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• Mitapivat demonstrated statistically significant improvement in hemoglobin response compared with placebo, with rapid onset and durable effects
• New analyses showed patients in mitapivat arm had clinically meaningful reduction in transfusion burden compared with placebo
• Patients in mitapivat arm who achieved hemoglobin response had clinically meaningful benefits across measures of sickle cell pain crises, fatigue, and other patient-reported outcomes
• Mitapivat was well-tolerated, with a safety profile consistent with previous trials of mitapivat in sickle cell disease
• Company to host investor conference call and webcast today at 9:00 a.m. ET (3:00 p.m. CEST)

CAMBRIDGE, Mass., June 13, 2026 (GLOBE NEWSWIRE) — Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today presented detailed results from the 52-week double-blind period of the global RISE UP Phase 3 trial of mitapivat, an oral pyruvate kinase (PK) activator, in patients aged 16 years or older with sickle cell disease. These efficacy and safety results, which include new transfusion burden and hemoglobin responder analyses reinforcing the strong anti-hemolytic profile of mitapivat, were presented during the distinguished Plenary Abstracts Session at the 31st European Hematology Association (EHA) Congress (EHA 2026) in Stockholm, Sweden.

In November 2025, topline results from RISE UP demonstrated a significant improvement in the trial’s primary endpoint of hemoglobin response with mitapivat compared with placebo. The trial also met two key secondary endpoints, showing rapid and durable improvements in hemoglobin concentration and indirect bilirubin, a marker of hemolysis (red blood cell destruction). Although mitapivat showed a reduction in the annualized rate of sickle cell pain crises (SCPCs) compared with placebo, this primary endpoint did not reach statistical significance, and there was no overall difference between mitapivat and placebo for the key secondary endpoint measuring patient-reported fatigue. However, patients in the mitapivat arm who achieved a hemoglobin response experienced clinically meaningful reductions in the annualized rate of SCPCs and related hospitalizations, as well as improvements in fatigue.

New RISE UP analyses, not previously disclosed by the company, further highlight the potential for mitapivat to offer clinical benefits for patients with sickle cell disease, as evidenced by a clinically meaningful reduction in transfusion burden and, for hemoglobin responders in the mitapivat arm, improvements observed across additional measures of pain and physical function.

“Patients living with sickle cell disease are in critical need of new treatments that can effectively manage the debilitating impact of their condition,” said Biree Andemariam, M.D., Professor of Medicine and American Red Cross Endowed Chair in Transfusion Medicine, University of Connecticut Health, and a RISE UP trial investigator. “The RISE UP Phase 3 data presented today showcase the strong anti-hemolytic profile of mitapivat, with rapid and durable improvements in both hemoglobin and indirect bilirubin as well as a meaningful reduction in transfusion burden. Importantly, this anti-hemolytic effect is translating to clear clinical benefits, including improvements for hemoglobin responders across measures of sickle cell pain crises, pain, sleep, and physical function compared with non-responders. Together, these data reinforce the potential for mitapivat to improve the relentless physical toll that comes with living with sickle cell disease.”

New RISE UP Phase 3 Trial Results at EHA 2026

Reduction in Transfusion Burden

New analyses from RISE UP show that mitapivat was associated with a clinically meaningful reduction in transfusion burden compared with placebo. Patients in the mitapivat arm had a 41.1% relative reduction in the proportion of patients requiring blood transfusions compared with placebo (23.9% with mitapivat vs. 40.6% with placebo), as well as a 55.9% relative reduction in average red blood cell units transfused per patient compared with placebo (0.70 units with mitapivat vs. 1.59 with placebo). These benefits were observed regardless of whether patients were also taking hydroxyurea. A reduction in transfusion burden in sickle cell disease can reflect decreased dependence on supportive care.

Hemoglobin Responders Post-Hoc Analysis

As previously reported, 40.6% of patients in the mitapivat arm achieved the primary endpoint of hemoglobin response (≥1.0 g/dL increase from baseline in average hemoglobin from Week 24 through Week 52) compared with 2.9% in the placebo arm, a statistically significant improvement (2-sided p<0.0001). Among these hemoglobin responders, the mean change from baseline in average hemoglobin concentration from Week 24 through Week 52 was 1.6 g/dL.

A post-hoc analysis showed patients in the mitapivat arm who achieved a hemoglobin response also experienced clinically meaningful reductions in pain crises and related hospitalizations, including a 26% reduction in the annualized rate of SCPCs (2.20 for responders vs. 2.98 for non-responders) and 34% fewer related hospitalizations (1.16 for responders vs. 1.76 for non-responders). These patients also had improvements in healthcare utilization, with a 53% reduction in the annualized rate of emergency room visits for SCPCs (1.11 for responders vs. 2.33 for non-responders) and a 37% decrease in the annualized rate of hospitalization days for SCPCs (7.83 for responders vs. 12.34 for non-responders).

Hemoglobin responders in the mitapivat arm also reported greater improvements in patient-reported fatigue scores than non-responders (-5.19 for responders vs. -2.55 for non-responders), as measured by change from baseline in average Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue 13a Short Form scores from Week 24 through Week 52. The magnitude of this improvement in hemoglobin responders exceeded the predefined 4.1-point threshold required to be considered clinically meaningful.

