Crinetics Submits New Drug Application for Paltusotine for the Treatment of Acromegaly

Rhea-AI Summary

Crinetics Pharmaceuticals (Nasdaq: CRNX) has submitted a New Drug Application (NDA) to the FDA for paltusotine, a novel once-daily, oral treatment for acromegaly. Paltusotine is the first selectively-targeted somatostatin receptor type 2 nonpeptide agonist developed for long-term maintenance therapy of acromegaly. The NDA is supported by data from 18 clinical trials, including two successful Phase 3 trials that met all primary and secondary endpoints. These trials demonstrated that paltusotine was well-tolerated and effective in achieving biochemical control and patient-reported symptom control compared to placebo. Crinetics expects to receive notification from the FDA on the NDA status in December.

Positive

• Submission of New Drug Application (NDA) to FDA for paltusotine
• Successful completion of two Phase 3 trials meeting all primary and secondary endpoints
• Paltusotine demonstrated efficacy in biochemical control and symptom management
• Potential to be the first once-daily, oral treatment for acromegaly

Negative

• None.

Insights

Biotechnology Analyst

The submission of an NDA for paltusotine marks a significant milestone for Crinetics Pharmaceuticals. This once-daily, oral treatment for acromegaly could potentially disrupt the current treatment landscape, which primarily relies on injectable therapies. The comprehensive data from 18 clinical trials, including two successful Phase 3 studies, provides a strong foundation for FDA review.

Key points to consider:

• Paltusotine met all primary and secondary endpoints in Phase 3 trials
• The drug demonstrated efficacy in both untreated and previously treated patients
• If approved, it would be the first oral somatostatin receptor type 2 agonist for acromegaly

The potential market impact is substantial, given the current $4.14 billion market cap. Approval could significantly boost Crinetics' revenue and market position. However, investors should be aware that FDA review typically takes 10-12 months, with potential for delays or requests for additional information.

The broader implications for Crinetics are also noteworthy. Success with paltusotine could validate their drug development platform and potentially accelerate progress for other pipeline candidates targeting endocrine conditions. This could lead to long-term growth opportunities beyond acromegaly.

Endocrinologist

The development of paltusotine represents a potential paradigm shift in acromegaly treatment. Current standard therapies often involve monthly injections, which can be burdensome for patients. An oral, once-daily option could significantly improve treatment adherence and quality of life for those living with acromegaly.

The efficacy data from the Phase 3 PATHFNDR program is particularly encouraging:

• Biochemical control was achieved, indicating effective management of growth hormone and IGF-1 levels
• Patient-reported symptom control suggests meaningful clinical benefits
• The drug was well-tolerated, which is important for long-term management of a chronic condition

From a clinical perspective, paltusotine's selectivity for somatostatin receptor type 2 may offer a more targeted approach, potentially reducing off-target effects seen with broader-acting somatostatin analogs. This could translate to a better side effect profile and improved patient outcomes.

If approved, paltusotine could become a first-line treatment option, particularly for patients struggling with injectable therapies or those newly diagnosed. The potential to simplify treatment regimens could also reduce the burden on healthcare systems.

SAN DIEGO, Sept. 26, 2024 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for paltusotine, the first once-daily, oral, selectively-targeted somatostatin receptor type 2 nonpeptide agonist in development for the proposed treatment and long-term maintenance therapy of acromegaly.

“This NDA submission brings us one step closer to our goal of delivering a new generation of therapy that can help people living with acromegaly,” said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. “Based on the comprehensive data from the Phase 3 PATHFNDR program, we are excited about the significance of this potential advancement for the acromegaly community, as well as what it represents to Crinetics as a company. Paltusotine is the leading candidate of a deep, innovative pipeline – the first of many therapeutic candidates that have been purposefully designed in-house to transform the lives of people impacted by a wide range of endocrine conditions.”

The NDA is supported by data from 18 clinical trials, including two Phase 3 trials that evaluated paltusotine for the treatment of acromegaly in medically untreated and treated patients. All primary and secondary endpoints were met in both Phase 3 studies. Treatment with paltusotine was well-tolerated and resulted in biochemical control and patient reported symptom control compared to placebo.

Crinetics anticipates receiving notification from the FDA on the status of the NDA submission in December.

ABOUT PALTUSOTINE
Crinetics’ lead development candidate, paltusotine, is the first investigational once-daily, oral, selectively-targeted somatostatin receptor type 2 (SST2) nonpeptide agonist that has completed Phase 3 clinical development for acromegaly and is in Phase 2 clinical development for carcinoid syndrome associated with neuroendocrine tumors. It was designed by Crinetics with the goal of providing a once-daily, oral option for reliable and consistent control of acromegaly. In Phase 3 studies, once-daily, oral paltusotine maintained IGF-1 levels and symptom control in patients with acromegaly who were switched from monthly injectable medications (PATHFNDR-1) and rapidly decreased IGF-1 levels and symptom burden in medically untreated acromegaly patients (PATHFNDR-2). IGF-1 is the primary biomarker endocrinologists use to manage acromegaly patients. Results from the Phase 2 study in carcinoid syndrome will provide an opportunity for paltusotine to potentially demonstrate utility in an investigational, Phase 3 trial for another important indication related to the treatment of symptoms in patients with neuroendocrine tumors.

