeneron and its AMD drug Eylea.

In the initial complaint in 2020, Novartis alleged to the US International Trade Commission that certain pre-filled syringes for the intravitreal injection, and ultimately Regeneron’s delivery system for Eylea, were infringing on Novartis’ patent. Regeneron filed a petition to review Novartis’ claims in 2021.

'Tis the sea­son: GSK ad­dress­es win­ter virus surges with celebri­ty and in­flu­encer vac­cine aware­ness cam­paigns

GSK is rounding up the usual suspects this winter — flu, respiratory syncytial and even shingles viruses — for multiple marketing efforts all aimed at encouraging vaccinations.

Mom influencers take center stage in its “Flu is a Family Affair” campaign to reach family decision-makers or “chief health officers.” GSK is asking them in the digital campaign to take care of themselves, and take the family along, when they go to the pharmacy or doctor’s office for a flu vaccine.

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