In the mitapivat arm, improvements across several additional patient-reported outcomes, including measures of pain, sleep, and physical function, were observed for hemoglobin responders compared with non-responders:

• PROMIS Pain Intensity 1a: The mean change from baseline was -1.63 points for hemoglobin responders and -0.59 for non-responders, with a least squares mean (LSM) difference of -1.04 (95% confidence interval [CI]: -1.66 to -0.42), favoring hemoglobin responders. The LSM difference is used throughout to represent the model-adjusted difference between hemoglobin responders and non-responders.
• Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) Pain Impact: The mean change from baseline was 4.09 points for hemoglobin responders and 0.85 for non-responders, with an LSM difference of 3.24 (95% CI: 1.18 to 5.30), favoring hemoglobin responders.
• PROMIS Physical Functioning 8a: The mean change from baseline was 5.30 points for hemoglobin responders and 1.79 for non-responders, with an LSM difference of 3.51 (95% CI: 0.62 to 6.39), favoring hemoglobin responders.
• ASCQ-Me Sleep Impact: The mean change from baseline was 2.39 points for hemoglobin responders and -0.48 for non-responders, with an LSM difference of 2.87 (95% CI: 0.22 to 5.53), favoring hemoglobin responders.
• EuroQol-5 Dimension Visual Analog Scale (EQ-5D VAS): The mean change from baseline was 3.27 points for hemoglobin responders and -6.77 for non-responders, with an LSM difference of 10.04 (95% CI: 2.41 to 17.66), favoring hemoglobin responders.

“Having the opportunity to present these comprehensive results during the EHA 2026 Plenary Session highlights the strength of the RISE UP Phase 3 data – the first pivotal trial to validate pyruvate kinase activation as a new treatment approach in sickle cell disease,” said Sarah Gheuens, M.D., Ph.D., Chief Medical Officer and Head of R&D, Agios. “Building on over a decade of clinical experience with mitapivat across several hemolytic anemias, these results reinforce both its consistent benefits and its well-established safety profile, which is supported by over 1,300 patient-years of data. Taken together, mitapivat represents a differentiated anti-hemolytic approach that can provide meaningful clinical benefits for patients with sickle cell disease – an underserved population in desperate need of innovative therapies.”

Safety Profile

Mitapivat was well-tolerated, with a safety profile consistent with previous trials of mitapivat in sickle cell disease. The percentage of patients with any reported treatment-emergent adverse events was similar between the mitapivat and placebo arms (97.1% vs. 98.6%, respectively). No treatment-related deaths occurred during the trial.

EHA 2026 Investor Event

Agios will host a conference call and live webcast during EHA 2026 today, June 13, 2026, at 9:00 a.m. ET (3:00 p.m. CEST). The live webcast will be accessible on the Investors section of the company’s website (www.agios.com) under the “Events & Presentations” tab. A replay of the webcast will be available on the company’s website approximately two hours after the event.

About Sickle Cell Disease

Sickle cell disease is a rare, inherited blood disorder caused by the production of abnormal hemoglobin that disrupts the ability of red blood cells to carry oxygen throughout the body. As a result, red blood cells become rigid and sickle-shaped, causing deformation of red blood cell membranes and the premature death of the cells. These effects lead to chronic hemolytic anemia, vaso-occlusion, and a cascade of severe and life-threatening complications, including long-term damage to the lungs, kidneys, and cardiovascular system. Due to its physical toll, sickle cell disease imposes a profound burden on patients and their families, marked by increased healthcare needs and early mortality.

About Mitapivat in Sickle Cell Disease

Mitapivat, an oral pyruvate kinase (PK) activator, is designed to enhance the process by which red blood cells produce energy. This approach has the potential to improve red blood cell health by increasing ATP levels to support increased energy demands and lowering levels of a molecule called 2,3-diphosphoglycerate (2,3-DPG). In sickle cell disease, increased stress on red blood cells results in elevated levels of 2,3-DPG, which raises the likelihood that red blood cells develop the abnormal “sickle” shape that triggers vaso-occlusive crises. In May 2026, Agios announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the accelerated approval of mitapivat in sickle cell disease.

About the RISE UP Phase 3 Trial

The global RISE UP Phase 3 trial (NCT05031780) is evaluating the efficacy and safety of mitapivat in patients with sickle cell disease aged 16 years or older, representative of the global population. The trial included a 52-week, double-blind, randomized, placebo-controlled period, in which 207 participants were randomized 2:1 to receive oral mitapivat (100 mg) twice daily (n=138) or matched-placebo (n=69).

To comprehensively evaluate objective measures of hemolysis alongside other clinically relevant outcomes in sickle cell disease, the double-blind period of RISE UP included two primary endpoints – hemoglobin response and annualized rate of sickle cell pain crises – as well as five key secondary endpoints:

• Average change from baseline in hemoglobin concentration from Week 24 through Week 52
• Average change from baseline in indirect bilirubin from Week 24 through Week 52
• Average change from baseline in Patient Reported Outcome Measurement Information System Fatigue 13a (PROMIS Fatigue) Short Form scores from Week 24 through Week 52
• Annualized frequency of hospitalizations for sickle cell pain crises
• Average change from baseline in percent reticulocyte levels from Week 24 through Week 52

Of the 176 participants who completed the double-blind period of the trial, nearly all (n=174/176) opted to transition into a 216-week open-label extension (OLE) period, during which all participants receive mitapivat.

About Agios: Fueled by Connections to Transform Rare Diseases™

At Agios, our vision is to redefine the future of rare disease treatment. Fueled by connections, we build trusted partnerships with communities – collaborating to develop and deliver innovative medicines that have the potential to transform lives. With a foundation in hematology, we combine biological expertise with real-world insights to advance a growing pipeline of rare disease medicines that reflect the priorities of the people we serve. Agios is a commercial-stage biopharmaceutical company headquartered in Cambridge, Massachusetts. To learn more, visit www.agios.com and follow us on LinkedIn and X.

