Ronald Park, Ambys Medicines CEO
When Ambys Medicines, a Third Rock Ventures-backed regenerative play, launched in 2018, it came with some serious fanfare for its liver disease-focused approach. Now, after years of quiet development, the company has a new trough of money to play with as it slowly approaches the clinic.
Ambys closed $107 million in total Series A funding — with a $47 million extension add — Tuesday with a lead program creating healthy versions of liver cells still more than a year from the clinic, the biotech said.
Ambys, which launched back in mid-2018 with a goal to rewrite the rules against severe liver disease using a broad range of modalities, says it’s now ready to bring its first shot at that mission to the clinic by Q2 2023. The program, dubbed AMI-918, is an off-the-shelf cell therapy looking to replace damaged liver tissue with healthy cells, Ambys said.
Meanwhile, the company is working on a follow-up program for which the details are still very slim. In a release, the company described the program this way:
Ambys’s second program is designed to bring the promise of its liver-cell platform to many more patients by providing extended durability of replacement cells and easier dosing and administration without the need for immunosuppressive therapy, which is intended to enable access to the broader population of patients with chronic liver disease. This follow-up program aims to slow disease progression and ultimately reverse disease in patients with decompensated liver disease. This advanced technology has the potential to benefit more than a half-million liver disease patients in the U.S. alone each year.
Ambys launched with much fanfare and $140 million in backing from Third Rock Ventures as part of a regenerative plan of attack against liver disease. Third Rock has once again led this round of funding with participation from Takeda, Schroders Capital, Laurion Capital, Smilegate Investment, and Alexandria Venture Investments.
Named after the salamander Ambystoma mexicanum famed for its regenerative abilities, the biotech promised to leverage both cell and gene therapy approaches in its pipeline. In its years since launch, Ambys has erected a clinical-scale manufacturing facility for its cells the biotech says has allowed it to produce its off-the-shelf cells at scale — a significant logistical hurdle for the company’s future chances.
‘When we started this effort, it was a moonshot, given the tremendous challenge of producing the types of cells needed at scale,’ CSO Markus Grompe said in a release. ‘With the opening of our production facility earlier this year, we are well on our way to overcoming that hurdle and will be able to produce high-quality, mature liver cells to support clinical development and early commercialization activities.’
CALQUENCE is a registered trademark of the AstraZeneca group of companies.
At the 2021 American Society of Hematology (ASH) Annual Meeting & Exposition, blood cancer researchers from around the world gathered virtually to discuss the progress that has been made in the field of hematology. Over the past decade, that progress has been tremendous. We’ve seen not only breakthrough approaches to care, but also significant improvement upon existing novel treatments and exploring combinations within those medicines.1 These advances have transformed expectations of what a blood cancer diagnosis now means for patients. While we’ve come a long way, I believe the most exciting scientific discovery is yet to come, and that future advances will truly transform patient care.
Alzheimer’s disease researchers along with medical professors from Harvard and Johns Hopkins issued a formal statement Monday asking the FDA to quickly pull Biogen’s Aduhelm from the market.
‘An accelerated withdrawal would mitigate some of the harm of its unwarranted accelerated approval,’ they wrote to FDA, explaining how Aduhelm ‘did not meet the FDA’s own criteria for accelerated approval based on surrogate markers because amyloid plaque does not correlate well with symptoms, severity of disease or progression.’
Unlock this story instantly and join 126,200+ biopharma pros reading Endpoints daily — and it’s free.
Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Valeriano Di Domenico)
In a surprise move, Biogen announced Monday that it will cut the price of its controversial Alzheimer’s drug Aduhelm in half, slashing the cost from $56,000 to $28,000.
The sudden discount marks a sudden turnaround for the big biotech as it struggles to turn around a drug whose stuck-in-the-mud sales and political ramifications have sent the company into turmoil and triggered the ousting of its longtime chief scientist. Biogen’s leadership had resisted calls since June to reduce the price of the drug.
