Hypogonadism

SALT LAKE CITY, March 7, 2024 /PRNewswire/ -- Lipocine Inc. (NASDAQ: LPCN), a biopharmaceutical company focused on treating Central Nervous System ("CNS") disorders, today announced financial results for the year ended December 31, 2023 and provided a corporate update. Neuroactive Steroids Lipocine announced positive topline results from a pharmacokinetics ("PK") pilot bridge study of LPCN 1154 (oral brexanolone). Lipocine is developing LPCN 1154 for postpartum depression ("PPD") The bridge study results demonstrated comparative pharmacokinetics of LPCN 1154 to an approved IV infusion brexanolone The study identified a dosing regimen of LPCN 1154 to be tested in a single confirmatory pivotal PK study required for NDA filing LPCN 1154 treatment was well-tolerated with no hypoxia or sedation-related adverse events The Company completed a successful meeting with the U.S. Food and Drug Administration ("FDA") during which it confirmed with the FDA its proposal for a 505(b)(2) NDA filing based on a single pivotal study comparing exposure of LPCN 1154 with the approved IV infusion of brexanolone. The use of exposure parameters and criteria to assess comparable exposure to IV infusion brexanolone was also agreed upon In January 2024, Lipocine completed a single arm PK study (N=8) utilizing the "to be marketed" LPCN 1154 formulation and target dosing regimen. PK exposure results of the scaled-up formulation were consistent with the PK performance of the formulation evaluated in the pilot study. The study results confirm the 48-hour dosing regimen for the planned pivotal PK study The company anticipates beginning the LPCN 1154 pivotal study in Q1 '24. Top line results from the study are expected by the end of Q2 '24. Assuming the study meets expectations, the company plans to file an NDA at the end of Q4 '24 Lipocine has identified a lead neuroactive steroid candidate for the management of essential tremor and is planning to submit a protocol for a proof-of-concept phase 2 study for Essential Tremor ("ET") in the first half of 2024 LPCN 1148 Lipocine announced positive topline results from its Phase 2 proof-of-concept ("POC") study evaluating LPCN 1148 for the management of decompensated cirrhosis The study met the primary endpoint: treatment with LPCN 1148 increased L3 skeletal muscle index (L3-SMI) relative to placebo (P <0.01) Fewer hepatic encephalopathy (HE) events of grade >1 were observed in the LPCN 1148 treatment arm relative to placebo (P < 0.05) More patients on LPCN 1148 reported symptom improvement compared to those on placebo (P < 0.05) LPCN 1148 was well-tolerated, with AE rates and severities similar to placebo Posters on LPCN 1148 and LPCN 1144 were presented at the European Association for the Study of the Liver (EASL) Congress 2023, in Vienna, Austria, June 21 – 24, 2023 Updated results from the Phase 2 POC study were presented at The American Association for the Study of Liver Diseases (AASLD) – The Liver Meeting® 2023, in Boston, MA, November 2023 After week 24, all patients in the study entered into a single arm open-label extension ("OLE") stage and received LPCN 1148. Patients converting from placebo to LPCN 1148 in this OLE showed improvement in sarcopenia End of study results from the POC study are expected by the end of Q2 '24 TLANDO™ In January 2024, Lipocine and Verity Pharma entered into an exclusive License Agreement under which Verity Pharma will market TLANDO in the United States and, if approved, in Canada Verity Pharma is responsible for regulatory and marketing obligations in the U.S. and Canada, and for all further development of TLANDO and TLANDO XR Verity Pharma agreed to pay Lipocine a license fee totaling $11 million. Upon execution of the agreement and upon transition of the commercialization of TLANDO to Verity, Lipocine received initial payments of $2.5 million and $5 million. Further payments of $2.5 million and $1 million are due from Verity Pharma no later than January 1, 2025, and January 1, 2026, respectively Lipocine is entitled to receive up to $259 million in development and sales-based commercial milestone payments, as well as tiered royalty payments ranging from 12% up to 18% on net sales of TLANDO franchise products Year Ended December 31, 2023 Financial Results Lipocine reported a net loss of $16.4 million, or ($3.14) per diluted share, for the year ended December 31, 2023, compared with a net loss of $10.8 million, or ($2.15) per diluted share, for the year ended December 31, 2022. During 2023 Lipocine recognized a non-cash minimum guaranteed royalties reversal of variable consideration revenue of $2.9 million related to the termination of the Antares License Agreement. The reversal of revenue is due to the fact that, as a result of the termination of the license agreement, the Company will not receive anticipated minimum royalties that were previously recorded for the Antares License Agreement. In 2022, the Company recorded revenue of $500,000 related to a non-refundable cash fee received from Antares. Research and development expenses were $10.2 million and $8.6 million, respectively, for the years ended December 31, 2023 and 2022. The increase in research and development