Available Information about Agios

To achieve broad dissemination, Agios may disclose information to the public through a variety of disclosure channels including press releases, SEC filings, and public conference calls and webcasts. Some of the information distributed through these disclosure channels may be considered material information. Investors and others should note that Agios plans to use its website (www.agios.com) as a distribution channel to announce and give notice of Agios’ upcoming events and presentations (including, but not limited to, presentations at medical or healthcare conferences). Such information, which may be deemed material, will be available on the Investors section of the company’s website under the “Events & Presentations” tab. In addition, you may sign up to automatically receive email alerts about Agios’ upcoming events and presentations (“Calendar Alerts”) by visiting the “Email Alerts” option under the “IR Resources” tab of the Investors section of the company’s website and submitting your email address.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of mitapivat and the potential benefits of Agios’ strategic plans and focus. The words “anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” “vision,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios’ current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios’ product candidates will successfully continue. There can be no guarantee that any positive developments in Agios’ business will result in stock price appreciation. Management’s expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation: risks and uncertainties related to the impact of pandemics or other public health emergencies to Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Agios’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios’ ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios’ ability to establish and maintain key collaborations; uncertainty regarding any royalty payments related to the sale of its oncology business or any milestone or royalty payments related to its in-licensing of AG-236 or cevidoplenib, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of Agios’ cash and cash equivalents; and general economic and market conditions. These and other risks are described in greater detail under the caption “Risk Factors” included in Agios’ public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

Contacts:
Investor Contact
Morgan Sanford, Vice President, Investor Relations
Agios Pharmaceuticals
[email protected]

Media Contact

Eamonn Nolan, Senior Director, Corporate Communications
Agios Pharmaceuticals
[email protected]

 — Patients with PWS treated with setmelanotide therapy (N=17) achieved clinically meaningful BMI or BMI z-score reductions, reductions in fat mass with preservation of lean mass, and improvements in hyperphagia and anxiety measures —   

— Positive results reinforce rationale for Phase 3 development of MC4R agonism in PWS —

— Company to hold conference call on Saturday, June 13, at 8 a.m. CT, 9 a.m. ET —

BOSTON, June 13, 2026 (GLOBE NEWSWIRE) — Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced preliminary data from a Phase 2 trial evaluating setmelanotide in patients with Prader-Willi syndrome (PWS) delivered during the Endocrine Society’s Annual Meeting (ENDO 2026) in Chicago.

“Patients and families living with PWS face severe hyperphagia and obesity due to underlying MC4R pathway dysfunction and have limited effective treatment options. These results show that MC4R agonism has the potential to deliver sustained and durable improvements in outcomes across BMI, hyperphagia scores, body composition, and food-related behaviors and anxiety,” said Jennifer Miller, M.D., University of Florida Division of Endocrinology, Department of Pediatrics in the College of Medicine, the principal investigator for this Phase 2 trial. “Importantly, such reductions in HQ-CT score and anxiety, as well as weight reduction, have the potential to ease the burden not only on patients, but also on their caregivers who manage the daily challenges of this disease.”

Rhythm enrolled 18 patients with PWS aged 6-23 years old with a BMI ≥30 kg/m² for patients ≥18 years of age or BMI ≥95th percentile for age and sex for patients younger than 18 in this Phase 2 trial. The 52-week trial remains ongoing, and 17 patients remain on active therapy as of June 12, 2026.

Results from the six-month analysis demonstrate that treatment with setmelanotide was associated with improvements across multiple clinically relevant endpoints, as of a data cut off date of May 7, 2026. Highlights include:

• Consistent BMI reductions in pediatric and adult patients at Month 6:
  • -3.06% mean reduction in BMI (N=17 pts);
  • -3.11% mean reduction in BMI in adult patients (n=10); with six achieving >2.5% BMI reduction, and four achieving >4% BMI reduction;
  • −3.00% mean reduction in BMI in pediatric patients (n=7);
  • −0.35 mean reduction in BMI z-score from baseline in pediatric patients (n=7);
  • Five (5) of seven pediatric patients achieved clinically meaningful BMI z-score reduction >0.2;
• Setmelanotide achieved preservation of lean mass and reductions in fat mass across 16 patients with data available from DEXA scans:
  • +0.74% mean gain in lean mass and -4.19% mean loss in fat mass across 16 patients;
  • Six (6) of nine adult patients achieved >5% reduction in fat mass;
  • Five (5) of seven pediatric patients gained ≥2.95% in lean mass;
• Clinically meaningful improvement in hyperphagia score observed in patients with moderate to severe hyperphagia, defined as a ≥7-point reduction in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score
  • Eight (8) of 10 patients who entered trial with moderate to severe hyperphagia (>13 at baseline) achieved clinically meaningful improvement of 7 points or better.
• Improvement in PWS Anxiousness and Distress Behaviors Questionnaire (PADQ) which measures anxiousness, emotional distress, and behavioral dysregulation.
  • Of the 15 patients who had a baseline score >11, 10 patients achieved clinically meaningful improvement of ≥11 points; and
• Safety and tolerability results have been consistent with the well-established profile observed with setmelanotide.

“These results demonstrate the potential for MC4R agonists to address the underlying biology of this severe disease and increased our confidence to advance into Phase 3 trials for PWS,” said David Meeker, M.D., Chairman, Chief Executive Officer and President of Rhythm.

About Prader-Willi Syndrome

PWS is a rare genetic disorder that results in a number of physical, mental and behavioral problems. A key feature of PWS is a constant sense of hunger that usually begins at about 2 years of age. PWS is estimated to affect approximately 400,000 people worldwide. Rhythm estimates there are between 12,500 and 16,000 patients living with PWS in the United States and a similar number in Europe, based on updated internal prevalence estimates developed using a bottoms-up methodology analyzing incidence, age-specific survival and claims-based validation. Further, the Company estimates that 80% to 90% of PWS patients are living with hyperphagia and obesity, or approximately 8,500 – 12,750 patients. There are currently limited therapeutic options that effectively reduce the extreme hyperphagia and address low resting energy expenditure associated with PWS.

