CinFina Pharma Announces FDA Clearance of Investigational New Drug Application and First Participants Dosed in Phase 1 Trial of CIN-110 for the Treatment of Obesity

CIN-110 is a potent and highly selective large molecule peptide YY (PYY3-36) with an extended half-life being developed as a monotherapy and co-administration agent to induce satiety, reduce appetite and impact glucose metabolism

CIN-110 is the second therapeutic in CinFina’s metabolic portfolio to reach the in-human clinical trial milestone

CINCINNATI – March 26, 2024 – CinFina Pharma, a CinRx portfolio company dedicated to advancing a portfolio of high-impact treatment options for obesity and metabolic diseases, announced the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug Application (IND) for CIN-110, a PYY3-36 analog, allowing the first in-human clinical study to proceed. With the commencement of the trial, CinFina also announced the first cohort of participants has been dosed. The trial will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and immunogenicity of CIN-110 in a randomized, double-blind, placebo-controlled, single ascending dose study in otherwise healthy subjects with obesity.

“Despite the emergence of new medicines to help address the obesity epidemic, there remains an unmet medical need for therapeutics that are well tolerated for long-term treatment,” said Dr. Jon Isaacsohn, Founder and Chief Executive Officer at CinRx Pharma. “As demonstrated by promising preclinical efficacy data, CIN-110 has the potential to meet these needs and provide important therapeutic advantages to patients in monotherapy and combination settings, including durability of weight loss. We look forward to continuing to explore its safety and tolerability profiles in this important Phase 1 study.”

CIN-110 is a stable and long-acting analog of  PYY3-36 being developed both as a monotherapy and co-administration agent for obesity. PYY3-36 is an endogenous hormone secreted in the gut, which activates the neuropeptide Y2 receptor (Y2R) to reduce appetite and food intake. Unlike prior attempts at developing a PYY focused therapeutic, CIN-110 is uniquely designed to limit severe nausea and emesis and still deliver on the promise of effective, long-term weight loss. CIN-110 selectively binds and activates Y2R, the target for native PYY3-36. Pharmacodynamic evaluation of CIN-110 demonstrated that repeat subcutaneous dosing leads to significant reduction of food intake and body weight in obese, non-human primates and rodents. Additionally, data shows CIN-110 led to an improvement of glucose homeostasis and insulin sensitivity in animal models. The toxicology program results indicated that CIN-110 was well tolerated with no adverse findings noted in either non-human primates or rodents. The results from the preclinical efficacy and safety studies support the progression of CIN-110 to human clinical trials.

 

“The preclinical program of CIN-110 underscores the potential for PYY to be a differentiated approach to treat obesity. This uniquely engineered molecule holds promise to expand the current therapeutic paradigm beyond GLP-1s with its selectivity, potency and half-life extension,” said Brian Murphy, M.D., Chief Medical Officer at CinRx Pharma... CinFina Pharma's Press Release -

Tetra Pharm Technologies advances CB1 antagonist, TPT0701, for appetite suppression into preclinical testing

The Danish biopharmaceutical company, Tetra Pharm Technologies, proudly announces a significant milestone in the realm of appetite suppression research having developed a cutting-edge compound leveraging their innovative platform technology.

COPENHAGEN, Denmark, Feb. 8, 2024 -- Following the successful filing of Intellectual Property (IP) for their applied platform technology targeting the endocannabinoid system (ECS), Tetra Pharm Technologies, is moving forward with its compound for appetite suppression, TPT0701.

Endocannabinoid system modulation has long been recognized as a promising avenue for appetite regulation. Through rigorous research and development efforts, Tetra Pharm Technologies has unlocked the potential of this biological system to address one of the most pressing health challenges of our time – excessive appetite and weight gain.

TPT0701 was originally designed in 2022 for obese patients suffering from schizophrenia but will now move into the development stage as appetite suppressing medication for general weight loss.

Dr. Morten Allesø, Chief Scientific Officer, Tetra Pharm Technologies, said: "With our new, proprietary platform technology as our secret weapon to unlock the endless possibilities of the endocannabinoid system, we are now ready to finalize TPT0701 for pre-clinical testing in 2024".

"Compounds, such as TPT0701, display physicochemical properties that render them difficult to formulate by means of conventional approaches. Our enabling platform technology, now with an IP application supporting it, can accommodate exactly these types of molecules and will help overcome otherwise hampering drug delivery challenges such as poor absorption and ultimately low bioavailability. Indeed, a drug is only as good as its delivery system, and with a check mark secured on both the formulation technology and the pharmacological mode of action established in early drug discovery, we are confident in the ongoing success of TPT0701 as it progresses through development," added Morten Allesø.

