Larimar Therapeutics Announces Dosing of Adolescents in Nomlabofusp Pediatric Pharmacokinetic Run-In Study for Patients with Friedreich’s Ataxia
Larimar Therapeutics (LRMR) has initiated dosing of adolescents aged 12-17 in its pediatric pharmacokinetic (PK) run-in study for nomlabofusp, a treatment for Friedreich's ataxia (FA). The study participants receive weight-based doses equivalent to the 50 mg adult dose and are randomized 2:1 to receive either nomlabofusp or placebo daily for seven days.
Adolescents who complete the PK run-in study will be eligible to participate in the ongoing open label extension (OLE) study. The company plans to initiate a second cohort for children aged 2-11 in the first half of 2025. Long-term 50 mg data from adults in the OLE study and available adolescent PK run-in data are expected by mid-2025.
The OLE study evaluates safety, tolerability, PK, and FXN levels in buccal and skin cells, along with exploratory pharmacodynamic markers and clinical outcome measures following long-term subcutaneous administration of nomlabofusp.
Larimar Therapeutics (LRMR) ha avviato la somministrazione a ragazzi di età compresa tra 12 e 17 anni nel suo studio di farmacocinetica pediatrica (PK) per nomlabofusp, un trattamento per l'atassia di Friedreich (FA). I partecipanti allo studio ricevono dosi basate sul peso equivalenti alla dose per adulti di 50 mg e sono randomizzati 2:1 per ricevere quotidianamente nomlabofusp o placebo per sette giorni.
I ragazzi che completano lo studio PK saranno idonei a partecipare allo studio in aperto esteso (OLE) in corso. L'azienda prevede di avviare una seconda coorte per bambini di età compresa tra 2 e 11 anni nella prima metà del 2025. I dati a lungo termine di 50 mg degli adulti nell'OLE e i dati disponibili dello studio PK sui ragazzi sono previsti per metà 2025.
Lo studio OLE valuta la sicurezza, la tollerabilità, la farmacocinetica e i livelli di FXN nelle cellule buccali e cutanee, insieme a marcatori farmacodinamici esplorativi e misure di esito clinico dopo somministrazione sottocutanea a lungo termine di nomlabofusp.
Larimar Therapeutics (LRMR) ha iniciado la dosificación en adolescentes de 12 a 17 años en su estudio de inicio de farmacocinética pediátrica (PK) para nomlabofusp, un tratamiento para la ataxia de Friedreich (FA). Los participantes del estudio reciben dosis basadas en el peso equivalentes a la dosis de 50 mg para adultos y son aleatorizados en una proporción de 2:1 para recibir ya sea nomlabofusp o un placebo diariamente durante siete días.
Los adolescentes que completen el estudio de inicio de PK serán elegibles para participar en el estudio en abierto extendido (OLE) que está en curso. La compañía planea iniciar una segunda cohorte para niños de 2 a 11 años en la primera mitad de 2025. Se espera que los datos a largo plazo de 50 mg de adultos en el estudio OLE y los datos de PK disponibles para adolescentes estén listos para mediados de 2025.
El estudio OLE evalúa la seguridad, tolerabilidad, PK y niveles de FXN en células bucales y dérmicas, junto con marcadores farmacodinámicos exploratorios y medidas de resultado clínico después de la administración subcutánea a largo plazo de nomlabofusp.
라리마르 테라퓨틱스 (LRMR)는 프리드리히 운동실조증(FA) 치료제인 노멀로바푸스프에 대한 소아 약리학적(PK) 예비 연구에서 12세에서 17세의 청소년에 대한 투약을 시작했습니다. 연구 참가자들은 성인 용량인 50mg에 해당하는 체중 기반의 용량을 받으며, 무작위 2:1 비율로 노멀로바푸스프 또는 위약을 매일 7일 동안 받습니다.
PK 예비 연구를 완료한 청소년은 진행 중인 개방형 연장 연구(OLE)에 참여할 자격이 주어집니다. 회사는 2025년 상반기에 2세에서 11세 어린이를 대상으로 두 번째 코호트를 시작할 계획입니다. OLE 연구에서 성인에 대한 장기 50mg 데이터와 사용 가능한 청소년 PK 예비 데이터는 2025년 중반에 예상됩니다.
OLE 연구는 노멀로바푸스프의 장기 피하 투여 후 구강 및 피부세포에서의 안전성, 내약성, PK 및 FXN 수준과 탐색적 약리역학적 지표 및 임상 결과 측정을 평가합니다.
