Just weeks after MeiraGTx touted their latest update on data emerging from their gene therapy alliance with J&J on inherited retinal diseases, the pharma giant is back with an acquisition tailored to beef up their R&D efforts in the field.
This deal is focused on HMR59, an early-stage gene therapy at a small biotech called Hemera Biosciences. They’re keeping the terms under wraps for now, but J&J is grabbing a once-and-done gene therapy that is designed to protect levels of CD59, a key protein that guards the retina.
Very little is on display about this Waltham, MA-based biotech. The only drug they refer to on their website is HMR59. There’s no word what, if anything, is left to work on. According to LinkedIn, Adam Rogers, who has an MD from Emory, launched the company 10 years ago.
By protecting CD59 in the eye, they believe the treatment can play a big role protecting the vision of patients suffering from geographic atrophy, a severe form of age-related macular degeneration.
J&J based the deal on the Phase I study that’s been completed, with another early-stage study now in the clinic focused on safety.
There’s been considerable work launched for gene therapies in this field since Spark — now owned by Roche — won a pioneering OK for their treatment for Leber congenital amaurosis, a rare genetic retinal disease 3 years ago. Just last summer Biogen added to that with an in-licensing pact on a gene therapy for inherited retinal diseases.
“Geographic atrophy is a devastating form of AMD that impacts the ability to accomplish everyday tasks, such as reading, driving, cooking, or even seeing faces,” said James List, Janssen’s global therapeutic area head, cardiovascular & metabolism. “Our aim with this novel, single-administration gene therapy is to use our development expertise and deep heritage in vision care to help improve patient outcomes by intervening early, halting the progression to blindness, and preserving more years of sight.”
In 2006, a clinical trial called ILLUMINATE halted abruptly. Trial subjects treated with an investigational drug called torcetrapib started experiencing unexpected cardiovascular problems, in some cases resulting in death. After 15 years and nearly 1 billion dollars, the development of torcetrapib froze.
Nine years later, a paper published in the journal Circulation explained how the ILLUMINATE disaster could have been predicted and prevented if just 9 proteins had been measured in clinical trial participants following initial treatments.
In the oncology world, there’s no better hunting ground for cancer R&D execs than Genentech. The biotech franchise at Roche has worked on some of the leading drugs in the field, proven themselves with blockbuster returns, and carries weight for whatever it says and does.
The exodus of R&D talent out of the South San Francisco hub is a testament to their success.
Now one of their top research execs has been raided by a top China biotech player to satisfy not just their need for an oncology R&D chief as they build up their muscle in discovery and drug development, but also add a spotter for new cancer drug deals.
Pfizer and BioNTech have scored a historic OK for their Covid-19 vaccine in the UK, where the first doses are expected to be delivered within days.
Days after the British government authorized its Medicines & Healthcare Products Regulatory Agency to make a call independent of the European Union — from which it’s in the process of exiting — the regulators gave the green light for emergency use of BNT162b2.
Two years after tiptoeing into the protein degradation space with a discovery pact centered on neurodegeneration, AbbVie is wading deeper to explore the more common applications in cancer and immunological diseases.
The pharma giant is handing Frontier Medicines $55 million upfront to get things started while promising to reimburse its preclinical R&D costs. In addition to drug candidates that target E3 ligases — a key member of the body’s natural degradation system — Frontier will also be scouting small molecule binders to targets.
After recording close to a billion dollars in investment earnings over 9 months, driven mostly by Moderna’s soaring stock, Merck is cashing out.
The company sold its stake in $MRNA — which it’s held since 2015 — in the first half of the quarter, causing a temporary 2.5% dip in Moderna’s share price before the market opened on Wednesday. By late morning, the stock was back up 3.4%. The news comes just two weeks before an FDA advisory committee meeting to discuss Moderna’s application for emergency use of its Covid-19 vaccine.
Eighteen months after Exelixis signed on to partner with Iconic Therapeutics on an antibody-drug conjugate, more money is flowing.
Exelixis is picking up its exclusive option on the tissue factor-targeting ADC now known as XB002 (formerly ICON-2), Iconic’s lead program. Exelixis is adding $20 million to the $7.5 million it paid upfront. Part of that goes to ZymeWorks, which assisted with their ZymeLink ADC tech.
Six years after Merck and Bristol Myers Squibb captured the attention of the oncology world with the first approval of their PD-1 drugs Keytruda and Opdivo, sales revenue has started to level off after a host of rivals joined the hunt for new OKs for metastatic conditions, where the FDA has proven quick to act.
But a key analyst covering biopharma believes that there’s a vast, still largely untapped frontier for new approvals to come in the adjuvant setting that could once again ignite the growth of these leading cancer franchises. And once again, he’s pointing to the 2 leaders in the field as the most likely players to come out ahead — way, way ahead.
Roughly five weeks after buying out Asklepios — also known as AskBio — for up to $4 billion, Bayer is hammering home its focus on cell and gene therapies.
The Big Pharma announced Wednesday that it is establishing a cell and gene therapy platform within its pharmaceuticals division in order to consolidate all of its related projects under one umbrella. Researchers will continue working and developing experimental drugs on their own, Bayer said, while the execs will lay out and define the overall strategy and look to swing some more deals.
Roche and Blueprint Medicines are continuing to put pressure on rivals Eli Lilly and Loxo, as their Gavreto drug picked up a new approval Wednesday.
The companies announced that the FDA has given the thumbs-up for Gavreto to treat RET-mutation and RET-fusion positive thyroid cancers, less than three months after the drug received its first approval in non-small cell lung cancer with RET fusions. Gavreto is now set to fully compete with Lilly’s Retevmo after the latter’s brief period as the only developer with an OK’ed medicine for this specific gene alteration.
通过在眼睛中保护CD59，他们认为这种治疗可以在保护患者的视力方面起到很大的作用，这是一种不同形式的年龄相关的Mac Degraphic Atrophy（一种年龄相关的Mac Degraphic）。
自从Spark（现为罗氏公司所有）3年前因治疗Leber Congeni-tal Amaurosis（一种罕见的遗传病）而获得PioNeering OK后，该领域的基因治疗就开始了一些可持续的工作。就在去年夏天，mer bio-gen公司又加入了一项针对她的视网膜疾病的基因治疗的协议。