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Drugs for the rare hormone disorder acromegaly include older engineered peptides administered as frequent injections. A new FDA approval brings acromegaly patients the choice of a once-daily pill that also gives its developer, Crinetics Pharmaceuticals, the opportunity to compete with and take market share from blockbuster products that are well-established in the acromegaly space.
The new drug, paltusotine, is the first commercial product for Crinetics, which specializes in developing therapies for endocrine disorders. The late Thursday regulatory decision covers the pill’s use as a first-line acromegaly treatment, putting it on par with the first-line injectable peptides currently sold by Novartis and Ipsen. San Diego-based Crinetics will market its new product under the brand name Palsonify.
Acromegaly develops when the pituitary gland produces too much growth hormone, resulting in skeletal changes such as joint pain and abnormally large hands and feet. This chronic disease also causes problems in organs such as the heart and lungs. Acromegaly is typically caused by the growth of a benign tumor on the gland. Surgical removal of the tumor is the standard first treatment option and can be curative. Still, the pituitary gland’s location in the brain makes this surgery difficult. For those who either aren’t eligible for surgery or had an unsuccessful surgery, drugs are the next choice.
In the body, growth hormone is suppressed by another hormone called somatostatin. The first-line drugs from Novartis and Ipsen are peptides engineered to be analogs of somatostatin. These drugs are also approved to treat neuroendocrine tumors, which are most commonly found in the gut. Novartis’s two formulations of the drug octreotide, brand name Sandostatin, accounted for more than $1.2 billion in sales in 2024 while Ipsen’s lanreotide, brand name Somatuline, generated €1.1 billion (about $1.3 billion) in 2024 revenue. But both products are older drugs now facing generic competition.
Crinetics’s Palsonify is a small molecule designed to bind to and activate somatostatin receptor type 2 nonpeptide (SST2), the receptor that native somatostatin binds to. The company evaluated this drug in two placebo-controlled Phase 3 studies. The main goal of both was achieving biochemical control, defined as growth hormone levels within the normal range.
In the first trial, which enrolled 111 adult acromegaly patients, 56% of participants who received the study drug achieved biochemical control at 24 weeks compared with 5% of those in the placebo group. The second study enrolled 58 adults previously treated with other drugs and switched to Palsonify. Results at 36 weeks showed 86% of patients who switched to the study drug maintained biochemical control compared to 4% of those in the placebo arm.
The Crinetics drug was well tolerated by patients. No serious adverse events were reported; the most common side effects included gastrointestinal problems, such as nausea, diarrhea, and loss of appetite. Crinetics said these problems were classified as mild to moderate and typically lasted between six and 18 days. Furthermore, the company said no patients who received Palsonify showed clinically significant increases in tumor volume while clinically significant decreases were observed in four patients. No such change was reported for the placebo groups.
Prior to Palsonify’s approval, the only pill available for acromegaly was Chiesi Farmaceutici’s oral version of octreotide, branded as Mycapssa. But the FDA’s 2020 approval of this drug only covered its use as a maintenance treatment for patients who have responded to the injectable Novartis or Ipsen drugs. For acromegaly patients who don’t respond to these therapies, the next-line of treatment is injectable drugs that block growth hormone.
Crinetics points to research indicating that the available peptide drugs are inadequate for achieving biochemical control and managing disease symptoms. The company’s executives believe Palsonify can capture patients looking to switch from currently available therapies. Chief Commercial Officer Isabel Kalofonos said real-world data show that 80% of patients on injectable acromegaly drugs discontinue or switch therapies within five years, reflecting the inadequacy of current products.
“Unlike other treatments, Palsonify is the first and only once daily, oral SST2 selective agonist that has the potential to achieve rapid biochemical and symptom control that is consistently maintained over time based on the Phase 3 data,” Kalofonos said during a Thursday evening conference call.
Palsonify’s annual list price is $290,000, which Kalofonos said is within the price range of other acromegaly products. The company expects the drug will launch in early October.
In a note sent to investors Friday, Leerink Partners analyst Joseph Schwartz referenced the biblical story of David and Goliath to illustrate the prospects of Crinetics in acromegaly. Palsonify’s label and attractive price position the company to challenge the pharma goliaths in this market, he said. Schwartz added that the drug’s broad label, which includes patient-reported outcome benefits, will likely resonate with clinicians.
“Management appeared confident in Palsonify’s potential to capture market share over time, though they anticipate a gradual launch as patients navigate the appointment system to consult with their physicians,” Schwartz said. “Additionally, management detects a potential shift in the acromegaly market, with legacy players scaling back their support for older treatments like octreotide and lanreotide. This could open the door for new entrants like [Crinetics] to gain traction with the first new treatment in decades.”
A submission for Palsonify is still under review in Europe, where a regulatory decision is expected in the first half of 2026. The company has partnered with Sanwa Kagaku Kenkyuso to develop and commercialize the drug for acromegaly in Japan. Growth of the Crinetics product could also come from indication expansion. A Phase 3 study is underway in carcinoid syndrome associated with neuroendocrine tumors.
Photo by Crinetics Pharmaceuticals
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