Rallybio Announces Preliminary Phase 1 Multiple Ascending Dose Data for RLYB116, an Innovative Subcutaneously Injected Inhibitor of Complement Component 5
- RLYB116 demonstrated sustained mean reductions in free C5 of greater than 93%, including at Day 29 with measurement prior to the last dose.
- The mean estimated elimination half-life for RLYB116 was >300 hours, demonstrating a long-acting effect.
- The Company extended its cash runway to the third quarter of 2025, providing financial stability.
- The most common adverse event in the cohort was injection site reaction (ISR), occurring in 60% of participants.
- A participant receiving the 150 mg dose experienced liver enzyme test elevation, resulting in discontinuation and a reduction in the dose for the 3rd cohort from 150 mg to 125 mg.
Insights
The recent data from Rallybio Corporation's Phase 1 multiple ascending dose study of RLYB116 presents significant findings within the field of complement-mediated diseases, particularly generalized myasthenia gravis (gMG). The reported mean reduction of greater than 93% in free C5, a protein involved in the immune response, underscores the drug's potential efficacy. Moreover, the extension of the company's financial runway into the third quarter of 2025 indicates a strategic prioritization of resources that could impact the company's stock performance, as it suggests confidence in the drug's development pathway and potential market impact.
The pharmacokinetics and pharmacodynamics data, indicating low inter-subject variability and consistent increases in exposure relative to dose, are crucial for understanding the drug's behavior in the body and its scalability. The extended half-life of >300 hours suggests less frequent dosing, which could enhance patient compliance. However, the occurrence of injection site reactions in 60% of participants, although mild, is a concern that needs addressing to ensure patient comfort and adherence to treatment regimens.
From an investment perspective, the company's decision to focus on manufacturing process improvements before proceeding to Phase 2 trials indicates a careful approach to drug development, with the aim of broadening the therapeutic indications of RLYB116. This could potentially open up a larger market for the drug, addressing a wider range of complement-mediated diseases beyond gMG, such as paroxysmal nocturnal hemoglobinuria and antiphospholipid syndrome.
Analyzing the financial implications of the recent Phase 1 study results for RLYB116, the positive preliminary data could be a catalyst for Rallybio's stock performance, subject to market perception and regulatory progress. The company's updated cash runway guidance, now extending into Q3 2025, provides a more extended period for the development of RLYB116 and RLYB212 without the immediate need for additional capital raising, which may be favorably viewed by investors concerned about dilution. The strategic allocation of resources towards manufacturing enhancements rather than rushing into Phase 2 trials demonstrates fiscal prudence and a focus on long-term value creation.
However, it is important to note that the biotech sector is highly volatile and dependent on clinical outcomes, regulatory approvals and market acceptance. Investors should consider the inherent risks of drug development, including the possibility of adverse events in later-stage trials, regulatory hurdles and competition from other therapies. The investment in the manufacturing process, while potentially increasing the drug's market applicability, also represents a significant cost that must be weighed against the anticipated benefits.
The therapeutic landscape for complement-mediated diseases is evolving and RLYB116's promising results position it as a potential key player in this specialty pharmaceutical sector. The drug's once-a-week subcutaneous administration could differentiate it from competitors, offering convenience and potentially better patient adherence compared to more frequent dosing schedules. The focus on subcutaneous delivery aligns with current industry trends towards less invasive and more patient-friendly drug administration methods.
Moreover, the company's strategic decision to enhance the manufacturing process before advancing to later-stage clinical trials is indicative of a broader industry trend where companies are increasingly investing in production capabilities early on to avoid future bottlenecks. This could give Rallybio a competitive advantage if RLYB116 is approved for market release. Nevertheless, the company will need to navigate a complex regulatory environment and demonstrate clear benefits over existing treatments to gain a significant market share.
-- 100 mg Results Demonstrated a Mean Reduction of Greater than
-- Data Supports the Study of RLYB116 as a Differentiated Therapeutic for the Treatment of Generalized Myasthenia Gravis --
-- Company Announces Extension of Runway to 3Q 2025 As Part of Portfolio Prioritization --
-- Conference Call and Webcast Today at 8:30 AM Eastern Time --
The Phase 1 MAD study for RLYB116 evaluated the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous RLYB116 in healthy participants with multiple dose administration. The MAD study utilized an adaptive design and included four cohorts of 12 participants receiving doses of up to 200 mg per week of RLYB116 or placebo, with a four-week treatment duration and a 10-week follow-up period.
The preliminary results showed:
-
A 100 mg low volume (1 mL) once-a-week dose of subcutaneously administered RLYB116 achieved sustained mean reductions in free C5 of greater than
93% , including at Day 29 with measurement prior to the last dose. The reduction in free C5 at 24 hours after the first dose of 100 mg was greater than99% . These data and additional work we have conducted with RLYB116 reinforce our confidence that RLYB116 has the potential to be an effective treatment for patients with certain complement-mediated diseases, including generalized myasthenia gravis (gMG). - RLYB116 also demonstrated low inter-subject variability and consistent increases in exposure relative to dose. The mean estimated elimination half-life for RLYB116 was >300 hours.