Conference Call Information

Rhythm Pharmaceuticals will host a live conference call and webcast at 8 a.m. CT/ 9 a.m. ET on Saturday, June 13, to discuss these data. Participants may register for the conference call here. It is recommended that participants join the call ten minutes prior to the scheduled start.

A webcast of the call will also be available under “Events and Presentations” in the Investor Relations section of the Rhythm Pharmaceuticals website at https://ir.rhythmtx.com/. The archived webcast will be available on Rhythm Pharmaceuticals’ website approximately two hours after the conference call and will be available for 30 days following the call.

Multiple Rhythm presentations from ENDO 2026 will be available in the afternoon on Monday, June 15, at: https://hcp.rhythmtx.com/publications-presentations/

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) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 4 years and older with acquired hypothalamic obesity, adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. The European Commission (EC) has authorized setmelanotide for the treatment of obesity and control of hunger in patients 4 years of age and above with acquired hypothalamic obesity; and both the 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 2 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 bivamelagon and 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 to reduce excess body weight and maintain weight reduction long term in adults and pediatric patients aged 4 years and older with acquired hypothalamic obesity, in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

In the European Union and the United Kingdom, 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 2 years of age and above. In the European Union and the United Kingdom, 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 acquired HO, BBS, or POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity.

Important Safety Information

CONTRAINDICATIONS

Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported.

WARNINGS AND PRECAUTIONS

Disturbance in Sexual Arousal: Spontaneous penile erections and increased frequency of penile erections in males have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.

Depression and Suicidal Ideation: Depression and suicidal ideation have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE 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 IMCIVREE.

Skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Development of New Melanocytic Nevi: Generalized or focal increases in skin pigmentation occurred in the majority of IMCIVREE-treated patients. IMCIVREE may also cause development of new melanocytic nevi or darkening of pre-existing nevi. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions.

Acute Adrenal Insufficiency with Acquired HO: Patients with acquired HO and secondary adrenal insufficiency reported serious adverse reactions related to acute adrenal insufficiency in 5% of IMCIVREE-treated patients and no placebo-treated patients. In patients with secondary adrenal insufficiency, monitor for clinical signs of acute adrenal insufficiency.

Sodium Imbalance in Patients with Acquired HO and Central Diabetes Insipidus: Patients with acquired HO and concomitant central diabetes insipidus (DI)/arginine vasopressin (AVP) deficiency reported hyponatremia in 6% of IMCIVREE-treated patients and 2% of placebo-treated patients and hypernatremia in 5% of IMCIVREE-treated patients and 4% of placebo-treated patients. Monitor serum sodium levels with changes in fluid intake and hydration status. Adjust the doses of concomitant therapies for DI/AVP deficiency as needed.

ADVERSE REACTIONS

Most common adverse reactions (incidence ≥20% in at least 1 indication) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection.

USE IN SPECIFIC POPULATIONS

Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus.

To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or http://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 safety, efficacy, potential benefits of, and clinical design or progress, potential regulatory submissions, approvals and timing thereof for any of our products or product candidates at any dosage or in any indication; the presentation of clinical data and results from our trials, including the ongoing Phase 2 trial of setmelanotide in patients with PWS, clinical and real-world efficacy and safety data related to the use of setmelanotide and bivamelagon in patients with acquired hypothalamic obesity BBS and our participation in upcoming events and presentations, including at ENDO and the content, date and timing of any of the foregoing. 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, unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives, risks associated with the laws and regulations governing our international operations and the costs of any related compliance programs, 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 other important factors, including those discussed under the caption “Risk Factors” in Rhythm’s Quarterly Report on Form 10-Q for the three months ended March 31, 2026, 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 Contacts:

David Connolly
Head of Investor Relations and Corporate Communications
Rhythm Pharmaceuticals, Inc.
857-264-4280
[email protected]  

Kate Walsh
Director, Corporate Communications
Rhythm Pharmaceuticals, Inc.
(857) 264-4280
[email protected]

• Data from HAELO Phase 3 clinical trial presented today in a late-breaking oral session at European Academy of Allergy & Clinical Immunology Annual Congress 2026

• HAELO manuscript simultaneously published in the New England Journal of Medicine

CAMBRIDGE, Mass., June 13, 2026 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (Nasdaq: NTLA), a leading biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies, today presented additional positive results from the global Phase 3 HAELO clinical trial of lonvo-z (formerly NTLA-2002) for hereditary angioedema (HAE) in a late-breaking oral presentation at the European Academy of Allergy & Clinical Immunology (EAACI) Annual Congress 2026 in Istanbul, Türkiye. Results from the trial were simultaneously published in the New England Journal of Medicine. The presentation and publication can be accessed from the Scientific Publications and Presentations section of intelliatx.com.

As previously announced, HAELO met its primary endpoint with an 87% reduction (p<0.0001) in mean monthly attacks in the lonvo-z arm vs. the placebo arm during the efficacy evaluation period (weeks 5 to 28). In addition, 62% of patients in the lonvo-z arm were entirely attack free and therapy free for the six-month efficacy evaluation period, compared with 11% of patients in the placebo arm (p<0.0001), a key secondary endpoint. Today, Intellia reported data for the trial’s other key secondary endpoints:

<sup>AE-QoL: Angioedema Quality of Life score, which is a validated, angioedema-specific patient-reported outcome measure with a lower score indicating improved quality of life. A 6-point reduction is considered to be a clinically important improvement in AE-QoL.

