US biotech secures $150M to develop potential treatments for Parkinson’s
1 therapy, bevemipretide, was shown to ease brain inflammation in mice
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- Massachusetts-based biotech Mighty Therapeutics has raised $150 million to advance treatments for Parkinson's and other diseases.
- One drug in its pipeline is bevemipretide, which was shown to reduce brain inflammation in mice.
- The company's work targets the dysfunction of mitochondria, which are energy-producing structures in cells.
Mighty Therapeutics, a Massachusetts-based biotech company, announced that it has raised $150 million to support the clinical development of its treatment candidates for Parkinson’s disease and other neurodegenerative conditions.
The new funding is expected to expedite pipeline initiatives in Parkinson’s as well as ongoing late-stage drug development programs in other diseases, according to a company press release detailing the financing.
The biotech’s work focuses on mitochondria, small energy-producing structures inside cells that serve as their powerhouses, whose dysfunction has been linked to a number of conditions marked by nerve cell loss, including Parkinson’s and amyotrophic lateral sclerosis (ALS).
In Mighty’s pipeline is bevemipretide (SBT-272), a small molecule designed to improve mitochondrial function. In a mouse model of Parkinson’s, the treatment candidate was found to reduce markers of brain inflammation.
The developer says it will use the new funding to “progress commercial and clinical development of [a] new class of mitochondrial targeted medicines.”
“These financings … [support] our continued leadership of the burgeoning field of mitochondrial medicine across a broad range of therapeutic areas,” said Reenie McCarthy, Mighty’s CEO. “With our strong commercial momentum and promising pipeline, we are poised for long-term growth and continued patient impact.”
Parkinson’s is caused by the progressive loss of dopaminergic neurons, which are nerve cells that produce dopamine, a signaling molecule that plays a role in voluntary movement. Several mechanisms are thought to contribute to neuronal damage and loss in Parkinson’s, including the accumulation of toxic clumps, or aggregates, of misfolded alpha-synuclein protein and mitochondrial dysfunction.
Misfolded alpha-synuclein can bind to cardiolipin, a fatty molecule found in the inner membrane of mitochondria, reducing their ability to produce energy.
Bevemipretide being developed as a treatment for Parkinson’s and ALS
The company is developing bevemipretide as a treatment for Parkinson’s and ALS, another progressive neurological disorder that damages nerve cells and causes disability. The small molecule targets cardiolipin, which is expected to improve mitochondrial function and to prevent oxidative stress, a process that causes cellular damage and is also thought to contribute to Parkinson’s.
Results from its use in a mouse model of Parkinson’s demonstrated that bevemipretide reduced alpha-synuclein clumping, increased dopaminergic neuron survival, and lowered the levels of markers of brain inflammation.
In an early clinical trial testing an under-the-skin, or subcutaneous, injection formulation of bevemipretide in healthy volunteers, interim data showed several doses were safe and well tolerated. Doses expected to safely achieve therapeutic concentrations in the brain were selected for further evaluation.
The preclinical development of bevemipretide was supported by a two-year grant from The Michael J. Fox Foundation for Parkinson’s Research.
The newly announced funding comes from two sources. First, the company secured access to up to $125 million in financing through an agreement with K2 HealthVentures, including an initial $30 million. Second, the company received a $25 million investment from its founding investor, Morningside, as part of the first round of its Series B fundraising.
“We are deeply gratified by Morningside’s ongoing support, and very excited to partner with K2 HealthVentures to accelerate and broaden our efforts to address the unmet medical needs of the many individuals living with diseases of mitochondrial dysfunction,” McCarthy said.
The U.S. Food and Drug Administration has granted orphan drug status to bevemipretide for ALS. That designation aims to accelerate the development of new treatments for rare diseases, offering regulatory support and certain financial incentives.
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