Hyperpigmentation

SKNV supports FDA's efforts to combat adverse effects associated with the use of OTC topical numbing creams for dermatologic procedures. POMPANO BEACH, Fla., April 4, 2024 /PRNewswire/ -- SKNV is increasing its manufacturing capacity to meet the growing demands of healthcare providers for topical numbing creams to support the FDA's recent efforts to combat unapproved and misbranded over-the-counter analgesic products. The Agency's warnings underscore the importance of prescribing Rx medications made from FDA-registered 503B outsourcing facilities, such as SKNV. SKNV's various prescription-based numbing formulations that offer different levels of potency are only made available to registered physicians at . SKNV, an FDA-Registered 503B Outsourcing Facility, plays a critical role in ensuring that patients have access to quality topical numbing medications. "SKNV manufactures a broad variety of topical numbing formulations providing prescribers a wide selection of solutions to choose from. These can readily be stocked in their office to meet a wider variety of patient needs," said SKNV Founder and Chief Executive Officer, Spencer Malkin. "We've built strong relationships with our physician customers and work diligently to offer them solutions that make a clear clinical difference for their patients, as determined by the prescriber," he added. Topical anesthetics have been valuable in providing non-invasive anesthesia for more than thirty years. Their role in reducing discomfort has been proven in vascular, pigmented, and epilation laser treatments, as well as for cosmetic injectables.1 "SKNV makes wonderful topical medications for skin rejuvenation and dermatologic procedures. I am especially grateful for their topical numbing cream. It works quickly and can be used to improve pain control for many types of dermatologic treatments from freezing small growths to doing full-face skin resurfacing," said Jennifer Reichel, MD. SKNV is committed to supporting the FDA's mission to stop the sale of illegal, high-risk numbing cream products. Healthcare professionals and patients should report any adverse reactions to medications to the Agency's MedWatch Program. ABOUT SKNV SKNV is an innovative pharmacy revolutionizing dermatology across the United States with its customized medication solutions. Focused on empowering providers and delivering customized topical medications for skin conditions like acne, melasma, and rosacea, SKNV redefines prescription care. Utilizing a state-of-the-art FDA-Registered 503B Outsourcing Facility ensures high-quality, tailored formulations without potentially harmful excipients such as allergens and unwanted inactives. SKNV eliminates traditional pharmacy hurdles such as prior authorizations, offering accessible and affordable care that enhances patient satisfaction. Through innovation and a commitment to personalized care, SKNV is setting new standards in dermatological health. For more information about SKNV and its comprehensive list of medications, please visit . To learn more about the FDA please visit: 1. Gaitan S, Markus R. Anesthesia methods in laser resurfacing. Semin Plast Surg. 2012;26:117–24. [PMC free article] [PubMed] [Google Scholar] SOURCE SKNV

