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Data Demonstrating ENPP1 Inhibition as a Therapeutic Approach for Later-onset Hypophosphatasia Presented at the American Society for Bone and Mineral Research 2024 Annual Meeting

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Rallybio (Nasdaq: RLYB) presented nonclinical data at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting, demonstrating ENPP1 inhibition as a potential therapeutic approach for hypophosphatasia (HPP). The study, using an early lead ENPP1 inhibitor (REV101) in a mouse model of later-onset HPP, showed promising results:

1. Oral dosing lowered PPi by 30%
2. Improved mineralization of long and vertebrate bones
3. ENPP1 inhibition was safe and well-tolerated

Rallybio and Exscientia plc (Nasdaq: EXAI) are developing an improved ENPP1 inhibitor for HPP treatment, with a development candidate expected to be nominated in Q4 2024. This approach could potentially address the unmet need in HPP patients, particularly adults.

Rallybio (Nasdaq: RLYB) ha presentato dati non clinici durante il 2024 Annual Meeting della American Society for Bone and Mineral Research (ASBMR), dimostrando che l'inibizione di ENPP1 rappresenta un potenziale approccio terapeutico per l'ipofosfatasia (HPP). Lo studio, che ha utilizzato un inibitore precoce di ENPP1 (REV101) in un modello murino di HPP a insorgenza tardiva, ha mostrato risultati promettenti:

1. La somministrazione orale ha ridotto il PPi del 30%
2. Migliorata mineralizzazione delle ossa lunghe e vertebrali
3. L'inibizione di ENPP1 è stata sicura e ben tollerata

Rallybio e Exscientia plc (Nasdaq: EXAI) stanno sviluppando un inibitore di ENPP1 migliorato per il trattamento dell'HPP, con un candidato per lo sviluppo previsto per essere nominato nel quarto trimestre del 2024. Questo approccio potrebbe potenzialmente risolvere le esigenze insoddisfatte nei pazienti affetti da HPP, in particolare negli adulti.

Rallybio (Nasdaq: RLYB) presentó datos no clínicos en la Reunión Anual de la American Society for Bone and Mineral Research (ASBMR) 2024, demostrando que la inhibición de ENPP1 es un enfoque terapéutico potencial para la hipofosfatasia (HPP). El estudio, que utilizó un inhibidor temprano de ENPP1 (REV101) en un modelo de ratón de HPP de aparición tardía, mostró resultados prometedores:

1. La administración oral redujo el PPi en un 30%
2. Mejoró la mineralización de los huesos largos y vertebrales
3. La inhibición de ENPP1 fue segura y bien tolerada

Rallybio y Exscientia plc (Nasdaq: EXAI) están desarrollando un inhibidor de ENPP1 mejorado para el tratamiento de HPP, con un candidato de desarrollo que se espera ser nominado en el cuarto trimestre de 2024. Este enfoque podría abordar la necesidad insatisfecha en pacientes con HPP, particularmente en adultos.

Rallybio (Nasdaq: RLYB)는 2024년 미국 골대사학회(ASBMR) 연례 회의에서 비임상 데이터를 발표하며 ENPP1 억제저인산효소증 (HPP) 치료를 위한 잠재적 치료 접근법임을 입증했습니다. 이 연구는 후기 발현 HPP 마우스 모델에서 초기 ENPP1 억제제(REV101)를 사용하여 유망한 결과를 보였습니다:

1. 경구 투여 시 PPi가 30% 감소함
2. 긴 뼈와 척추의 미네랄화 개선
3. ENPP1 억제가 안전하고 잘 견딜 수 있음

Rallybio와 Exscientia plc (Nasdaq: EXAI)는 HPP 치료를 위한 개선된 ENPP1 억제제를 개발하고 있으며, 2024년 4분기에 개발 후보가 지명될 것으로 예상하고 있습니다. 이 접근법은 특히 성인 HPP 환자들의 충족되지 않은 필요를 해결할 수 있습니다.