-
In comparison to 100 mg weekly administration, higher concentrations of RLYB116 were observed in a cohort with 100 mg administered twice per week and were associated with a greater than
97% mean reduction in free C5. -
RLYB116 administered as a 100 mg once-a-week dose was observed to be generally well tolerated. The most common adverse event (AE) in the cohort was injection site reaction (ISR), which occurred in
60% of the participants in the cohort. All AEs during subcutaneous administration with the 100 mg weekly dose were mild in severity. -
The ISR rate for all participants in the 4 cohorts was
59% and all were mild in severity. There were no serious AEs reported for participants receiving study treatment. - A participant with a history of hepatitis A receiving the 150 mg dose experienced liver enzyme test elevation that resulted in discontinuation and a reduction in the dose for the 3rd cohort from 150 mg to 125 mg.
- The measurement of anti-drug antibody (ADA) formation in the study did not demonstrate an effect on PK or PD parameters and did not appear to be associated with an effect on AE incidence or severity.
“The preliminary results from this Phase 1 multiple ascending dose study of RLYB116 support continued development in patients with gMG,” said Eric Watsky, M.D., RLYB116 Program Lead for Rallybio. “We are encouraged by the free C5 reductions demonstrated by RLYB116 as well as the exposures achieved with subcutaneous administration. Through enhancements in the manufacturing process, we believe we have the opportunity to increase the dose of RLYB116 and improve the tolerability thereby opening up the opportunity to treat a wider range of complement mediated diseases. Our market research is consistent with our belief that an effective, once-a-week, well-tolerated therapy that can be rapidly self-administered with an autoinjector would be an attractive alternative for patients.”
RLYB116 Near-Term Development Plans and Cash Runway
The preliminary data from the Phase 1 MAD study confirm improvements made to date in the manufacturing process will enable the Company to advance RLYB116 into studies in patients. Rather than immediately proceed to a Phase 2 study in gMG, the Company intends to prioritize near-term investments in RLYB116 in the manufacturing process. The Company expects that the additional manufacturing work will improve tolerability at higher doses with a low injection volume and infrequent subcutaneous administration. The Company believes such enhancements will enable higher exposure to RLYB116 and potentially increase C5 reduction, which can result in treating a broader range of complement-mediated diseases, including paroxysmal nocturnal hemoglobinuria and antiphospholipid syndrome. In addition, this will allow the Company to direct available cash resources to advance RLYB212, its product candidate for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT).
The Company is also updating its cash runway guidance and now expects its current cash, cash equivalents and marketable securities to extend the runway into the third quarter of 2025.
“We are pleased to see substantial reductions in free C5 with once weekly subcutaneous dosing of RLYB116,” said Stephen Uden, M.D., Chief Executive Officer of Rallybio. “The Phase 1 MAD data show us that RLYB116 can be a potential therapeutic to treat gMG and other complement mediated diseases. In the spirit of managing our cash runway to realize the most value from our portfolio, we have decided to focus our RLYB116 investments on the manufacturing process with a goal of expanding the scope of therapeutic indications and addressing unmet medical need. In parallel, we continue to advance our lead program, RLYB212, and have extended our runway into the third quarter of 2025.”
Conference Call Information
Rallybio will host a conference call and webcast today, December 20, 2023 at 8:30 a.m. Eastern Time to discuss the RLYB116 Phase 1 multiple ascending dose (MAD) study. The live webcast and replay may be accessed by visiting Rallybio’s website at http://investors.rallybio.com. In addition, key slides from the RLYB116 Phase 1 MAD study will be discussed on the conference call and are posted to the “Events and Presentations” section of the Rallybio website. A replay of the webcast will be available on the Rallybio website for 30 days following the event.
About RLYB116 Phase 1 Study
RLYB116 is an innovative, long-acting, subcutaneously injected inhibitor of C5 in development for the treatment of patients with complement-mediated diseases. Phase 1 in healthy participants included the study of RLYB116 as a single ascending dose and multiple ascending dose. The multiple ascending dose (MAD) study of RLYB116 included an adaptive single-blind design initiated in the first quarter of 2023 with a 4-week treatment duration to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of subcutaneous RLYB116 in healthy participants with multiple dose administration.
The MAD study of RLYB116 included 4 cohorts: Cohort 1 (weekly dosing of 100 mg), Cohort 2 (3 doses of 100 mg the first week followed by weekly dosing), Cohort 3 (150 mg weekly dosing reduced to 125 mg weekly dosing) and Cohort 4 (75 mg twice the first week followed by 100 mg twice per week).
Post-treatment / study follow-up continued for 10 weeks.
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
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 results from the Phase 1 MAD study of RLYB116; potential clinical effects and benefits of RLYB116, including for the treatment of gMG; the timing and initiation of future clinical studies for RLBY116; the success cost and timing of our clinical development of our product candidates, including RLYB212 and RLYB116; and statements concerning the Company’s anticipated use of cash and cash runway. 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 studies, and complete such clinical studies 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
View source version on businesswire.com: https://www.businesswire.com/news/home/20231219094324/en/
Investors
Ami Bavishi
Head of Investor Relations and Communications
475-47-RALLY (Ext. 282)
abavishi@rallybio.com
Hannah Deresiewicz
Stern Investor Relations, Inc.
212-362-1200
hannah.deresiewicz@sternir.com
Media
Jorge Gaeta
Mission North
(516) 430-7659
Rallybio@missionnorth.com
Source: Rallybio Corporation
FAQ
What are the preliminary Phase 1 MAD data for RLYB116?
What is the ticker symbol for Rallybio Corporation?
What is the expected cash runway for Rallybio Corporation?
What adverse events were reported in the Phase 1 MAD study for RLYB116?
What was the most notable outcome of the Phase 1 MAD study for RLYB116?