TPT0701 offers a novel approach to appetite suppression, aiming to provide individuals with a safe and effective means to manage their cravings and achieve their wellness goals. By specifically targeting the endocannabinoid system, Tetra Pharm Technologies' solution holds promise for combating obesity and related metabolic disorders, with far-reaching implications for public health.

"Obesity and its associated health risks have reached alarming levels worldwide, necessitating innovative approaches to address this pressing issue. Research and development are our top priority, and we will continue to invest in building our pipeline of new drug candidates targeting the endocannabinoid system and related disease indications", says Martin Rose, Chief Executive Officer, Tetra Pharm Technologies... Tetra Pharm Technologies' Press Release -

Scholar Rock Announces FDA Clearance of IND Application to Initiate Phase 2 Proof-of-Concept Trial with Apitegromab to Treat Obesity

• Randomized Phase 2 proof-of-concept trial with apitegromab in patients with obesity on GLP-1 therapies on track to initiate mid-2024
• Study aims to show the importance of selective myostatin inhibition in preservation of lean muscle mass as part of healthy, safe weight loss management
• Scholar Rock is also developing SRK-439, a novel investigational selective myostatin inhibitor, optimized for the treatment of obesity

CAMBRIDGE, Mass.--Jan. 23, 2024-- Scholar Rock (NASDAQ: SRRK) (the Company), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for its Phase 2 proof-of-concept trial of apitegromab to treat obesity in patients taking a GLP-1 receptor agonist (GLP-1 RA).

The Phase 2 trial is a randomized, double-blind, placebo-controlled, multi-center study to evaluate the effect of apitegromab, a highly selective myostatin inhibitor, to safely preserve lean muscle mass as an adjunctive therapy in overweight and obese adults who are taking a GLP-1 RA. Trial initiation is on track for mid-2024, and data from the apitegromab Phase 2 trial are expected in mid-2025. In parallel, Scholar Rock is developing SRK-439, a novel investigational selective myostatin inhibitor, optimized for the treatment of obesity. The Company plans to file an IND for SRK-439 in 2025.

“The FDA’s acceptance of our IND application to study apitegromab in obesity allows us to assess the effect of our highly selective myostatin inhibitor on preserving lean muscle mass, and safety and tolerability of our approach when combined with a GLP-1 RA. The IND builds on our encouraging apitegromab clinical and safety data to date and allows us to inform the development of our cardiometabolic program with SRK-439, a novel preclinical selective myostatin inhibitor optimized for development in cardiometabolic disorders,” said Jay Backstrom, M.D., MPH, President and Chief Executive Officer of Scholar Rock. “Maintaining lean mass during weight loss is important to overall metabolic health, and we look forward to initiating the Phase 2 proof-of-concept trial in apitegromab to validate our differentiated approach of selectively targeting only the pro- and latent forms of myostatin to retain muscle mass.”... Scholar Rock's Press Release -

Rhythm Pharmaceuticals Announces Completion of Screening for Enrollment in Setmelanotide Phase 3 Hypothalamic Obesity Trial and Additional Updates

-- Phase 3 hypothalamic obesity top-line data expected in 1H2025 --

-- Positive reimbursement decision achieved for IMCIVREE® (setmelanotide) to treat patients with Bardet-Biedl syndrome and POMC/LEPR deficiencies in Spain --

-- IND application for RM-718 accepted by the FDA --

BOSTON, Jan. 04, 2024 -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, provided a business update and announced it has completed screening for enrollment in the pivotal, Phase 3 clinical trial evaluating setmelanotide in patients with acquired hypothalamic obesity.

Rhythm closed screening and anticipates overenrolling the ongoing phase 3 hypothalamic obesity trial with more than 140 patients consented and going through active screening and baseline assessments. In this trial, patients with acquired hypothalamic obesity aged 4 years or older will be randomized 2:1 to setmelanotide therapy or placebo for a total of 60 weeks, including up to eight weeks for dose titration. The Company expects to obtain top-line study results in H1 2025.