Rallybio (Nasdaq: RLYB) a présenté des données non cliniques lors de la réunion annuelle 2024 de l'American Society for Bone and Mineral Research (ASBMR), démontrant que l'inhibition de l'ENPP1 pourrait être une approche thérapeutique potentielle pour l'hypophosphatasie (HPP). L'étude, utilisant un inhibiteur précoce de l'ENPP1 (REV101) dans un modèle murin de HPP à apparition tardive, a montré des résultats prometteurs :

1. L'administration orale a réduit le PPi de 30%
2. Amélioration de la minéralisation des os longs et vertébraux
3. L'inhibition de l'ENPP1 a été sûre et bien tolérée

Rallybio et Exscientia plc (Nasdaq: EXAI) développent un inhibiteur d'ENPP1 amélioré pour le traitement de l'HPP, avec un candidat au développement prévu pour être désigné au quatrième trimestre 2024. Cette approche pourrait potentiellement répondre aux besoins non satisfaits des patients atteints de HPP, en particulier des adultes.

Rallybio (Nasdaq: RLYB) präsentierte nichtklinische Daten auf dem 2024 Annual Meeting der American Society for Bone and Mineral Research (ASBMR), die zeigen, dass die ENPP1-Hemmung einen potenziellen therapeutischen Ansatz für die Hypophosphatasie (HPP) darstellt. Die Studie, die einen frühen ENPP1-Hemmer (REV101) in einem Mausmodell für spät einsetzende HPP verwendete, zeigte vielversprechende Ergebnisse:

1. Orale Dosierung senkte den PPi um 30%
2. Verbesserung der Mineralisierung von langen und Wirbelknochen
3. ENPP1-Hemmung war sicher und gut verträglich

Rallybio und Exscientia plc (Nasdaq: EXAI) entwickeln einen verbesserten ENPP1-Hemmer zur Behandlung von HPP, wobei ein Entwicklungskandidat im vierten Quartal 2024 nominiert werden soll. Dieser Ansatz könnte potenziell die unerfüllten Bedürfnisse von HPP-Patienten, insbesondere Erwachsenen, ansprechen.

Positive
  • Nonclinical data supports ENPP1 inhibition as a therapeutic approach for hypophosphatasia (HPP)
  • Early lead ENPP1 inhibitor (REV101) showed 30% reduction in PPi levels in HPP mouse model
  • Improved mineralization of long and vertebrate bones observed in the study
  • ENPP1 inhibition demonstrated to be safe and well-tolerated
  • Development candidate nomination expected in Q4 2024
Negative
  • None.

Insights

The presentation of nonclinical data on ENPP1 inhibition for hypophosphatasia (HPP) treatment at ASBMR 2024 is a significant development. The study demonstrates that oral dosing of an early lead ENPP1 inhibitor, REV101, reduced inorganic pyrophosphate (PPi) by 30% in adult HPP mice, improving bone mineralization. This is the first evidence showing ENPP1 as a druggable target for later-onset HPP.

Key points:

  • HPP is a rare genetic disorder with unmet needs, especially in adult patients
  • ENPP1 inhibition shows promise as a therapeutic approach
  • Rallybio and Exscientia's joint venture is developing an improved ENPP1 inhibitor
  • A development candidate is expected to be nominated in Q4 2024

While this news is encouraging for HPP patients, it's important to note that these are early-stage results. The path to a marketable drug still requires extensive clinical trials to prove safety and efficacy in humans. Investors should monitor the progress of the development candidate nomination and subsequent clinical trials.