ABOUT ACROMEGALY
Acromegaly is a serious rare disease generally caused by a pituitary adenoma, a benign tumor in the pituitary that secretes growth hormone (GH). Excess GH secretion causes excess secretion of insulin-like growth factor-1 (IGF-1) from the liver. Prolonged exposure to increased levels of IGF-1 and GH leads to progressive and serious systemic complications, often resulting in bone, joint, cardiovascular, metabolic, cerebrovascular, or respiratory disease. Acromegaly symptoms include headache, joint aches, fatigue, sleep apnea, severe sweating, hyperhidrosis/oily skin, bone and cartilage overgrowth, abnormal growth of hands and feet, enlargement of heart, liver, and other organs and alteration of facial features. Uncontrolled acromegaly results in increased mortality and has a debilitating impact on daily functioning and quality of life.

Surgical removal of pituitary adenomas, if possible, is the preferred initial treatment for most people with acromegaly. Pharmacotherapy is used for people who are not candidates for surgery, or when surgery is unsuccessful in achieving treatment goals. Approximately 50% of people with acromegaly prove to be candidates for pharmacotherapy. Injectable somatostatin receptor ligands are the most common initial pharmacologic treatment; however, these drugs require monthly depot injections with large gauge needles that are commonly associated with pain, injection site reactions, and an increased burden on the lives of patients.

ABOUT CRINETICS PHARMACEUTICALS
Crinetics Pharmaceuticals is a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapeutics for endocrine diseases and endocrine-related tumors. Crinetics’ lead development candidate, paltusotine, is the first investigational once-daily, oral, selectively-targeted somatostatin receptor type 2 (SST2) nonpeptide agonist that has completed Phase 3 clinical development for acromegaly and is in Phase 2 clinical development for carcinoid syndrome associated with neuroendocrine tumors. Crinetics is also developing atumelnant (CRN04894), an investigational, first-in-class, oral ACTH antagonist, that is currently completing Phase 2 clinical studies for the treatment of congenital adrenal hyperplasia and Cushing’s disease. All of the company’s drug candidates are orally delivered, small molecule new chemical entities resulting from in-house drug discovery efforts, including additional discovery programs addressing a variety of endocrine conditions such as hyperparathyroidism, polycystic kidney disease, Graves’ disease (including thyroid eye disease), diabetes, obesity and GPCR -targeted oncology indications.

FORWARD-LOOKING STATEMENTS
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical facts contained in this press release are forward-looking statements, including statements regarding the expected timing of notification from the FDA regarding the status of the NDA submission for paltusotine for the treatment or maintenance of treatment of acromegaly in the United States, and the plans and timelines for the commercial launch paltusotine, if approved or the potential pathway for regulatory approval for the use of paltusotine as a treatment for carcinoid syndrome. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplates,” “believes,” “estimates,” “predicts,” “potential,” “upcoming” or “continue” or the negative of these terms or other similar expressions. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, including, without limitation, initial or topline data that we report may change following completion or a more comprehensive review of the data related to the clinical studies and such data may not accurately reflect the complete results of a clinical study, and the FDA and other regulatory authorities may not agree with our interpretation of such results; we may not be able to obtain, maintain and enforce our patents and other intellectual property rights, and it may be prohibitively difficult or costly to protect such rights; geopolitical events may disrupt Crinetics’ business and that of the third parties on which it depends, including delaying or otherwise disrupting its clinical studies and preclinical studies, manufacturing and supply chain, or impairing employee productivity; unexpected adverse side effects or inadequate efficacy of the Company’s product candidates that may limit their development, regulatory approval and/or commercialization; the Company’s dependence on third parties in connection with product manufacturing, research and preclinical and clinical testing; regulatory developments in the United States and foreign countries; the timing and outcome of research, development and regulatory review is uncertain, and Crinetics’ drug candidates may not advance in development or be approved for marketing; any future impacts to our business resulting from geopolitical developments outside our control; and the other risks and uncertainties described in the Company’s periodic filings with the Securities and Exchange Commission (SEC). The events and circumstances reflected in the company’s forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Additional information on risks facing Crinetics can be found under the heading “Risk Factors” in Crinetics’ periodic filings with the SEC, including its annual report on Form 10-K for the year ended December 31, 2023 and its Quarterly reports on Form 10-Q for the quarters ended March 31, 2024 and June 30, 2024. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Except as required by applicable law, Crinetics does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Investors:
Gayathri Diwakar
Head of Investor Relations
gdiwakar@crinetics.com
(858) 345-6340

Media:
Natalie Badillo
Head of Corporate Communications
nbadillo@crinetics.com
(858) 345-6075 

FAQ

What is the purpose of Crinetics' New Drug Application for paltusotine?

Crinetics has submitted a New Drug Application (NDA) to the FDA for paltusotine as a potential treatment and long-term maintenance therapy for acromegaly.

How is paltusotine different from existing acromegaly treatments?

Paltusotine is the first once-daily, oral, selectively-targeted somatostatin receptor type 2 nonpeptide agonist developed for acromegaly treatment, potentially offering a new and more convenient option for patients.

What clinical data supports the NDA for paltusotine (CRNX)?

The NDA is supported by data from 18 clinical trials, including two Phase 3 trials that met all primary and secondary endpoints, demonstrating paltusotine's efficacy and tolerability in acromegaly patients.

When does Crinetics (CRNX) expect to hear back from the FDA regarding the paltusotine NDA?

Crinetics anticipates receiving notification from the FDA on the status of the NDA submission for paltusotine in December.