CI: Confidence interval</sup>

Favorable safety and tolerability data were observed for lonvo-z. The most common treatment emergent adverse events (TEAEs) during the primary observation period (infusion through week 28) that were higher in the lonvo-z group compared to placebo were infusion-related reaction, headache, fatigue, back pain, and upper respiratory tract infection. All reported TEAEs were mild or moderate and there were no serious adverse events observed in the lonvo-z arm.

“These are the first Phase 3 results to deliver on the much-heralded promise of in vivo CRISPR gene editing,” said John Leonard, M.D., Intellia President and Chief Executive Officer. “Regardless of age or prior use of long-term prophylaxis therapies, it was observed that a single lonvo-z treatment significantly reduced HAE attacks for all patients during the efficacy evaluation period, with all patients remaining LTP free as of the data cutoff. We thank the many patients, physicians and caregivers who participated in HAELO and are excited to be advancing this highly differentiated candidate toward a potential approval.”

Danny Cohn, M.D., Ph.D., Internist, Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, and a HAELO principal investigator, added, “As a clinician who has witnessed patients struggle with the unpredictability and emotional toll of HAE, the prospect of offering lasting freedom from attacks and chronic medication with a one-time treatment is incredibly exciting. These results give me confidence that many patients will soon have the potential to enjoy a normal life.”

Today’s presentation and publication also included supplemental demographics, data and analyses, including: 

• A time plot showing that the mean monthly attack rate for patients receiving lonvo-z through the data cutoff (February 10, 2026) was well below the reported rate in prescreening while patients were receiving standard-of-care therapy;
• Patient-level data demonstrating that all patients in the lonvo-z arm experienced attack-rate reductions from baseline during weeks 5 to 28;
• An analysis showing that meaningful attack-rate reductions were observed for all evaluated subgroups;
• A breakdown showing that 20% of the patients who enrolled in HAELO reported having complete disease control (no attacks) as their best response to prior long-term prophylaxis therapies; and
• A plasma kallikrein time plot showing that protein levels decreased substantially by the first measurement (day 15), reached a steady state by week 5 and remained stable through the data cutoff.

A rolling biologics license application (BLA) submission for lonvo-z was initiated in April with the U.S. Food and Drug Administration (FDA). The company continues to anticipate regulatory approval and a U.S. launch in the first half of 2027.

About Lonvo-z

Based on Nobel Prize-winning CRISPR/Cas9 technology, lonvo-z has the potential to become the first one-time treatment for hereditary angioedema (HAE). Lonvo-z is an in vivo CRISPR gene editing candidate that is intended to permanently lower kallikrein by inactivating the kallikrein B1 (KLKB1) gene with a single dose. Lonvo-z has received five notable regulatory designations: Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration (FDA), the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation (ODD) by the European Commission.

About Hereditary Angioedema

Hereditary angioedema (HAE) is a rare, genetic disease characterized by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body, which can be painful, debilitating and life-threatening. It is estimated that one in 50,000 people are affected by HAE. There are preventative and on-demand treatment options to help manage the condition, including long- and short-term prophylaxis used to prevent swelling attacks. Current treatment options often include lifelong therapies, which may require chronic intravenous (IV) or subcutaneous (SC) administration as often as twice per week or daily oral administration to ensure constant pathway suppression for disease control. Despite chronic administration, breakthrough attacks still occur. Kallikrein inhibition is a clinically validated strategy for the preventive treatment of HAE attacks.

About Intellia Therapeutics

Intellia Therapeutics, Inc. (Nasdaq: NTLA) is a leading clinical-stage biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies. The company’s mission is to transform the lives of people with severe diseases by developing and commercializing potentially curative treatments. With deep scientific, technical and clinical development experience, Intellia aims to reset the standard for medicine by durably treating the root causes of disease. Learn more at intelliatx.com and follow us @intelliatx.

Forward-Looking Statements
This press release contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia” or the “Company”) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s beliefs and expectations concerning: the success and advancement of its program for lonvoguran ziclumeran or “lonvo-z” (formerly NTLA-2002) for the treatment of hereditary angioedema (“HAE”), including its plan to complete the submission of a biologics license application (“BLA”) for lonvo-z, its expectations regarding review and approval of that BLA, and its expectations regarding a potential U.S. launch of lonvo-z in the first half of 2027; and the potential of one dose of lonvo-z to become the first one-time treatment for HAE and to permanently lower kallikrein by inactivating the kallikrein B1 (KLKB1) gene with a single dose.

Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: uncertainties related to the conduct of clinical studies and other development and commercialization requirements for its product candidates, including lonvo-z, including risks related to the ability to develop and successfully commercialize lonvo-z or any of Intellia’s product candidates; risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to Intellia’s relationship with third parties, including its contract manufacturers, collaborators, licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; risks related to the results of preclinical studies or clinical studies not being predictive of future results in connection with future studies; the risk that clinical study results will not be positive; and risks related to the potential delay of planned clinical trials or regulatory filings due to regulatory feedback or other developments. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission, including its quarterly report on Form 10-Q. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.

Investor
Contact:

Jason Fredette
Vice President, Investor Relations and Corporate Communications
Intellia Therapeutics, Inc.
[email protected]

Media Contact:

Mike Tattory
Vice President
LifeSci Communications
[email protected]

MIAMI, June 12, 2026 (GLOBE NEWSWIRE) — Defiance ETFs, a leader in thematic and leveraged exchange-traded funds, today announced that trading in shares of the Defiance Daily 2X Space ETF (Cboe BZX: SPCL) was temporarily halted by Cboe BZX Exchange, Inc. (the “Exchange”).

Trading was temporarily halted at 10:45 A.M. EDT today.

