Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)
AstraZeneca — or, more specifically, its rare disease subsidiary Alexion — is serious about getting into ATTR amyloidosis.
Just weeks after licensing a late-stage antisense candidate from Ionis, AstraZeneca has struck another deal to pick up a Phase Ib antibody hitting the same target, this time from Swiss biotech Neurimmune.
The upfront from Alexion comes in at $30 million, with the potential to add up to $730 million in milestones. Alexion is hoping the program would tackle transthyretin amyloid cardiomyopathy, or ATTR-CM, which is characterized by cardiac buildup of toxic amyloid fibrils.
As ATTR-CM can lead to progressive heart failure and be fatal, the deal fits into AstraZeneca’s broader ambitions to become the leader in heart failure, integrating into a portfolio that also features the SGLT2 inhibitor Farxiga. Eplontersen, the antisense compound it scored from Ionis weeks ago for $200 million in cash, is also being tested in ATTR cardiomyopathy (in addition to ATTR polyneuropathy). ‘With 30 years of experience in developing medicines for people with rare diseases, Alexion is uniquely positioned to advance innovative science for small patient populations who are frequently underdiagnosed,’ CEO Marc Dunoyer said.
Dubbed NI006, the Neurimmune drug specifically targets misfolded transthyretin and is designed to spur the removal of amyloid fibril deposits in the heart.
Neurimmune is known for its other amyloid-clearing antibody, the FDA-approved Alzheimer’s drug Aduhelm, which was still known as aducanumab when it was licensed to Biogen. It will be responsible for completing the current Phase Ib trial, with Alexion shouldering part of the costs before taking over clinical development, manufacturing and commercialization.
But Alexion will have plenty of catching up to do. Pfizer’s Vyndaqel and Vyndamax (two formulations of the same transthyretin-stabilizer, tafamidis) were approved two and a half years ago for ATTR-CM, and together they brought in more than $1 billion in sales just over the first nine months of 2021. Alnylam is also far ahead in testing its suite of RNAi therapies for the indication.
In a statement, Alexion noted that ‘multiple mechanisms of action’ will likely be required to address the needs of patients with various types and levels of severity of amyloidosis.
Their Staying Power Lies in their Patient-Centricity
Decentralized clinical trials (DCTs) were traditionally utilized in an isolated fashion prior to the COVID-19 pandemic. To continue their research within the constraints of the pandemic, sponsors and clinical investigators pivoted to a decentralized model out of necessity. At the onset, regulatory agencies offered some guidance on the digital approaches that are acceptable to ensure DCT approaches are applied in a way that maintains patient safety, as well as data quality and integrity.
A district court in Texas will likely tie up the FDA’s FOIA office for months, as the court ruled late Thursday that the agency must release all documents related to its review of Pfizer-BioNTech’s Covid-19 vaccine.
The order from district judge Mark Pittman, handed down late Thursday, notes that while the Court recognizes the ‘unduly burdensome’ challenges that this FOIA request may present to the FDA, there also ‘may not be’ a more important issue at the FDA right now than the pandemic, the Pfizer vaccine, getting every American vaccinated, and making sure to the American public that the process was not rushed.
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Exscientia CEO Andrew Hopkins and Sanofi CEO Paul Hudson
Drug R&D has for years had an abysmal track record of success, with the vast majority of drug candidates never making it to market. The promise of AI to shorten the discovery time for new drugs and up their chances of success has more big drugmakers buying in — and Sanofi is the latest.
Sanofi will pay $100 million upfront with a potential $5.2 billion in downstream milestones for access to up to 15 small molecule drugs from Exscientia, a red-hot UK deep learning company at the forefront of the so-called ‘AI-discovered’ drug R&D movement, the partners said Friday.
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Aligos Therapeutics took a beating Thursday after reporting it is stopping development on its lead program for chronic hepatitis B.
The biotech’s shares $ALGS closed down 57% and fell into penny stock territory Thursday, following a morning press release saying Aligos’ ALG-010133 program did not prove efficacious at the dose tested in a Phase I study. Additionally, the company concluded that higher doses were also unlikely to be effective, and ultimately decided to axe the candidate altogether.
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Belén Garijo, Merck KGaA CEO (Kevin Wolf/AP Images for EMD Serono)
Bursting at the seams and executing plans for swift expansion to support its manufacturing work for the mRNA vaccine out of Pfizer/BioNTech, Indianapolis-based Exelead has now been scooped up in a $780 million cash buyout deal.
Germany’s Merck KGaA, which bought out another mRNA manufacturer, AmpTec, early last year, has been beefing up its ops around lipids, which, in mRNA vaccines, play a key role in turning human cells into a mini—vaccine factories?
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After 18 years at the FDA and climbing the corporate ladder in biopharma, Vicki Goodman is now in the biotech C-suite for the first time. The new CMO and executive VP of product development for Exelixis started on Dec. 4, flying out to the biotech’s headquarters just outside sunny San Francisco before returning home and flying all the way back to Philly, where she is based.
Goodman got her passion for medicine as a young child — it didn’t surprise anyone that she majored in biochemistry before going through medical school and residency, finishing up in the early 2000s with an emphasis in internal medicine, medical oncology and hematology. But for her, there was an underlying desire to use science to help people and work on problems that impact people’s health.
Stéphane Bancel (AP Images, Boston Herald)
While an Omicron-specific booster of Moderna’s Covid-19 vaccine probably won’t be ready in the next couple of months, according to a Reuters report, CEO Stéphane Bancel says another shot will likely be necessary for the fall.
‘I still believe we’re going to need boosters in the fall of ’22 and forward,’ Bancel said at a Goldman Sachs-organized event, per Reuters.
The news comes days after Israel Prime Minister Naftali Bennett announced that a study conducted in the country suggests a fourth Covid-19 vaccine dose spurs a five-fold increase in antibodies just a week after being administered.
Roger Perlmutter, Eikon CEO
Roger Perlmutter hasn’t wasted any time since announcing his supposed retirement from Merck in October 2020. After leaving his perch as one of the most successful R&D chiefs in Big Pharma, he’s now snatching a cool half-billion dollars to develop ‘a battery of innovative tools’ for drug discovery at the young startup Eikon Therapeutics.
Eikon closed on a $517.8 million Series B round on Thursday morning, bringing the Hayward, CA-based company’s total raise to more than $668 million.
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Eli Lilly is beefing up its fleet of vehicles being deployed to carry drugs to the brain.
Enlisting Canada’s Entos Pharmaceuticals, Eli Lilly has grabbed rights to a suite of proteo-lipid vehicles (PLVs) as part of a research collaboration that spans multiple programs focused on diseases of the central and peripheral nervous system. Entos will receive an upfront of $50 million, part of it as an equity investment, to start developing PLVs for Lilly’s selection.
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