expenses was primarily due to an increase in contract research organization expense related to the LPCN 1154 clinical studies, an increase in TLANDO manufacturing related costs, an increase related to the LPCN 1148 Phase 2 POC study in male patients with cirrhosis, and an increase in personnel expense. These increases were offset by a decrease related to expenses incurred in 2022 for LPCN 1111 scale up costs, LPCN 1107 clinical study costs, and other research and development activities. General and administrative expenses were $4.9 million and $4.1 million, respectively, for the years ended December 31, 2023 and 2022. The increase in general and administrative expenses was primarily due to an increase in business development and strategic advisory services expenses, an increase in legal fees, an increase in personnel related costs, an increase in franchise taxes, an increase in director fees, and an increase in other general and administrative expenses. These increases were offset by a decrease in corporate insurance expense, a decrease related to 2022 expenses for the recruitment of two additional directors, and a decrease in various consulting and professional fees. As of December 31, 2023, Lipocine had $22.0 million of unrestricted cash, cash equivalents and marketable investment securities compared to $32.5 million at December 31, 2022. Subsequent to year end, Lipocine received payments of $2.5 million and $5 million as part of the initial license fee from Verity Pharma in connection with the agreement entered into in January 2024 whereby Verity will take over commercialization of TLANDO, as described above. About Lipocine Lipocine is a biopharmaceutical company leveraging its proprietary technology platform to augment therapeutics through effective oral delivery to develop differentiated products for CNS disorders. Lipocine has drug candidates in development as well as drug candidates for which we are exploring partnering. Our drug candidates represent enablement of differentiated, patient friendly oral delivery options for favorable benefit to risk profile which target large addressable markets with significant unmet medical needs. Lipocine's clinical development candidates include: LPCN 1154, oral brexanolone, for the potential treatment of postpartum depression, LPCN 2101 for the potential treatment of epilepsy, LPCN 2203 for the potential treatment of essential tremor and LPCN 1148, a novel androgen receptor agonist prodrug for oral administration targeted for the management of symptoms associated with liver cirrhosis. Lipocine is exploring partnering opportunities for LPCN 1107, our candidate for prevention of preterm birth, LPCN1154, for rapid relief of postpartum depression, LPCN 1148, for the management of decompensated cirrhosis, and LPCN 1144, our candidate for treatment of non-cirrhotic NASH. TLANDO, a novel oral prodrug of testosterone containing testosterone undecanoate developed by Lipocine, is approved by the FDA for conditions associated with a deficiency of endogenous testosterone, also known as hypogonadism, in adult males. For more information, please visit . Forward-Looking Statements This release contains "forward-looking statements" that are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and include statements that are not historical facts regarding our product development efforts, the application of our proprietary platform in developing new treatments for CNS disorders, our lead neuroactive steroid candidate for the management of essential tremor, the timing of studies and clinical trials, our product candidates and related clinical trials, our development of and filing of a NDA with the FDA for LPCN 1154, the receipt of license fees, milestone payments and royalty payments under our license agreement with Verity Pharma, and the potential uses and benefits of our product candidates. Investors are cautioned that all such forward-looking statements involve risks and uncertainties, including, without limitation, the risks that we may not be successful in developing product candidates to treat CNS disorders, we may not have sufficient capital to complete the development processes for our product candidates, we may not be able to enter into partnerships or other strategic relationships to monetize our non-core assets, the FDA will not approve any of our products, risks related to our products, expected product benefits not being realized, clinical and regulatory expectations and plans not being realized, new regulatory developments and requirements, risks related to the FDA approval process including the receipt of regulatory approvals and our ability to utilize a streamlined approval pathway for LPCN 1154, the results and timing of clinical trials, patient acceptance of Lipocine's products, the manufacturing and commercialization of Lipocine's products, and other risks detailed in Lipocine's filings with the SEC, including, without limitation, its Form 10-K and other reports on Forms 8-K and 10-Q, all of which can be obtained on the SEC website at . Lipocine assumes no obligation to update or revise publicly any forward-looking statements contained in this release, except as required by law. SOURCE Lipocine Inc.