SPCL’s trading halt was the result of the Exchange exercising its broad discretionary authority to halt trading in a listed ETF, as authorized by Exchange rules. According to the Exchange, it currently anticipates lifting the temporary halt of trading in SPCL shares and resuming trading no earlier than Monday, June 15, 2026.

During this temporary halt, investors are advised that while shares of SPCL cannot be bought or sold on the secondary market, the underlying portfolio assets remain secure and are not impacted by this temporary halt of trading initiated by the Exchange.

Defiance ETFs believes the temporary halt of trading in SPCL shares was in response to today’s significant market price volatility surrounding SpaceX’s completion of its initial public offering, and the commencement of trading in shares of SpaceX Class A common stock on Nasdaq (NASDAQ: SPCX).

Defiance ETFs will issue an update as soon as the Exchange authorizes the resumption of trading in shares of SPCL. For real-time updates on the status of SPCL, please monitor the Exchange’s Issuer Portal (SPCL) or contact your financial advisor.

For full fund details, the prospectus, holdings, and performance current to the most recent month-end, visit defianceetfs.com/spcl or call 833.333.9383.

An investment in SPCL is not a direct investment in the underlying securities. The Fund is not suitable for all investors. The Fund is designed to be utilized only by knowledgeable investors who understand the potential consequences of seeking daily leveraged (2X) investment results, understand the risks associated with the use of leverage, and are willing to monitor their portfolios frequently. The Fund is not intended to be used by, and is not appropriate for, investors who do not intend to actively monitor and manage their portfolios. The Fund pursues daily leveraged investment objectives, which means it is riskier than alternatives that do not use leverage. The Fund magnifies the performance of the Target Portfolio and is designed strictly for short-term use. For periods longer than a single day, the Fund’s performance will be the result of compounded daily returns, which is very likely to differ from 200% of the return of the Target Portfolio over the same period. It is possible that investors could lose their entire principal within a single trading day.

Important Disclosures

Defiance ETFs LLC is the ETF sponsor. The Fund’s investment adviser is Tidal Investments LLC (“Tidal” or the “Adviser”).

The Fund’s investment objectives, risks, charges, and expenses must be considered carefully before investing. The prospectus and summary prospectus contain this and other important information and can be obtained by calling 833.333.9383 or by visiting defianceetfs.com/spcl. Please read the prospectus and summary prospectus carefully before investing.

An investment in the Fund involves a high degree of risk. An investor could lose the full principal value of his or her investment within a single day.

Strategy and Reconstitution Risk. The Fund is actively managed and, per its recently amended Prospectus, may reconstitute its portfolio to consist of exposure to a single Space Company security in response to a “Material Space Event” – defined to include an initial public offering of a company, such as SpaceX, which the Adviser determines to be a significant participant in the space economy. SpaceX’s IPO, a Material Space Event, will result in the Fund holding all or a predominant portion of its portfolio in instruments providing exposure to SpaceX shares, subjecting existing and future Shareholders to a substantially more concentrated and potentially more volatile investment portfolio due to such an event. Fund investment results following a reconstitution in response to a Material Space Event may differ materially from prior results and the Fund may as a result temporarily deviate from its daily targeted exposure level. The Fund’s prospectus does not require the Adviser to provide advance notice before a reconstitution; however, the Fund’s Target Portfolio is published daily on its website at www.defianceetfs.com/spcl.

An investment in the Fund is not an investment in SpaceX. The Fund seeks to obtain exposure to SpaceX Class A common stock, and to other Space Company securities, through derivatives, not by holding the underlying securities directly. Fund holdings are subject to change at any time and should not be considered a recommendation to buy or sell any security.

Focused Portfolio and Concentration Risk. The Fund may seek exposure to one or a limited number of Space Company securities, including SpaceX. Given the Fund’s exposure is concentrated in a one or a limited number of underlying stocks, such as SpaceX, the Fund is subject to the price movements, business results, regulatory developments, and other risks specific to SpaceX or other Space Companies. The Fund is significantly less diversified than traditional ETFs, and its performance is more volatile than a fund seeking exposure to a broader market sector or seeking to track a broad-based securities index.

Leverage, Compounding and Daily Reset Risk. The Fund seeks daily investment results equal to 200% of the daily performance of a Target Portfolio consisting of one or a limited number of Space Company securities, which may include or consistent entirely of SpaceX Class A common stock due to the Material Space Event. The Fund obtains exposure in excess of its net assets through leverage, which magnifies both gains and losses. The Fund’s returns over periods longer than a single day will likely differ, in amount and possibly direction, from its stated daily target. For periods longer than a single day, the Fund will lose money if its Target Portfolio performance is flat, and it is possible that the Fund will lose money even if its Target Portfolio’s performance increases. The Fund is intended for short-term use and is not appropriate for investors who do not intend to actively monitor and manage their portfolios.

Newly Public Company Risk. SpaceX has recently completed, or is in the process of completing, its initial public offering. The first day of trading in a newly public company’s securities frequently involves extraordinary market activity and may differ significantly from subsequent trading days. For example, trading in SpaceX common stock may be characterized by substantial price volatility, rapid price movements, significant differences between the IPO price and the opening market price, wide bid-ask spreads, trading imbalances, limited liquidity, trading halts, and other market disruptions. These conditions may make it difficult for market participants to value SpaceX common stock and may contribute to significant fluctuations in the market price of the Fund’s Shares.

SpaceX-Specific Risks. The Fund’s exposure to SpaceX stock will subject it to risks specific to SpaceX, including its expected status as a controlled company with voting power concentrated in founder Elon Musk through Class B common stock (10 votes per share), the Fund’s dependence on Mr. Musk’s services and reputation, and the execution risk associated with unproven or novel technologies such as the Starship program, next-generation Starlink satellites, and orbital AI initiatives.