“2023 was a truly transformational year for Rhythm, as we made excellent progress with the execution of our IMCIVREE® (setmelanotide) commercial plan while also advancing important development efforts to expand the number of patients living with hyperphagia and severe obesity that could potentially benefit from this therapy,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. “We believe the rapid over enrollment in less than one year and execution of our Phase 3 hypothalamic obesity trial is a strong indicator of the unmet need for an effective therapy for patients who have no approved therapeutic options and the community’s excitement around setmelanotide’s potential to make a meaningful difference in their lives.”... Rhythm Pharmaceuticals' Press Release -

Keros Therapeutics to Develop KER-065 for the Treatment of Obesity

Keros commenced a randomized, double-blind, placebo-controlled, two-part Phase 1 clinical trial to evaluate single and multiple ascending doses of KER-065 in healthy volunteers

Keros expects to report initial data from this Phase 1 clinical trial in the first quarter of 2025

Preclinical data showed potential proof-of-mechanism of KER-065 for the treatment of obesity

Keros believes these preclinical data support developing KER-065 for the treatment of obesity, and Keros plans to initiate a proof-of-concept trial of KER-065 in obese patients following completion of this Phase 1 clinical trial

LEXINGTON, Mass., Jan. 03, 2024 - Keros Therapeutics, Inc. (“Keros” or “we”) (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta (“TGF-ß”) family of proteins, announced that it plans to develop KER-065, a novel ligand trap designed to bind to and inhibit TGF-ß ligands, including myostatin (GDF8) and activin A, for the treatment of obesity.

“Obesity is a complex and chronic disease associated with numerous comorbidities and a growing prevalence in patients. We believe there is a need for additional treatment options, including one that leads to weight loss without an associated loss of muscle and a potential for frailty. Based on our preclinical data, we believe that KER-065 has the potential to treat obesity without those limitations, by increasing skeletal muscle, reducing fat mass through an increase in energy expenditure, improving insulin sensitivity and improving cardiac function,” said Jasbir S. Seehra, Ph.D., President and Chief Executive Officer of Keros. “To that end, we recently commenced our Phase 1 clinical trial evaluating KER-065 in healthy volunteers and, following its successful completion, plan to initiate a proof-of-concept trial of KER-065 in obese patients.”... Keros Therapeutics' Press Release - 

Resalis Therapeutics Raises €10 Million Series A to Complete First Clinical Trial for RES-010 in Obesity

January 04, 2024- TORINO, Italy- Resalis Therapeutics announced the closing of a €10 million ($11 million) Series A financing round led by Sunstone Life Science Ventures with participation from existing investors including Claris Ventures and angel investors. The proceeds will be used to initiate and complete the first-in-human Phase 1 clinical trial and reach Phase 2 readiness for Resalis’ lead program, RES-010, in obesity. RES-010 is a non-coding RNA-based compound designed to provide a disease-modifying approach in obesity with longer-lasting weight reduction and the ability to extend treatment durability in combination with approved therapeutics, such as GLP-1 receptor agonists. In conjunction with the financing, Claus Andersson, PhD, General Partner of Sunstone Life Science Ventures, will join Resalis’ Board of Directors.

“The successful close of our Series A is an important milestone for Resalis because it will enable us to reach the clinic and to achieve the next significant inflection point with our lead candidate, RES-010. With RES-010 we aim to provide a therapeutic that precisely reduces fat mass in obesity with the potential to extend the durability of existing therapies. We value the trust and support of our new and current investors, and eagerly look forward to this next phase of development,” said Alessandro Toniolo, CEO of Resalis Therapeutics.

“Resalis’ therapeutic approach is based on groundbreaking science that has rapidly led to the development of a lead candidate, now on the cusp of clinical evaluation. Preserving muscle mass is key to provide a sustained and meaningful clinical benefit, and we share the team’s vision that this non-GLP1/GIP therapeutic will significantly improve both effect and tolerability of today’s therapies. This investment underscores our belief in the company’s vision and the transformative potential of its lead candidate to make a meaningful impact for obese patients,” said Claus Andersson, General Partner at Sunstone Life Science Ventures.

Pietro Puglisi, Managing Partner at Claris Ventures and Chairman of the Board at Resalis Therapeutics, added: “In recent years, there has been remarkable progress in the treatment of metabolic disorders. Yet, there persists a critical need for innovative solutions that can both provide orthogonal therapeutic effects and pave the way for combination therapies. I look forward to the continued collaboration with the outstanding Resalis team and welcoming Claus as a new member of the Resalis Board.”

Resalis has a deep understanding of the field of ncRNAs and RNA-targeted therapeutics in human health and metabolic disorders. The company’s lead candidate, RES-010, is an antisense oligonucleotide that targets miR-22, a central player in the regulation of lipid metabolism and energy consumption. In multiple proof-of-concept studies in large and small animal models, RES-010 has shown the potential to provide a safe and longer-lasting therapeutic effect, alone or in combination with approved drugs. Resalis plans to initiate a Phase 1 clinical study evaluating the safety and efficacy of RES-010 in the first half of 2024. RES-010 is patent-protected in the US, Japan and China with patents pending in the EU... Resalis Therapeutics' Press Release [PDF] -