Unlock this story instantly and join 126,200+ biopharma pros reading Endpoints daily — and it’s free.
Angie You and Volker Schellenberger, Amunix
Sanofi is crashing the year-end M&A party with a deal of its own.
Immuno-oncology is the name of the game as it swallows Mountain View, CA-based Amunix for $1 billion upfront and up to $225 million in biobucks, tagging a suite of T cell engagers and cytokine therapies as well as a tech platform for making ‘conditionally activated biologics.’
‘The Amunix technology platform utilizes a next generation smart biologics approach to precisely tailor-deliver medicines to become active only in tumor tissues while sparing normal tissues,’ said Sanofi R&D chief John Reed, ‘thus bringing the promise of more effective and safer treatment options for cancer patients.’
Unlock this story instantly and join 126,200+ biopharma pros reading Endpoints daily — and it’s free.
Graphic: Alexander Lefterov for Endpoints News
Drug pricing reform has been a political football for years, with both Donald Trump and Joe Biden championing changes during their presidencies. Little has moved the needle on Capitol Hill, however, thanks in part to the drug industry’s powerful lobbyists.
In the most recent example, Democrats tried to allow Medicare to negotiate drug prices — an immediate non-starter for biopharma proponents. After months of negotiation, the measure fell apart in favor of provisions on a small subset of drugs that passed the House but marked a far cry from Biden’s promises and what many activists had hoped for. The bill, included as part of Biden’s broad social policy agenda, now appears dead after Democrats failed to secure 50 votes in the Senate.
Unlock this story instantly and join 126,200+ biopharma pros reading Endpoints daily — and it’s free.
The FDA may soon sign off on two new pills from Merck and Pfizer to help treat Covid-19 before those infected end up in the hospital, according to a Bloomberg report.
An announcement from the agency may come as early as Wednesday, according to three Bloomberg sources, which would create more options for those who are unvaccinated and need quick and early treatment.
Most recently, the FDA’s antimicrobial drugs advisory committee on Tuesday voted 13-10 in favor of the Merck pill’s benefits outweighing the risks for adults within 5 days of developing Covid symptoms. But Merck offered little explanation for why its potential Covid-19 antiviral was less effective in reducing Covid hospitalizations and deaths in a full analysis of a Phase III trial versus an interim look, and mutagenesis concerns remain.
New research from Johns Hopkins shows convalescent plasma may work in the outpatient space, which could be key as the Omicron variant has extinguished several monoclonal antibody treatments.
The results of this placebo-controlled, multicenter trial led by Hopkins researchers showed that convalescent plasma reduced the need for hospitalization among more than 1,000 outpatients with Covid-19 who participated.
David Ricks, Eli Lilly CEO (David Paul Morris/Bloomberg via Getty Images)
Just 6 days after Eli Lilly execs ID’d its $1.1 billion atopic dermatitis drug lebrikizumab as one of its top late-stage drug candidates, the pharma giant has followed up with another round of positive late-stage data as it paves the way to planned regulatory filings in 2022.
The Christmas week statement from Lilly included little by way of hard data — the big outfit typically reserves that for its science conferences — but researchers touted a clean sweep of the primary and all secondaries in its 3rd round of late-stage results.
Unlock this story instantly and join 126,200+ biopharma pros reading Endpoints daily — and it’s free.
Right as the new Omicron variant is poised to increase rapidly across the US, the federal government has effectively run out of the only monoclonal antibody treatment that works against it, and at least one major hospital system is now halting all mAb infusions.
Late last month, the federal government paused shipments of GlaxoSmithKline and Vir’s mAb treatment sotrovimab in order to conserve supplies of the only treatment that might work against the Omicron variant. Last week, however, HHS told Endpoints News that the move to hold back sotrovimab was unrelated to Omicron, and due to a surplus of Eli Lilly mAbs, which aren’t effective against Omicron.
https://endpts.com/third-rocks-ambys-closes-a-megaround-with-the-clinic-still-a-ways-off-for-its-regenerative-cells-for-liver-disease/