Initial Trading Day IPO Exposure Risk. The Fund expects to seek exposure to the performance of SpaceX common stock measured from the opening market price of SpaceX common stock on its first day of exchange trading. The Fund will not seek to provide exposure to the difference between the IPO offering price and the opening market price of SpaceX common stock. There can be no assurance that the Fund will be able to obtain, maintain, or rebalance its desired level of exposure to the performance of SpaceX common stock during its in initial day of trading.

Derivatives Capacity Constraints Risk. Because SpaceX will be a newly public company, the markets for swap agreements, options contracts, and other instruments that the Fund may use to obtain leveraged exposure may be limited, illiquid, volatile, costly, or unavailable. Counterparties may impose exposure limits, exchanges may impose position limits or other restrictions, and market participants may be unwilling or unable to provide the Fund with the desired level of exposure. These constraints may increase tracking error, cause the Fund to return substantially less than its desired daily leveraged exposure to the performance of SpaceX stock, or prevent the Fund from achieving its investment objective. These risks may be particularly pronounced during the period immediately following an IPO, when trading volumes, liquidity conditions, derivatives availability, counterparty capacity, price discovery, and market volatility may be highly uncertain.

Derivatives and Non-Diversification Risk. The Fund uses swap agreements and/or listed options contracts to obtain economic exposure to its Target Portfolio securities, which are subject to counterparty, liquidity, valuation, correlation, and leverage risks, as well as the risk that a derivative will not perform as expected. The Fund is classified as non-diversified and may invest a larger portion of its assets providing exposure to a single issuer.

Tax Risk. The Fund’s use of swaps and other derivatives may produce taxable income, including ordinary income and short-term capital gains, which are generally taxable at higher rates than long-term capital gains.

Past performance does not guarantee future results. Fund holdings and exposures are subject to change at any time and should not be considered recommendations to buy or sell any security.

Defiance Daily 2X Space ETF is distributed by Foreside Fund Services, LLC.

About Defiance ETFs

Founded in 2018, Defiance is a leading ETF issuer specializing in thematic, income, and leveraged ETFs. Our first-mover leveraged single-stock ETFs empower investors to take amplified positions in high-growth companies, providing precise leverage exposure without the need to open a margin account.

Media Contact: Brenda Hentschel | [email protected] | 201.705.3758

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/e7d44c46-c91a-4fa4-9361-fe178f1ad310

DALLAS, June 12, 2026 (GLOBE NEWSWIRE) — Arcadia Biosciences, Inc.^® (Nasdaq: RKDA), a producer and marketer of innovative wellness products, announced today the closing of its previously announced private placement priced at-the-market under Nasdaq rules for the purchase and sale of 3,883,496 shares of its common stock (or pre-funded warrants in lieu thereof), Series A-1 preferred investment options to purchase up to an aggregate of 3,883,496 shares of common stock and Series A-2 preferred investment options to purchase up to an aggregate of 3,883,496 shares of common stock at a purchase price of $1.03 per share of common stock (or pre-funded warrant in lieu thereof) and associated preferred investment options.

H.C. Wainwright & Co. acted as the exclusive placement agent for the offering.

The Series A-1 preferred investment options have an exercise price of $0.91 per share, will be exercisable beginning on the effective date of stockholder approval of the issuance of the shares of common stock upon exercise of the Series A-1 preferred investment options and will expire five years from the effective date of stockholder approval. The Series A-2 preferred investment options have an exercise price of $0.91 per share, are exercisable immediately upon issuance and will expire twenty-four months from the effective date of the Resale Registration Statement (as defined below).

The aggregate gross proceeds to the company from the offering were approximately $4 million before deducting placement agent fees and other offering expenses. Arcadia intends to use the net proceeds from the offering for working capital and general corporate purposes.

The securities described above were offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the “Act”), and Regulation D promulgated thereunder and, along with the shares of common stock underlying the Series A-1 preferred investment options and Series A-2 preferred investment options, have not been registered under the Act or applicable state securities laws. Accordingly, the securities may not be offered or sold in the United States absent registration with the SEC or an applicable exemption from such registration requirements. Pursuant to a registration rights agreement, the Company has agreed to file one or more registration statements with the SEC covering the resale of the unregistered securities to be issued in the offering (the “Resale Registration Statement”).

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

About Arcadia Biosciences, Inc.

Since 2002, Arcadia Biosciences (Nasdaq: RKDA) has been innovating high-value, healthy ingredients to meet consumer demands for healthier choices. With its roots in agricultural innovation, Arcadia cultivates next-generation wellness products. For more information, visit www.arcadiabio.com.

Safe Harbor Statement

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements relate to future events or future results of operations concerning the company and its products, including, but not limited to, statements concerning the following matters: the receipt of any required stockholder approvals; and the anticipated use of proceeds from the offering. Undue reliance should not be placed on any forward-looking statements. Forward-looking statements are only predictions and are subject to known and unknown risks and uncertainties that could cause actual results to differ materially from the results anticipated by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risks set forth in filings that the company makes with the Securities and Exchange Commission from time to time, including in Arcadia’s Annual Report on Form 10-K for the year ended December 31, 2025 (the 2025 Form 10-K), and other filings that the company makes with the SEC. Forward-looking statements concerning anticipated future activities also assume that the company has sufficient funding to continue its operations and planned activities, which may not be the case. As described in greater detail in the 2025 Form 10-K, the company may require additional funding in the future to continue its operations and planned activities. There are no assurances that required funding will be available at all or will be available in sufficient amounts or on reasonable terms. The company may seek to raise additional funds through equity or debt financings, through transactions involving its other assets, or through other transactions. Any sale of additional equity securities could result in dilution to company stockholders. Reported results should not be considered as an indication of future performance. Forward-looking statements made in this press release speak only as of the date hereof, and except as required by law, Arcadia Biosciences, Inc. disclaims any obligation to update these forward-looking statements or to reflect events or circumstances arising after the date of this press release.