-- Proceeds from financing and existing cash on-hand sufficient to fund planned operations into 2026 --BOSTON, April 01, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today announced that it has signed an investment agreement with current shareholders, led by Perceptive Advisors LLC and its Discovery Fund and a life-sciences focused institutional investor, for the sale of its series A convertible preferred stock (“Preferred Stock”) for gross proceeds of $150 million to the Company. The Company intends to use the proceeds from the offering to fund its clinical development programs and commercialization activities, for working capital, and for general corporate purposes. The transaction is expected to close on or about April 15, 2024, subject to the satisfaction of customary closing conditions. Based on its current operating plans, Rhythm expects the net proceeds from the sale of Preferred Stock, in addition to its cash, cash equivalents and short-term investments as of December 31, 2023, will be sufficient to fund its operating expenses and capital expenditure requirements into 2026. “We are pleased to announce this convertible preferred stock offering, led by Perceptive Advisors and its Discovery Fund, and we are pleased to receive continued support from a second, existing shareholder who chose to participate in this financing,” said David Meeker, M.D., Chairman, Chief Executive Officer and President of Rhythm. “This financing is expected to extend our cash runway well into 2026 and beyond multiple potentially value-creating milestones including the topline data readout from our phase 3 trial in hypothalamic obesity in the first half of 2025.” “Rhythm – with its lead asset IMCIVREE® (setmelanotide) approved and available in 14 countries, including the United States, to treat certain rare melanocortin-4 receptor diseases – has executed very well against its development, regulatory and commercial strategies on a global level,” said Konstantin Poukalov, Managing Director and Perceptive Discovery Co-Head. “We believe the Company has a clear and achievable global vision to address the unmet needs in additional rare MC4R pathway diseases, including hypothalamic obesity, with setmelanotide and its additional pipeline assets.” Following the expiration or termination of any applicable waiting period under the HSR Act, the Preferred Stock will be convertible into common stock at any time at an initial conversion rate of 20.8333 shares of common stock per $1,000 of liquidation preference, implying a conversion price of $48 per share, which is a 19% premium to the Company’s 10-day trailing volume weighted average price. The conversion rate is subject to customary adjustments, and adjustment in respect of certain dilutive issuances. The Company also can require conversion if the price of its common stock exceeds 250% of the implied conversion price for 20 trading days in a 30-trading day period, subject to certain requirements. The Company has the right to redeem all, but not less than all, of the Preferred Stock for the then applicable liquidation preference, which is initially par, on and after the five-year anniversary of its issuance. Holders of the Preferred Stock will be entitled to a 6% cumulative annual dividend, commencing on the second anniversary of closing. Dividends will be compounded quarterly, and payable in cash or in kind at the Company’s option. The Preferred Stock will vote with the common stock on an as-converted basis, subject to satisfaction of certain antitrust-related conditions. Holders of the Preferred Stock are entitled to 175% of the liquidation preference upon certain corporate events, including a change of control or liquidation of the Company. The Company has agreed to grant the investors certain registration rights with respect to the common stock underlying the Preferred Stock. J. Wood Capital Advisors acted as financial advisor and Latham & Watkins LLP acted as legal counsel to the Company on the transaction. Ropes & Gray LLP acted as legal counsel to Perceptive Advisors. This press release shall not constitute an offer to sell or a solicitation of an offer to buy the Preferred Stock, nor shall there be any sale of the Preferred Stock in any state or jurisdiction in which such offer, solicitation or sale would be unlawful under the securities laws of any such state or jurisdiction. About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families 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. 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 UseSetmelanotide 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 benignOther types of obesity not related to POMC, PCSK1 or LEPR deficiency, or BBS, including obesity associated with other genetic syndromes and general (polygenic) obesity. ContraindicationPrior 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 Prescribing Information 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 of, and our business strategy and plans for, our drug products, the anticipated benefits of and activities under the investment transaction with Perceptive, our use of proceeds from the transaction with Perceptive, the expected timing for closing of the transaction, our expectation that the transaction with Perceptive will provide us capital to execute our corporate strategy in 2024 and 2025; and the issuance of dividends, and the sufficiency of our cash, cash equivalents and short-term investments to fund our operations. Statements using word 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, whether the conditions for the closing of the investment transaction; 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 Annual Report on Form 10-K for the year ended December 31, 2023 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. Corporate Contact:David ConnollyHead of Investor Relations and Corporate CommunicationsRhythm Pharmaceuticals, Inc.857-264-4280dconnolly@rhythmtx.com Media Contact:Adam DaleyBerry & Company Public Relations212-253-8881adaley@berrypr.com

Pictured: 3D illustration of arrows reflecting changes in direction/iStock/Olivier Le Moal Despite having an FDA-approved product, Gamida Cell is entering into a restructuring agreement in which it will become a private company under the ownership of Highbridge Capital Management amid financial struggles, the company announced Wednesday. Under the agreement, Gamida Cell will become a private company wholly owned by Highbridge Capital Management—the biotech’s principal lender. The move is designed to give the company some long-term runway and support the commercialization of Omisirge, an FDA-approved nicotinamide modified allogeneic hematopoietic progenitor cell therapy. This comes after the biotech announced in January that it was seeking “strategic alternatives” and was focused on an asset sale, merger, or other deal. “In March 2023, Gamida Cell embarked on an extensive strategic process to address its capital structure and liquidity constraints by partnering Omisirge with a third party,” Gamida Cell CEO Abbey Jenkins said in Wednesday’s statement. “Unfortunately, that process did not yield any actionable alternatives. This restructuring will enable Gamida Cell to remain a going concern. It will support our ongoing efforts to make Omisirge available to more transplant centers and their patients as a potentially lifesaving donor source option.” The terms of the restructuring will see Highbridge convert $75 million of its existing unsecured convertible senior note into equity for Gamida Cell. The biotech will also get $30 million of new capital from Highbridge when the restructuring goes into effect. The biotech will now be off the stock market and wholly owned by Highbridge, with all ordinary shares expected to be canceled. The reorganized Gamida Cell will also issue contingent value rights with a maximum value of $27.5 million to shareholders. Gamida Cell’s main product, Omisirge, was approved by the FDA in April 2023 after a lengthy regulatory road and is indicated for patients who are scheduled to undergo umbilical cord blood transplantation after chemotherapy or radiotherapy. However, the therapy does come with a boxed warning for potentially fatal conditions such as infusion reactions, graft failure, engraftment syndrome, and graft-versus-host disease. The company revealed in a third-quarter report in 2023 that 17 transplant centers were onboarded to provide Omisirge to patients, exceeding the company’s target of 10 to 15. However, the company said that it would continue to have a “lean launch effort” in 2024 due to “resource constraints” and—contingent on additional funding to extend its cash runway—would plan to onboard at least 40 transplant centers by the end of this year. “Despite Gamida Cell’s financial struggles, we believe in the potential of Omisirge to fulfill an important unmet need in stem cell transplant,” Jonathan Segal, co-chief investment officer at Highbridge Capital Management, said in a statement. “Subject to an approved budget from the Company’s new board of directors, we intend to provide the Company with additional capital to fund this potentially life-saving therapy.” The deal is expected to close in the second quarter of 2024 upon being approved by an Israeli court. Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.