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Home » Blueprint Says It’s Rare Disease Drug Could Be A Blockbuster
Innovation

Blueprint Says It’s Rare Disease Drug Could Be A Blockbuster

Press RoomBy Press Room22 February 20245 Mins Read
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Blueprint Says It’s Rare Disease Drug Could Be A Blockbuster

InnovationRx is your weekly digest of healthcare news. To get it in your inbox, subscribe here.

Around 22,000 people across the U.S. have a rare chronic condition called indolent systemic mastocytosis. It can manifest itself in a number of ways, from skin conditions to anaphylactic events to cognition, which can also make it challenging to get a diagnosis. Last May, the FDA approved the first-ever treatment: avapritinib, marketed by pharma company Blueprint Medicines as Ayvakit. It was a watershed moment for the small community of patients that had previously only been able to treat specific symptoms and not the disease.

On its 4th quarter earnings call last week, Blueprint announced that it now has over 1,000 ISM patients prescribed with Ayvakit already. The drug, which lists for about $445,000 a year, brought in $204 million in revenue in 2023, nearly double the $110 million it brought in last year. (The drug is approved for other indications as well.) The company announced with its earnings that it expects between $360 – 390 million in product revenue for the drug this year, and the company expects this to keep growing over the next few years as the drug finds its way into the hands of patients.

“We see this as being a $2 billion plus market,” CEO Kate Haviland told Forbes. The reason for that being the number of patients who could benefit from the drug, particularly because Ayvakit actually treats the root cause of ISM, not just its symptoms, and so far, she added, the safety profile has proven to be better than the previous standard of care. “It’s so important for these patients, who didn’t realize how much they had narrowed their lives until they got on the therapy,” she said. “They’re able to reclaim their life again.”

Autism Care Startup Elemy Is Ditching Its Therapists To Focus On Software

In 2021, Elemy boasted a more than billion dollar valuation built on providing in-person autism therapy to kids. Now it’s changed its name to Tilly and is selling software to the therapists it used to employ. This is the third rebrand in five years for Sprout Therapy-Elemy-Tilly, which raised a $219 million Series B a led by SoftBank Vision Fund 2 at a $1.15 billion valuation.

Read more here.

Pipeline & Deal Updates

M&A: DarioHealth, a publicly-traded company that offers software to manage chronic conditions, acquired mental health software startup Twill for $10 million in cash and 10 million shares, worth around $24.4 million as of Tuesday’s close. In the first 9 months of 2023, Dario generated $16.7 million in revenues and Twill had $13.8 million.

Extension: 9amHealth, a startup that helps patients manage diabetes, obesity and other chronic conditions, raised a $9.5 million in a Series A extension led by Cigna Group Ventures.

Cancer Detection: Freenome, a biotech startup developing blood tests for early cancer detection, raised $254 million in a financing round led by Roche.

Artificial Intelligence: UnityAI, a startup cofounded by the former chief data officer of HCA Healthcare Edmund Jackson, has raised a $4 million seed round led by Max Ventures to apply AI to improve hospital workflow.

Robotics: Surgical robotics company Medical Microsinstruments Inc. announced that it has raised a $110 million series C round led by Fidelity Management & Research Company. The financing is geared towards the commercialization of its surgical system as well as more clinical trials to expand its repertoire of surgeries.

Peptide Discovery: Biotech startup Insamo, which uses computational biology to discover new cyclic-peptide drugs, announced it has emerged from stealth with a $12 million seed round from firms including venBio, Merck’s venture arm and Playground Capital.

Cardiac Tech: Reprieve Cardiovascular, which aims to develop an automated fluid management system for heart failure patients, announced it has emerged from stealth with a $42 million series A round co-led by Sante Ventures and Lightstone Ventures.

Possible Long Covid Cause Identified: This Protein Might Be The Culprit—And Medication Might Cure It

Researchers have potentially linked an antiviral protein produced by the body during infection as the cause of long Covid, according to a new study, and the researchers are optimistic that medications can be developed to treat the condition.

Read more here.

Other Healthcare News

The FDA approved Xolair the first medication for treating allergic reactions to multiple foods, which was co-developed by Novartis and Genentech.

The first human implanted with a chip from Elon Musk’s computer-brain interface company Neuralink can move a computer mouse with thought alone, according to the tech billionaire.

GSK said its long-acting injectable HIV drug, Cabenuva, worked better than daily pills for some patients, according to interim results from a late-stage clinical trial.

Amazon will replace Walgreens Boots Alliance on the Dow Jones Industrial Average stock index.

The FDA issued a warning to consumers against using smart watches or rings to measure blood glucose.

A survey conducted by healthtech company Athenahealth found that 93% of physicians report feeling burned out on a regular basis, with a majority indicating they’ve considered leaving the profession.

Across Forbes

Green Hydrogen Could Help Save The Planet. This Startup Wants To Make It Cheap

This Miner Might Change The Future Of The iPhone

How RJ Scaringe Is Driving Rivian Straight At Elon Musk

What Else We are Reading

The Powerful Constraints on Medical Care in Catholic Hospitals Across America (KFF Health News)

Mind-reading devices are revealing the brain’s secrets (Nature)

Fearing prosecution, UAB pauses in vitro fertilization after Alabama embryo court ruling (AL.com)

Artificial Intelligence healthcare HealthTech Software venture capital
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