Arcadia Biosciences Contact
:

T.J. Schaefer
[email protected]

SAN DIEGO, June 12, 2026 (GLOBE NEWSWIRE) — Belite Bio, Inc (NASDAQ: BLTE) (“Belite Bio^®” or the “Company”), a clinical-stage drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced the completion of its rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for tinlarebant. Tinlarebant is an investigational, once-daily oral therapy for the treatment of Stargardt disease type 1 (STGD1), a rare, inherited retinal disease caused by mutations in the ABCA4 gene that leads to progressive and irreversible vision loss. STGD1 affects an estimated 53,000 people in the U.S. alone, and there are currently no approved treatment options for the disease.

The rolling NDA was initiated in April 2026 and was submitted under Breakthrough Therapy Designation (BTD), which was granted by the FDA due to the high unmet need among patients living with STGD1. The completed application will undergo the 60-day review period with the FDA, and if accepted, a Prescription Drug User Fee Act (PDUFA) target action date will be assigned.

“The completion of our NDA submission marks a pivotal moment for Belite Bio and represents an important step forward for those affected by Stargardt disease who have long faced a future of progressive vision loss without an approved treatment option,” said Dr. Tom Lin, Chairman and Chief Executive Officer of Belite Bio. “We are immensely grateful to everyone who made this milestone possible, including the patients, families, and investigators who participated in our clinical trials, and the entire Belite team. We look forward to honoring them by working with the FDA to advance tinlarebant through the regulatory review process, while continuing to focus on our commercial preparedness activities in anticipation of a timely and efficient launch following potential approval.”

“Stargardt disease places a profound burden on patients, often affecting them early in life and steadily diminishing central vision during critical years of education and independence,” said Dr. Hendrik Scholl, Chief Medical Officer of Belite Bio. “We believe that the results from the Phase 3 DRAGON trial, which demonstrated tinlarebant’s ability to significantly reduce the growth rate of retinal lesions as compared to placebo, underscore its benefit and pave the way for it to potentially become the first approved therapy for this devastating disease.”

About Tinlarebant (a/k/a LBS-008)

Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in STGD1 and also contribute to disease progression in geographic atrophy (GA), or advanced dry age-related macular degeneration (AMD). Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, tinlarebant reduces the formation of bisretinoids. Tinlarebant has been granted Breakthrough Therapy Designation, Fast Track Designation, and Rare Pediatric Disease Designation in the U.S., Orphan Drug Designation in the U.S., Europe, Japan, and Switzerland, and Sakigake Designation in Japan for the treatment of STGD1.

About Belite Bio

Belite Bio is a clinical-stage drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical need, such as Stargardt disease type 1 (STGD1) and geographic atrophy (GA) in advanced dry age-related macular degeneration (AMD), in addition to specific metabolic diseases. Belite Bio’s lead candidate, tinlarebant, is an oral therapy intended to reduce the accumulation of bisretinoid toxins in the eye. The Company has completed a Phase 3 trial (DRAGON) in adolescent and adult subjects with STGD1, which met its primary endpoint, and the drug is currently being evaluated in a Phase 2/3 trial (DRAGON II) in adolescent and adult subjects with STGD1 and a Phase 3 trial (PHOENIX) in subjects with GA. For more information, follow us on X, Instagram, LinkedIn, and Facebook, or visit us at www.belitebio.com.

Important Cautions Regarding 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, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements relate to future expectations, plans and prospects, as well as other statements regarding matters that are not historical facts. These statements include but are not limited to statements regarding the estimated STGD1 patient population in the U.S., Belite Bio’s advancement of regulatory milestones and planned commercialization of its product candidates, Belite Bio’s commercial preparedness and the timing and execution of a potential product launch following regulatory approval, the ability of tinlarebant to treat STGD1 and GA, the timing for the FDA to review Belite Bio’s NDA for tinlarebant, the potential acceptance by the FDA and the potential assignment of the PDUFA target action date, as well as any other statements regarding matters that are not historical facts, and any other statements containing the words “may”, “will”, “expect”, “believe”, “target”, “plan”, “intend”, “continue”, “hope”, “potential”, “anticipate”, “estimate”, “look forward”, and other similar expressions. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors related to Belite Bio’s business, including but not limited to Belite Bio’s ability to demonstrate the safety and efficacy of its drug candidates; the clinical results for its drug candidates, which may not support further development or regulatory approval; expectations for the timing of initiation, enrollment and completion of, and data relating to, its clinical trials; the timing to complete any ancillary clinical trials and/or to receive the interim/final data of such clinical trials; the timing to communicate with and submit trial data to regulatory authorities for drug approval in various jurisdictions; the content and timing of decisions made by the relevant regulatory authorities regarding regulatory approval of Belite Bio’s drug candidates; Belite Bio’s ability to successfully commercialize tinlarebant, if approved, including its ability to build out commercial infrastructure, achieve market acceptance, and execute a timely product launch; timing for Belite Bio to share additional data at upcoming medical meetings; the potential efficacy of tinlarebant to set a new benchmark for future research in inherited retinal disorders, as well as those risks more fully discussed in the “Risk Factors” section in Belite Bio’s filings with the U.S. Securities and Exchange Commission. All forward-looking statements are based on information currently available to Belite Bio, and Belite Bio undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Media and Investor Relations Contact:

Jennifer Wu / [email protected]
Argot Partners / [email protected]