melanocortin receptor

Two poster presentations demonstrate ANI’s commitment to research in membranous nephropathy carePRINCETON, N.J., Oct. 24, 2024 (GLOBE NEWSWIRE) -- ANI Pharmaceuticals, Inc. (ANI or the Company) (Nasdaq: ANIP) today announced that the Company will present new pre-clinical data highlighting its membranous nephropathy (MN) research during two poster presentations at the American Society of Nephrology (ASN) Kidney Week 2024 in San Diego, CA, October 23-27. "We are excited to share our data with the ASN 2024 community. These data shed light of the potential role of melanocortin receptors in the underlying disease pathologies within nephrotic syndrome,” said Mary Pao, MD, PhD, Chief Medical Officer of ANI “This research contributes to the development of valuable research tools and models for studying membranous nephropathy. The findings represent the first steps in ANI’s commitment to advancing the science underlying certain disease pathologies.” Poster Title: THSD7A-Associated Membranous Nephropathy Involves Both Complement-Mediated and Autonomous Podocyte InjuryPoster Session: Pathology and Lab Medicine - 1Abstract: FR-PO955Presentation Time: Friday, Oct. 25, 10:00 AM-12:00 PMLead Author: Jing Liu Poster Title: Activation of Podocyte-Specific MC5R Signaling by Melanocortin Therapy Protects Against THSD7A-Associated Membranous NephropathyPoster Session: Glomerular Diseases: Therapeutic StrategiesAbstract: SA-PO715Presentation Time: Saturday, Oct. 26, 10:00 AM-12:00 PMLead Author: Jing Liu The abstracts are available online at asn-online.org. For more information during the conference, visit the ANI Pharmaceuticals booth. About ANI Pharmaceuticals, Inc. ANI Pharmaceuticals, Inc. (Nasdaq: ANIP) is a diversified biopharmaceutical company serving patients in need by developing, manufacturing, and marketing high-quality branded and generic prescription pharmaceutical products, including for diseases with high unmet medical need. ANI is focused on delivering sustainable growth by scaling up its Rare Disease business, strengthening its Generics business with enhanced research and development capabilities, delivering innovation in Established Brands, and leveraging its U.S. based manufacturing footprint. For more information, visit www.anipharmaceuticals.com. Forward-Looking Statements This press release contains not only historical information, but also forward-looking statements made pursuant to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements represent the Company’s expectations or beliefs concerning future events, including statements regarding the benefits of the acquisition of Alimera Sciences. These forward-looking statements generally are identified by the words “believe,” “project,” “expect,” “anticipate,” “estimate,” “intend,” “continue,” “strategy,” “future,” “opportunity,” “plan,” “may,” “should,” “will,” “shall,” “would” other words of similar meaning, derivations of such words and the use of future dates. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties. The following factors, among others, could cause actual results to differ materially from those described in these forward-looking statements: (i) the ability to implement business plans, forecasts, and other expectations in connection with the acquisition and integration of Alimera Sciences, Inc. (“Alimera”) and identify and realize additional opportunities and, in particular, the possibility that the Company is unable to achieve anticipated synergies, (ii) costs and regulatory requirements relating to contract manufacturing arrangements, (iii) costs or delays associated with manufacturing (including the sources and any changes in sources thereof) of the Company’s products, (iv) delays or failures in retaining and obtaining continuing and future product approvals from the FDA, and other regulatory issues relating to the Company's business and products, (v) market trends for the Company’s products, and the ability to achieve anticipated sales for such products, (vi) risks that the acquisition of Alimera may disrupt current plans and operations of the Company and potential difficulties of the Company in retaining employees of Alimera and/or maintaining business relationships of Alimera, (vii) the impact of any litigation to which the Company is, or may become, a party, including in connection with the acquisition and integration thereof, (viii) volatility in the Company’s stock price, including as a result of the acquisition, (ix) changes in competitive and regulated industries in which the Company operates, variations in operating performance across competitors, changes in laws and regulations affecting the Company’s business, and changes in the Company’s capital structure as a result of the acquisition, (x) regulatory and other approvals relating to product development and manufacturing, (xi) the Company’s ability, and that of its suppliers, development partners, and manufacturing partners, to comply with laws, regulations and standards that govern or affect the pharmaceutical and biotechnology industries and/or the Company and its products, (xii) costs incurred in connection with the acquisition of Alimera and the possibility that the Company is unable to realize anticipated benefits of the acquisition or to realize estimated pro forma results and underlying assumptions, (xiii) delays in production, increased costs and potential loss of revenues if there is a change in manufacturers or manufacturing processes due to the limited number of suppliers for the Company’s raw materials, active pharmaceutical ingredients, excipients and other materials, (xiv) the Company’s reliance on single source third-party contract manufacturing supply for certain of its key products, (xv) changes in policy or actions that may be taken by the FDA, United States Drug Enforcement Administration, and other regulatory agencies, including among other things, drug recalls, regulatory approvals, facility inspections and potential enforcement actions, (xvi) the impact of legislative or regulatory reform on the pricing for the Company’s products, (xvii) the Company’ ability to maintain the services of its key executives and other personnel, and (xviii) general business and economic conditions, such as inflationary pressures, geopolitical conditions including, but not limited to, the conflict between Russia and the Ukraine, the conflict between Israel and Gaza, conflicts related to the attacks on cargo ships in the Red Sea, and the effects and duration of outbreaks of public health emergencies. More detailed information on these and additional factors that could affect the Company’s actual results are described in the Company’s filings with the Securities and Exchange Commission (“SEC”), including its most recent annual report on Form 10-K and quarterly reports on Form 10-Q, as well as other filings with the SEC. All forward-looking statements in this news release speak only as of the date of this news release and are based on the Company’s current beliefs, assumptions, and expectations. The Company undertakes no obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise. Investor Relations:Lisa M. Wilson, In-Site Communications, Inc.T: 212-452-2793E: lwilson@insitecony.com Media Contact: Chris SaundersT: 484-794-6525E: chris.saunders@anipharmaceuticals.com Source: ANI Pharmaceuticals, Inc.