This announcement represents a significant milestone in Rallybio's pipeline development. The positive nonclinical data for ENPP1 inhibition in HPP treatment showcases the company's potential in addressing rare diseases with high unmet needs. Several factors make this news impactful:

  • Novel approach: Targeting ENPP1 for HPP is innovative and could differentiate Rallybio in the market
  • Collaboration strength: The joint venture with Exscientia, an AI-driven drug discovery company, could accelerate development and improve chances of success
  • Near-term catalyst: The expected nomination of a development candidate in Q4 2024 provides a clear milestone for investors to watch
  • Market potential: While HPP is rare, successful rare disease treatments can command high prices, potentially leading to significant revenues if approved

However, investors should be cautious as early-stage success doesn't guarantee clinical or commercial success. The company's $46.5 million market cap suggests it's still in a speculative phase. Future funding needs and clinical trial outcomes will be critical for Rallybio's long-term prospects.

NEW HAVEN, Conn.--(BUSINESS WIRE)-- Rallybio Corporation (Nasdaq: RLYB), a clinical-stage biotechnology company translating scientific advances into transformative therapies for patients with devastating rare diseases, announced today that nonclinical data demonstrating ENPP1 inhibition as a therapeutic approach for the treatment of patients with hypophosphatasia (HPP) was presented at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting. ASBMR is being held September 27 – 30, 2024 in Toronto, Canada.

“From my team’s earlier work published in 2002 and 2005, we knew that ENPP1 could be a targetable molecule to modulate TNAP’s all-important substrate PPi. I am delighted that we have now been able to demonstrate the efficacy of this principle in our mouse model of later-onset HPP,” said José Luis Millán, Ph.D., Professor, Human Genetics Program at Sanford Children’s Health Research Center, Sanford Burnham Prebys Medical Discovery Institute, and author of the study.

HPP is a rare, genetic, metabolic disorder characterized by poor bone mineralization. The disease has a broad spectrum of symptoms and severity ranging from the life-threatening perinatal- and infantile-onset form to the less severe juvenile-onset form that can manifest with frequent bone fractures, significant joint and bone pain, and joint swelling. HPP is caused by loss-of-function mutations in the gene that encodes tissue-nonspecific alkaline phosphatase (TNAP), whose deficiency results in the accumulation of extracellular inorganic pyrophosphate (PPi), an inhibitor of bone mineralization.

“The data presented at ASBMR supports ENPP1 inhibition as a therapeutic approach for patients with hypophosphatasia,” said Stephen Uden, MD, Chief Executive Officer of Rallybio. “There is significant unmet patient need in HPP, particularly in adults. We believe this data with an early lead ENPP1 inhibitor from our joint venture with Exscientia is very promising and gives us enthusiasm that the development candidate we expect to nominate in the fourth quarter could be a safe and effective treatment to meaningfully improve the lives of patients suffering from HPP.”

This nonclinical study was designed to assess whether ENPP1 could be a druggable target to treat the non-lethal forms of HPP using an early lead ENPP1 inhibitor and the Alpl-/Prx1 mouse, which is a model of later-onset HPP.

Results indicate that oral dosing of an early lead ENPP1 inhibitor, REV101, to adult HPP mice lowered PPi by 30%, leading to improvements in mineralization of long and vertebrate bones. Furthermore, data showed that ENPP1 inhibition was safe and well-tolerated, and, for the first time, showed that ENPP1 is a druggable target for later-onset HPP.

Rallybio and Exscientia plc (Nasdaq: EXAI) are developing an ENPP1 inhibitor with improved properties compared with REV101 as a differentiated therapy to address the unmet need in patients with HPP. Rallybio and Exscientia expect to nominate a development candidate in the fourth quarter of 2024.

Details of yesterday’s poster presentation:

Title: ENPP1 Inhibition as a Therapeutic Approach for Later-onset Hypophosphatasia
Date/Time: September 29, 2024, 2:15pm – 3:45pm EDT
Presenter: Dr. José Luis Millán
Poster Number: Sun-LB 552

The poster will be available in the Publications & Presentations section of Rallybio’s website following the conclusion of the conference.