Larimar Therapeutics (LRMR) a lancé la dose chez des adolescents âgés de 12 à 17 ans dans son étude de pharmacocinétique pédiatrique (PK) pour nomlabofusp, un traitement pour l'ataxie de Friedreich (FA). Les participants à l'étude reçoivent des doses basées sur le poids équivalentes à la dose adulte de 50 mg et sont randomisés 2:1 pour recevoir soit nomlabofusp, soit un placebo chaque jour pendant sept jours.
Les adolescents qui terminent l'étude PK seront éligibles pour participer à l'étude ouverte d'extension en cours (OLE). La société prévoit de lancer une deuxième cohorte pour les enfants âgés de 2 à 11 ans dans la première moitié de 2025. Les données à long terme sur les 50 mg d'adultes dans l'étude OLE et les données PK disponibles concernant les adolescents devraient être prêtes d'ici la mi-2025.
L'étude OLE évalue la sécurité, la tolérance, la PK et les niveaux de FXN dans les cellules buccales et cutanées, ainsi que des marqueurs pharmacodynamiques exploratoires et des mesures des résultats cliniques après une administration sous-cutanée à long terme de nomlabofusp.
Larimar Therapeutics (LRMR) hat die Dosierung von Jugendlichen im Alter von 12 bis 17 Jahren in seiner pädiatrischen Pharmakokinetik- (PK) Vorstudie für nomlabofusp, eine Behandlung für die Friedreich-Ataxie (FA), eingeleitet. Die Studienmitglieder erhalten gewichtsabhängige Dosen, die der Erwachsenendosis von 50 mg entsprechen, und werden im Verhältnis 2:1 randomisiert, um entweder nomlabofusp oder ein Placebo täglich über sieben Tage zu erhalten.
Jugendliche, die die PK-Vorstudi abschließen, sind berechtigt, an der laufenden offenen Verlängerungsstudie (OLE) teilzunehmen. Das Unternehmen plant, in der ersten Jahreshälfte 2025 eine zweite Kohorte für Kinder im Alter von 2 bis 11 Jahren zu starten. Langfristige 50 mg-Daten von Erwachsenen in der OLE-Studie und verfügbare PK-Vorstudi-Daten von Jugendlichen werden bis Mitte 2025 erwartet.
Die OLE-Studie bewertet Sicherheit, Verträglichkeit, PK und FXN-Spiegel in Mund- und Hautzellen sowie explorative pharmakodynamische Marker und klinische Ergebniskennzahlen nach einer langfristigen subkutanen Verabreichung von nomlabofusp.
- Expansion of clinical program to include pediatric patients
- Clear pathway for transition of pediatric patients to OLE study
- Structured approach with multiple age cohorts (12-17 and 2-11)
- None.
Insights
The initiation of adolescent dosing in Larimar's pediatric pharmacokinetic study represents a significant milestone in the clinical development of nomlabofusp for Friedreich's ataxia (FA). FA is a rare, progressive neurodegenerative disorder affecting approximately 1 in 50,000 people, with most cases diagnosed in childhood. Currently, there are treatment options, making this development particularly noteworthy.
The study's design demonstrates a methodical approach to pediatric drug development:
- Weight-based dosing equivalent to adult 50 mg dose ensures appropriate drug exposure
- 2:1 randomization ratio maximizes treatment exposure while maintaining scientific validity
- Stepwise age group progression (adolescents first, then children) follows standard safety protocols
- Integration with the open-label extension study provides continuity of treatment
From a clinical development perspective, this expansion into pediatric populations is important as FA typically manifests in childhood. Success in this program could significantly expand nomlabofusp's potential market reach. The planned data readout in mid-2025 will be particularly important as it will provide both long-term adult data and initial pediatric safety/PK data, potentially de-risking the program substantially.
However, investors should note several critical factors:
- Pediatric studies often face unique challenges in recruitment and retention
- Safety standards for pediatric populations are particularly stringent
- The step-wise approach, while prudent, means full pediatric data will take time to mature
This development positions Larimar to potentially address an important unmet medical need in a rare disease space, where successful treatments can command premium pricing. The systematic approach to pediatric development, combined with the parallel adult program, suggests a well-thought-out clinical strategy that could enhance the drug's commercial potential if successful.
- Adolescents receive a weight-based dose equivalent to the 50 mg adult dose
- Adolescents 12-17 years old who complete participation in the pharmacokinetic (PK) run-in study will be eligible to screen in the ongoing open label extension (OLE) study
- Larimar is continuing to enroll adolescent patients and plans to initiate a cohort of children 2-11 years old in 1H 2025
- Long-term 50 mg data from adults in OLE study and available adolescent PK run-in data expected mid-2025
BALA CYNWYD, Pa., Jan. 23, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced that dosing of adolescents 12-17 years old has started in the Company’s pediatric PK run-in study for patients with Friedreich’s ataxia (FA).