BOSTON and LONDON, Oct. 24, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM) and Axovia Therapeutics Ltd. today announced a joint research collaboration designed to advance the understanding of Bardet-Biedl syndrome (BBS). “We believe there is a significant need and opportunity with the growing awareness of BBS to better understand the disease and its epidemiology,” said Prof. Phil Beales, Axovia Therapeutics’ Chief Executive Officer and Co-Founder. “At Axovia, we are utilizing gene therapy to address the loss of sight and obesity experienced by patients with BBS, and we are pleased to be collaborating with Rhythm, a global company that has pioneered research in the melanocortin-4 receptor pathway and developed the first and only commercially available drug to help treat hyperphagia and severe obesity in patients living with BBS. Rhythm and Axovia Therapeutics are natural collaborators in such an effort.” “Prof. Beales is a leading expert on BBS and was involved in creating the original diagnostic criteria for patients living with this disease,” said David Meeker, M.D., Chairman, Chief Executive Officer and President of Rhythm. “We are excited to work together with Axovia Therapeutics to advance our shared understanding of the disease and pool the knowledge we have gained from our respective screening efforts with the goal of further improving the lives of patients and their families living with BBS.” About Bardet-Biedl syndrome (BBS)BBS is a rare autosomal recessive ciliopathy that presents with a variety of signs and symptoms that evolve over time including visual impairment, renal disease, polydactyly, genital abnormalities, cognitive impairment, hyperphagia and early-onset, severe obesity arising from impairment of the hypothalamic MC4R pathway. In the United States, BBS affects approximately 4,000 to 5,000 individuals with similar prevalence in Europe. About Axovia TherapeuticsAxovia Therapeutics is leading the development of therapies that address the genetic causes of blindness and obesity which are driven by ciliopathies. Ciliopathies are a group of more than 50 inherited genetic diseases linked to more than 950 genes that impact the function of cilia which are critical for protein transport and cellular signaling. The company is positioned to initiate clinical studies for its lead program for Bardet-Biedl Syndrome (BBS), AXV-101, in mid-2025 based on robust preclinical data, scaled manufacturing and established patient registries. The initial subretinal study is designed to halt photoreceptor cell death and retinal degeneration and the CNS delivery program, which will begin in 2026, will seek to address hyperphagia and obesity. AXV-101 has achieved U.S. Food and Drug Administration Orphan Drug Designation and Rare Pediatric Disease Designation. Axovia was formed following decades of work on ciliopathies at University College London by co-founders Professor Phil Beales and Dr. Victor Hernandez. About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients living with rare neuroendocrine diseases. Rhythm’s lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing, or patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS). Both the European Commission (EC) and the UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. The EC has also authorized setmelanotide for control of hunger and treatment of obesity in children as young as 2 years old, living with BBS or POMC, PCSK1, or LEPR deficiency. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists LB54640 and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm’s headquarters is in Boston, MA. Setmelanotide IndicationIn the United States, setmelanotide is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to POMC, PCSK1 or LEPR deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1 or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS) or BBS. In the European Union, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. In Europe, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology. Limitations of Use Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective: Obesity due to suspected POMC, PCSK1 or LEPR deficiency with POMC, PCSK1 or LEPR variants classified as benign or likely benign.Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, or BBS, including obesity associated with other genetic syndromes and general (polygenic) obesity. Contraindication Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. WARNINGS AND PRECAUTIONS Skin Pigmentation and Darkening of Pre-Existing Nevi: Generalized increased skin pigmentation and darkening of pre-existing nevi have occurred because of its pharmacologic effect. Full body skin examinations prior to initiation and periodically during treatment should be conducted to monitor pre-existing and new pigmentary lesions. Heart rate and blood pressure monitoring: In Europe, heart rate and blood pressure should be monitored as part of standard clinical practice at each medical visit (at least every 6 months) for patients treated with setmelanotide. Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Patients who have an erection lasting longer than 4 hours should seek emergency medical attention. Depression and Suicidal Ideation: Depression and suicidal ideation have occurred. Patients should be monitored for new onset or worsening depression or suicidal thoughts or behaviors. Consideration should be given to discontinuing setmelanotide if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur. Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue setmelanotide. Pediatric Population: The prescribing physician should periodically assess response to setmelanotide therapy. In growing children, the impact of weight loss on growth and maturation should be evaluated. In Europe, the prescribing physician should monitor growth (height and weight) using age- and sex-appropriate growth curves. Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: Setmelanotide is not approved for use in neonates or infants. Serious and fatal adverse reactions including “gasping syndrome” can occur in neonates and low birth weight infants treated with benzyl alcohol-preserved drugs. ADVERSE REACTIONS Most common adverse reactions (incidence ≥20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection. USE IN SPECIFIC POPULATIONS Lactation: Not recommended when breastfeeding. To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe. Please see the full U.S. Prescribing Information and EU Summary of Product Characteristics for additional Important Safety Information. Forward-looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress, potential regulatory submissions, approvals and timing thereof of setmelanotide and our other product candidates, and our business strategy and plans, the potential benefits of a joint research collaboration between Rhythm and Axovia Therapeutics, and the potential of Rhythm’s and Axovia Therapeutics’ drug candidates to provide treatment options for patients with BBS. Statements using words such as “expect”, “anticipate”, “believe”, “may”, “will” and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our ability to successfully commercialize setmelanotide, our liquidity and expenses, our ability to retain our key employees and consultants, and to attract, retain and motivate qualified personnel, and general economic conditions, and the other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the three months ended June 30, 2024 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise. Rhythm Contacts: Corporate:David ConnollyHead of Investor Relations and Corporate CommunicationsRhythm Pharmaceuticals, Inc.857-264-4280dconnolly@rhythmtx.com Media:Sheryl SeapyReal Chemistry(949) 903-4750sseapy@realchemistry.com Axovia Contact:Professor Phil BealesChief Executive Officerinvestors@axovia.com

Alpha-9 Oncology has raised $175m in a Series C funding round to advance its pipeline of radiopharmaceuticals. Credit: eamesBot via Shutterstock. Alpha-9 Oncology is the latest radiopharma to raise funds, drawing in $175m in Series C financing, to advance its radiopharmaceutical candidates through clinical trials and expand its research and development (R&D) capabilities. Vancouver, Canada-based Alpha-9 shared that it will advance its pipeline of preclinical candidates to the “clinic-ready” stage. While the company has not disclosed which of its assets in particular will be advanced, the company has several preclinical programs targeting MC1R, PSMA, GRPR, CXCR4, and B1R, as per GlobalData’s Pharma Intelligence Center. In May 2024, Alpha-9 announced the dosing of the first patient in its Phase I study for the MC1R programme, dubbed A9-3202. This peptide-based radiopharmaceutical targets the melanocortin 1 receptor for imaging locally advanced or metastatic melanoma. The trial is evaluating A9-3202’s tumour uptake and distribution in normal tissue among participants with melanoma. This latest financing comes after a flood of investment into the radiopharma arena over the last few years. Earlier this month, Aktis Oncology raised $175m to support the development of its new radiopharmaceuticals, including a first-in-class Nectin-4-targeted miniprotein radioconjugate. Big pharma has jumped on the radiopharma train too, with Bristol Myers Squibb (BMS), Novartis , and Eli Lilly signing acquisition deals worth $4.2bn , $1.75bn , and $1.4bn, respectively. Alpha-9’s Series C – co-led by Lightspeed Venture Partners and Ascenta Capital – builds on a $75m Series B in December 2022, and a $8.9m Series A in 2021, three years after the company spun-out from the University of British Columbia. Other participating investors in this round included a16z Bio + Health, RA Capital Management, Janus Henderson Investors and Samsara BioCapital. See Also: UPFs fuelling ‘alarming rise of chronic diseases’ in india FDA grants ODD to Modalis’ MDL-101 to treat LAMA2-CMD Alpha-9 said its funding will also support ongoing investments in its discovery capabilities, as well as the expansion of manufacturing and isotope supply partnerships. The company recently completed the construction of a manufacturing facility in Vancouver, Canada. Last month, Telix Pharmaceuticals paid $230m upfront to acquire US-based radioisotope manufacturer RLS Radiopharmacies to expand its manufacturing footprint in the US. Venture financing for innovative radiopharmaceutical drugs has witnessed an approximately 550% increase from $63m in 2017 to $408m in 2023 YTD (year-to-date) total deal value in the US, according to GlobalData’s Pharma Intelligence Center Deals Database. GlobalData is the parent company of Pharmaceutical Technology. In the announcement accompanying the financing, Alpha-9’s CEO David Hirsch said: “Over the last few years, Alpha-9 has built a leading radiopharmaceutical company with a deep pipeline and robust infrastructure.”