SALT LAKE CITY, March 6, 2024 /PRNewswire/ -- Lipocine Inc. (NASDAQ: LPCN), a biopharmaceutical company focused on treating Central Nervous System (CNS) disorders, today announced that Dr. Mahesh V. Patel, President and Chief Executive Officer, will participate in a fireside chat and meet with investors at the 36th Annual Roth Conference, to take place in Laguna Niguel, CA, on March 18. Presentation Details Investors interested in arranging a 1x1 meeting with Lipocine should contact their Roth representative. About Lipocine Lipocine is a biopharmaceutical company leveraging its proprietary technology platform to augment therapeutics through effective oral delivery to develop differentiated products for CNS disorders. Lipocine has drug candidates in development as well as drug candidates for which we are exploring partnering. Our drug candidates represent enablement of differentiated, patient friendly oral delivery options for favorable benefit to risk profile which target large addressable markets with significant unmet medical needs. Lipocine's clinical development candidates include: LPCN 1154, oral brexanolone, for the potential treatment of postpartum depression, LPCN 2101 for the potential treatment of epilepsy, LPCN 2203 for the potential treatment of essential tremor and LPCN 1148, a novel androgen receptor agonist prodrug for oral administration targeted for the management of symptoms associated with liver cirrhosis. Lipocine is exploring partnering opportunities for LPCN 1107, our candidate for prevention of preterm birth, LPCN1154, for rapid relief of postpartum depression, LPCN 1148, for the management of decompensated cirrhosis, and LPCN 1144, our candidate for treatment of non-cirrhotic NASH. TLANDO, a novel oral prodrug of testosterone containing testosterone undecanoate developed by Lipocine, is approved by the FDA for conditions associated with a deficiency of endogenous testosterone, also known as hypogonadism, in adult males. For more information, please visit . Forward-Looking Statements This release contains "forward-looking statements" that are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and include statements that are not historical facts regarding our product development efforts, the application of our proprietary platform in developing new treatments for CNS disorders, our product candidates and related clinical trials, our development of and filing of a NDA with the FDA for LPCN 1154, and the potential uses and benefits of our product candidates. Investors are cautioned that all such forward-looking statements involve risks and uncertainties, including, without limitation, the risks that we may not be successful in developing product candidates to treat CNS disorders, we may not have sufficient capital to complete the development processes for our product candidates, we may not be able to enter into partnerships or other strategic relationships to monetize our non-core assets, the FDA will not approve any of our products, risks related to our products, expected product benefits not being realized, clinical and regulatory expectations and plans not being realized, new regulatory developments and requirements, risks related to the FDA approval process including the receipt of regulatory approvals and our ability to utilize a streamlined approval pathway for LPCN 1154, the results and timing of clinical trials, patient acceptance of Lipocine's products, the manufacturing and commercialization of Lipocine's products, and other risks detailed in Lipocine's filings with the SEC, including, without limitation, its Form 10-K and other reports on Forms 8-K and 10-Q, all of which can be obtained on the SEC website at . Lipocine assumes no obligation to update or revise publicly any forward-looking statements contained in this release, except as required by law. SOURCE Lipocine Inc.