About Rallybio
Rallybio (NASDAQ: RLYB) is a clinical-stage biotechnology company with a mission to develop and commercialize life-transforming therapies for patients with severe and rare diseases. Rallybio has built a broad pipeline of promising product candidates aimed at addressing diseases with unmet medical need in areas of maternal fetal health, complement dysregulation, hematology, and metabolic disorders. The Company has two clinical stage programs: RLYB212, an anti-HPA-1a antibody for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT) and RLYB116, an inhibitor of complement component 5 (C5), with the potential to treat several diseases of complement dysregulation, as well as additional programs in preclinical development. Rallybio is headquartered in New Haven, Connecticut. For more information, please visit www.rallybio.com and follow us on LinkedIn and Twitter.

Forward-Looking Statements
This press release contains forward-looking statements that are based on our management’s beliefs and assumptions and on currently available information. All statements, other than statements of historical facts contained in this press release are forward-looking statements. In some cases, forward-looking statements can be identified by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements in this press release include, but are not limited to, statements concerning the timing of development candidate nomination for Rallybio’ ENPP1 inhibitor, whether Rallybio’s small molecule would successfully inhibit ENPP1, or demonstrate efficacy in later-onset HPP, or otherwise be safe when administered to humans. The forward-looking statements in this press release are only predictions and are based largely on management’s current expectations and projections about future events and financial trends that management believes may affect Rallybio’s business, financial condition and results of operations. These forward-looking statements speak only as of the date of this press release and are subject to a number of known and unknown risks, uncertainties and assumptions, including, but not limited to, our ability to successfully initiate and conduct our planned clinical trials, including the FNAIT natural history study, and the Phase 2 clinical trial for RLYB212, and complete such clinical trials and obtain results on our expected timelines, or at all, whether our cash resources will be sufficient to fund our operating expenses and capital expenditure requirements and whether we will be successful raising additional capital, our ability to enter into strategic partnerships or other arrangements, competition from other biotechnology and pharmaceutical companies, and those risks and uncertainties described in Rallybio’s filings with the U.S. Securities and Exchange Commission (SEC), including Rallybio’s Quarterly Report on Form 10-Q for the period ended June 30, 2024, and subsequent filings with the SEC. The events and circumstances reflected in our forward-looking statements may not be achieved or occur and actual future results, levels of activity, performance and events and circumstances could differ materially from those projected in the forward-looking statements. Except as required by applicable law, we are not obligated to publicly update or revise any forward-looking statements contained in this press release, whether as a result of any new information, future events, changed circumstances or otherwise.

Investor Contacts

Samantha Tracy

Rallybio Corporation

(475) 47-RALLY (Ext. 282)

investors@rallybio.com

Kevin Lui

Precision AQ

(212) 698-8691

kevin.lui@precisionaq.com

Media Contact

Victoria Reynolds

Mission North

(760) 579-2134

rallybio@missionnorth.com

Source: Rallybio Corporation

FAQ

What is the potential therapeutic approach for hypophosphatasia (HPP) presented by Rallybio (RLYB)?

Rallybio (RLYB) presented data demonstrating ENPP1 inhibition as a potential therapeutic approach for hypophosphatasia (HPP) at the ASBMR 2024 Annual Meeting.

What were the key findings of Rallybio's (RLYB) nonclinical study on ENPP1 inhibition for HPP?

The study showed that oral dosing of an early lead ENPP1 inhibitor (REV101) lowered PPi by 30%, improved mineralization of long and vertebrate bones, and was safe and well-tolerated in a mouse model of later-onset HPP.

When does Rallybio (RLYB) expect to nominate a development candidate for HPP treatment?

Rallybio (RLYB) and Exscientia (EXAI) expect to nominate a development candidate for HPP treatment in the fourth quarter of 2024.

What is the unmet need that Rallybio's (RLYB) ENPP1 inhibitor aims to address?

Rallybio's (RLYB) ENPP1 inhibitor aims to address the significant unmet need in hypophosphatasia (HPP) patients, particularly in adults, by potentially providing a safe and effective treatment to improve their lives.

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