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.

What Will it Take to Re­al­ize the Promise and Po­ten­tial of Im­mune Cell Ther­a­pies?

What does it take to get to the finish line with a new cancer therapy – fast? With approvals in place and hundreds of immune cell therapy candidates in the pipeline, the global industry is poised to create a fundamental shift in cancer treatments towards precision medicine. At the same time, unique challenges associated with cell and process complexity present manufacturing bottlenecks that delay speed to market and heighten cost of goods sold (COGS) — these hurdles must be overcome to make precision treatments an option for every cancer patient. This series of articles highlights some of the key manufacturing challenges associated with the production of cell-based cancer therapies as well as the solutions needed to transcend them. Automation, process knowledge, scalability, and assured supply of high-quality starting material and reagents are all critical to realizing the full potential of CAR-based therapies and sustaining the momentum achieved in recent years. The articles will highlight leading-edge technologies that incorporate these features to integrate across workflows, accelerate timelines and reduce COGS – along with how these approaches are enabling the biopharmaceutical industry to cross the finish line faster with new treatment options for patients in need.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Amgen VP of R&D David Reese

Am­gen rolls out da­ta for KRAS in­hibitor com­bo study in col­orec­tal can­cer, hop­ing to move on from ug­ly ear­ly re­sults

With the first win for its KRAS inhibitor sotorasib in hand, Amgen is pushing ahead with an aggressive clinical plan to capitalize on its first-to-market standing. The drugmaker thinks combinations — in-house or otherwise — could offer a path forward, and one early readout from that strategy is bearing fruit.

A combination of Amgen’s sotorasib and its EGFR inhibitor Vectibix posted an overall response rate of 27% in 26 patients with advanced colorectal cancer (CRC) with the KRAS-G12C mutation, according to data from the larger Phase Ib/II CODEBREAK 101 study set to present at this weekend’s virtual ESMO Congress.

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Dan O'Day, Gilead CEO (Jim Watson/AFP via Getty Images)

Eu­ro­pean study finds that Gilead­'s Covid-19 an­tivi­ral remde­sivir shows no clin­i­cal ben­e­fit

Gilead’s remdesivir — or Veklury, as it’s marketed in the US — raked in around $2.8 billion last year as the only FDA-approved antiviral to treat Covid-19. But new data from a European study suggest the drug, which has been given to about half of hospitalized Covid patients in the country, has no actual benefit.

The open-label DisCoVeRy trial enrolled Covid-19 patients across 48 sites in Europe to test a handful of treatments, including remdesivir, lopinavir–ritonavir, lopinavir–ritonavir and interferon beta-1a, and hydroxychloroquine. To participate, patients had to show symptoms for seven days and require oxygen support. A total of 429 patients were randomized to receive remdesivir plus standard of care, while 428 were assigned to standard of care alone.

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Gri­fols drops $1B on Ger­man hold­ing com­pa­ny in con­tin­ued plas­ma push

One Spanish biotech is beefing up its plasma therapy operations, and on Friday, it announced that it’s doing so in a billion-dollar deal.

Grifols is now the largest shareholder of Biotest, a company valued at more than $1.8 billion. By teaming up, the two will try to increase the number of plasma therapies available and increase patient access around the world, Grifols said in a press release.

The company did so by acquiring holding company Tiancheng Pharmaceutical, the Germany-based owner of nearly 90% of Biotest shares, for nearly $1.27 billion. Grifols now owns nearly 90% of Biotest voting rights and almost 45% of the total share capital of Biotest.

Covid-19 roundup: FDA re­veals boost­er ad­comm ques­tion; Eli Lil­ly's an­ti­body cock­tail cleared for pre­ven­tion

The FDA released briefing documents this week from the agency and Pfizer each outlining their arguments for today’s Covid-19 booster shot adcomm, but one thing conspicuously missing was the question on which panel members would be voting. But late Thursday night, regulators published that question.

Adcomm members will be asked whether or not the safety and efficacy data from Pfizer/BioNTech’s original Phase III study “support approval” of a booster shot at least six months after the second dose in individuals older than 16. The question notably excludes the real-world data from Israel and other analyses that Pfizer and the Biden administration had said would be a centerpiece of their arguments for boosters.

A Pfiz­er part­ner wel­comes ex-ADC Ther­a­peu­tics CMO Jay Fein­gold to the team; Amid tough sled­ding, Im­muno­vant choos­es Eli Lil­ly alum as CFO

→ Last week we told you about the CMO revolving door at ADC Therapeutics, as Joseph Camardo replaced the departing Jay Feingold. The next opportunity for Feingold in the CMO slot has opened up at antibody-drug conjugate and mAb developer Pyxis Oncology, which has added several new execs and scientific advisory board members in recent months, including ex-Immunovant CFO Pamela Yanchik Connealy. Before his tenure at ADC, Feingold was Daiichi Sankyo’s VP of US medical affairs and chairman of the Global Medical Affairs Oversight Committee. Within weeks in March, Pyxis struck a licensing deal with Pfizer for two of its ADCs and raked in $152 million from a Series B round.

Carl Hansen, AbCellera CEO

Look­ing for the next mR­NA break­through, Mod­er­na taps Ab­Cellera in mys­te­ri­ous an­ti­body dis­cov­ery deal

Moderna’s success with its Covid-19 vaccine has busted the dam open on a range of potential mRNA therapeutics — and now the biotech is pushing to keep the cutting edge in-house. A new partnership with standout antibody discovery outfit AbCellera could help keep the ball in the competition’s court.

Moderna will partner with antibody player AbCellera on up to six undisclosed targets for a range of mRNA-encoded drugs harvested from AbCellera’s bustling discovery platform, the companies said Wednesday.

No­vo Nordisk Foun­da­tion tees up $47.5M to ex­plore the dri­vers of ge­net­ic dis­ease with the Broad In­sti­tute

The Broad Institute of Harvard and MIT played a significant role in mapping out genes as part of the Human Genome Project about two decades ago. Now, it’s joining forces with one of the industry’s largest research foundations in an effort to translate those maps.

The Novo Nordisk Foundation, which operates independently from the biotech Novo Nordisk, is teeing up $47.5 million to work with the Broad on mining genetic data in the hopes of better understanding how variants drive disease.