BOSTON, Feb. 07, 2024 (GLOBE NEWSWIRE) --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, today announced that the Italian Medicine Agency (AIFA) approved reimbursement (Gazzetta Ufficiale) for IMCIVREE® (setmelanotide) for the treatment of obesity and control of hunger associated with Bardet-Biedl syndrome (BBS). “We are pleased that AIFA has recognized the need for a precision medicine for obesity and control of hunger in BBS,” said Yann Mazabraud, Executive Vice President and Head of International at Rhythm. “Now families in Italy who are affected by this rare neuroendocrine disease will be able to access the first and only treatment option authorized in the European Union, and we look forward to working with the community in Italy to deliver IMCIVREE to patients as we aim to expand access in Europe country-by-country.” BBS is a rare melanocortin-4 receptor (MC4R) pathway disease with hallmark symptoms of hyperphagia, a pathological hunger that leads to abnormal food-seeking behaviors, and early-onset, severe obesity. BBS is also associated with cognitive impairment, polydactyly, renal dysfunction, hypogonadism, and visual impairment. In Italy, the Company estimates the prevalence of BBS to be between 1/125,000 and 1/160,000, with approximately 200 identified. AIFA previously approved reimbursement for IMCIVREE for the treatment of obesity and control of hunger associated with biallelic proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or biallelic leptin receptor (LEPR) deficiency. About Rhythm Pharmaceuticals Rhythm 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 RM-718 and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm’s headquarters is in Boston, MA. Setmelanotide Indication In 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 Bardet-Biedl syndrome (BBS) or genetically confirmed loss-of-function biallelic proopiomelanocortin (POMC), including PCSK1, deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 6 years of age and above. In Canada, setmelanotide is indicated for the treatment of obesity due to Bardet-Biedl syndrome (BBS) or genetically-confirmed biallelic pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency due to variants interpreted as pathogenic, likely pathogenic, or of uncertain significance in adults and children 6 years of age and above. Limitations of Use In the United States and Europe, Setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology. 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. WARNINGS AND PRECAUTIONS Skin Monitoring: Setmelanotide may lead to generalized increased skin pigmentation and darkening of pre-existing naevi because of its pharmacologic effect. Full body skin examinations should be conducted annually to monitor pre-existing and new skin pigmentary lesions before and during treatment with setmelanotide. Heart rate and blood pressure monitoring: 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. Prolonged penile erection: Spontaneous penile erections have been reported in clinical trials with setmelanotide. Patients who have a penile erection lasting longer than 4 hours should be instructed to seek emergency medical attention for potential treatment of priapism. Depression: In clinical trials, depression has been reported in patients treated with setmelanotide. Patients with depression should be monitored at each medical visit during treatment with setmelanotide. Consideration should be given to discontinuing setmelanotide if patients experience suicidal thoughts or behaviors. 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. The prescribing physician should monitor growth (height and weight) using age- and sex-appropriate growth curves. Excipients: This medicinal product contains 10 mg benzyl alcohol in each ml. Benzyl alcohol may cause allergic reactions. Patients who are pregnant or breastfeeding should be advised of the potential risk from the excipient benzyl alcohol, which might accumulate over time and cause metabolic acidosis. This medicinal product should be used with caution in patients with hepatic or renal impairment, because of the potential risk from the excipient benzyl alcohol which might accumulate over time and cause metabolic acidosis. Sodium: This medicinal product contains less than 1 mmol sodium (23 mg) per dose, that is to say essentially “sodium-free.” ADVERSE REACTIONS The most frequent adverse reactions are hyperpigmentation (51%), injection site reaction (39%), nausea (33%), and headache (26%). USE IN SPECIFIC POPULATIONS Pregnancy There are no data from the use of setmelanotide in pregnant women. Animal studies do not indicate direct harmful effects with respect to reproductive toxicity. However, administration of setmelanotide to pregnant rabbits resulted in decreased maternal food consumption leading to embryo-fetal effects. As a precautionary measure, setmelanotide should not be started during pregnancy or while attempting to get pregnant as weight loss during pregnancy may result in fetal harm. If a patient who is taking setmelanotide has reached a stable weight and becomes pregnant, consideration should be given to maintaining setmelanotide treatment as there was no proof of teratogenicity in the nonclinical data. If a patient who is taking setmelanotide and still losing weight gets pregnant, setmelanotide should either be discontinued, or the dose reduced while monitoring for the recommended weight gain during pregnancy. The treating physician should carefully monitor weight during pregnancy in a patient taking setmelanotide. Breast-feeding It is unknown whether setmelanotide is excreted in human milk. A nonclinical study showed that setmelanotide is excreted in the milk of nursing rats. No quantifiable setmelanotide concentrations were detected in plasma from nursing pups. A risk to the newborn/infant cannot be excluded. A decision must be made whether to discontinue breastfeeding or to discontinue/abstain from setmelanotide therapy taking into account the benefit of breastfeeding for the child and the benefit of therapy for the mother. Fertility No human data on the effect of setmelanotide on fertility are available. Animal studies did not indicate harmful effects with respect to fertility. To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337. See Summary of Product Characteristics’ APPENDIX V for a list of European national reporting systems to communicate adverse reactions. 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, potential regulatory submissions, approvals and timing thereof of setmelanotide, and our business strategy and plans, including regarding commercialization of IMCIVREE in Italy, the European Union, and elsewhere. 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, 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, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, 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 September 30, 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 Connolly Head of Investor Relations and Corporate Communications Rhythm Pharmaceuticals, Inc. 857-264-4280 dconnolly@rhythmtx.com Media Contact: Adam Daley Berry & Company Public Relations 212-253-8881 adaley@berrypr.com A PDF accompanying this announcement is available at