“Dosing adolescents is the first step in evaluating the safety and PK of nomlabofusp in pediatric patients with FA. We continue to enroll adolescents in our first cohort. This cohort will be followed by a second cohort of children 2-11 years old. We expect to transition both the adolescents and children into the ongoing OLE study after assessing safety and exposure data from each successive cohort,” said Dr. Rusty Clayton, Chief Medical Officer of Larimar. “We look forward to reporting long-term 50 mg data in adults from our OLE study, as well as available data from adolescents completing the pediatric PK run-in study, in mid- 2025.”
Study participants in the PK run-in study are randomized 2:1 to receive either nomlabofusp or placebo daily for seven days. Following assessment of safety and PK data of each successive cohort, participants will be eligible to screen for the OLE study. A cohort of children (2- 11 years old) is planned to initiate in the first half of 2025.
The OLE study is evaluating the safety and tolerability, PK, and FXN levels in buccal and skin cells, along with exploratory pharmacodynamic markers (lipid profiles and gene expression) and clinical outcome measures, following long-term subcutaneous administration of nomlabofusp.
About Larimar Therapeutics
Larimar Therapeutics, Inc. (Nasdaq: LRMR), is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. Larimar’s lead compound, nomlabofusp, is being developed as a potential treatment for Friedreich's ataxia. Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds. For more information, please visit: https://larimartx.com.
Forward-Looking Statements
This press release contains forward-looking statements that are based on Larimar’s management’s beliefs and assumptions and on information currently available to management. All statements contained in this release other than statements of historical fact are forward-looking statements, including but not limited to statements regarding Larimar’s ability to develop and commercialize nomlabofusp and other planned product candidates, Larimar’s planned research and development efforts, including the timing of its nomlabofusp clinical trials, interactions and filings with the FDA, expectations regarding potential for accelerated approval or accelerated access and time to market and overall development plan and other matters regarding Larimar’s business strategies, ability to raise capital, use of capital, results of operations and financial position, and plans and objectives for future operations.
In some cases, you can identify forward-looking statements by the words “may,” “will,” “could,” “would,” “should,” “expect,” “intend,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “ongoing” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. These statements involve risks, uncertainties and other factors that may cause actual results, performance, or achievements to be materially different from the information expressed or implied by these forward-looking statements. These risks, uncertainties and other factors include, among others, the success, cost and timing of Larimar’s product development activities, nonclinical studies and clinical trials, including nomlabofusp clinical milestones and continued interactions with the FDA; that preliminary clinical trial results may differ from final clinical trial results, that earlier non-clinical and clinical data and testing of nomlabofusp may not be predictive of the results or success of later clinical trials, and assessments; that the FDA may not ultimately agree with Larimar’s nomlabofusp development strategy; the potential impact of public health crises on Larimar’s future clinical trials, manufacturing, regulatory, nonclinical study timelines and operations, and general economic conditions; Larimar’s ability and the ability of third-party manufacturers Larimar engages, to optimize and scale nomlabofusp’s manufacturing process; Larimar’s ability to obtain regulatory approvals for nomlabofusp and future product candidates; Larimar’s ability to develop sales and marketing capabilities, whether alone or with potential future collaborators, and to successfully commercialize any approved product candidates; Larimar’s ability to raise the necessary capital to conduct its product development and commercialization activities; and other risks described in the filings made by Larimar with the Securities and Exchange Commission (SEC), including but not limited to Larimar’s periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the SEC and available at www.sec.gov. These forward-looking statements are based on a combination of facts and factors currently known by Larimar and its projections of the future, about which it cannot be certain. As a result, the forward-looking statements may not prove to be accurate. The forward-looking statements in this press release represent Larimar’s management’s views only as of the date hereof. Larimar undertakes no obligation to update any forward-looking statements for any reason, except as required by law.
Investor Contact:
Joyce Allaire
LifeSci Advisors
jallaire@lifesciadvisors.com
(212) 915-2569
Company Contact:
Michael Celano
Chief Financial Officer
mcelano@larimartx.com
(484) 414-2715
FAQ
When will Larimar (LRMR) report data from the nomlabofusp pediatric PK run-in study?
What age groups are included in LRMR's pediatric study for nomlabofusp?
What is the dosing protocol in LRMR's pediatric PK run-in study?
What endpoints are being evaluated in LRMR's nomlabofusp OLE study?
