Latest news with #RLYB116
Yahoo
08-07-2025
- Business
- Yahoo
Rallybio Sells Interest in REV102 Program to Recursion Pharmaceuticals
– Company Eligible to Receive Up to $25 Million, Including an Upfront Equity Payment of $7.5 Million – – Extends Rallybio Cash Runway to Mid-2027 – NEW HAVEN, Conn., July 08, 2025--(BUSINESS WIRE)--Rallybio Corporation (Nasdaq: RLYB), a clinical-stage biotechnology company translating scientific advances into transformative therapies for patients with devastating rare diseases, today announced that it has entered into a definitive agreement to sell its interest in REV102, an ENPP1 inhibitor in preclinical development for the treatment of patients with hypophosphatasia (HPP), to joint venture partner Recursion Pharmaceuticals for up to $25 million, including an upfront equity payment of $7.5 million and near term milestones. With the upfront payment, Rallybio expects its cash runway to extend into mid-2027. The REV102 program originated from a joint venture between Rallybio and Recursion focused on the discovery and development of novel, orally available small molecule inhibitors of ENPP1 for the treatment of patients with HPP. The lead candidate from the joint venture, REV102, entered into IND-enabling studies in early 2025. "The Rallybio team has long been committed to targeting ENPP1 to address a significant unmet need in patients with HPP. By combining Rallybio's expertise in HPP preclinical and translational research with Recursion's integrated AI/experimental platform, we transformed this concept into the first potential oral disease-modifying treatment for HPP," said Stephen Uden, M.D., Chief Executive Officer of Rallybio. "We look forward to the advancement of REV102 through key milestones and, ultimately, to the delivery of this important treatment to patients in need." Dr. Uden added, "For Rallybio, divesting this preclinical asset enables us to extend our cash runway while sharpening our focus on strategically advancing our pipeline in ways that leverage our drug development expertise. We eagerly anticipate the release of topline data from our ongoing RLYB116 confirmatory PK/PD study this year." Under the terms of the agreement, Rallybio is eligible to receive certain payments, including $7.5 million in upfront equity, a contingent equity payment of $12.5 million upon the initiation of additional preclinical studies, and a $5 million milestone payment in connection with the initiation of dosing in a Phase 1 clinical study, as defined in the agreement. Rallybio is also eligible to receive low single-digit royalties on all future net sales by Recursion. In addition, Rallybio may be eligible to receive certain payments in the event of Recursion's sale of the REV102 program. "We extend our sincere thanks to Rallybio for their invaluable partnership in advancing this program to its current stage," said David Hallett, Chief Scientific Officer of Recursion. "Having full ownership of this important program allows Recursion to accelerate the development of the first potential oral disease-modifying treatment to HPP patients, who currently face significant challenges with limited access to existing therapies." 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 pipeline of promising product candidates aimed at addressing diseases with unmet medical need in areas of complement dysregulation and hematology. The Company's lead program, RLYB116, is a differentiated C5 inhibitor with the potential to treat diseases of complement dysregulation, with an initial focus on immune platelet transfusion refractoriness (PTR) and refractory antiphospholipid syndrome (APS). Rallybio's pipeline also includes RLYB332, a preclinical long-acting matriptase-2 antibody for the treatment of diseases of iron overload. Rallybio is headquartered in New Haven, Connecticut. For more information, please visit and follow us on LinkedIn. Forward-Looking Statements This press release contains forward-looking statements that are based on our management's beliefs and assumptions and 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 extension of Rallybio's runway, whether REV102 will advance to anticipated milestones, and whether the contingent consideration will be received. 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 conduct the RLYB116 PK/PD confirmatory study, and complete such study 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 the Quarterly Report on Form 10-Q for the period ended March 31, 2025, 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. View source version on Contacts Investor ContactsSamantha TracyRallybio Corporation(475) 47-RALLY (Ext. 282)investors@ Kevin LuiPrecision AQ(212) Media Contactmedia@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
08-05-2025
- Business
- Business Wire
Rallybio Reports First Quarter 2025 Financial Results and Provides Business Updates
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, today reported first quarter financial results for the period ended March 31, 2025, and provided an update on recent company developments. 'We are focused on advancing RLYB116 into a confirmatory pharmacokinetic/pharmacodynamic study this quarter, and believe that data from this study will demonstrate RLYB116's potential to be a best-in-class therapeutic for patients with complement-driven diseases,' said Stephen Uden, M.D., Chief Executive Officer of Rallybio. 'Leveraging our team's deep expertise in the complement field, we are well-positioned to maximize RLYB116's transformative potential across multiple billion-dollar market opportunities. Additionally, we believe significant value resides within our preclinical pipeline, which includes REV102 and RLYB332. By advancing our innovative pipeline, we are committed to creating exceptional value for our shareholders while delivering life-changing therapies to patients who need them most.' Recent Business Highlights and Upcoming Milestones: RLYB116 Program Rallybio is on track to initiate dosing in the RLYB116 confirmatory clinical pharmacokinetic/pharmacodynamic (PK/PD) study in the second quarter of 2025. Results from Cohort 1 (150 mg once weekly) and Cohort 2 (225 mg once weekly) are anticipated in the third and fourth quarter of 2025, respectively. Data from this study are expected to demonstrate complete and sustained complement inhibition with improved tolerability of RLYB116. REV102 Program Investigational new drug application (IND)-enabling studies are underway for REV102, an ENPP1 inhibitor for the treatment of patients with hypophosphatasia (HPP), which is being developed through a joint venture with Recursion Pharmaceuticals. These studies are expected to support the initiation of a Phase 1 study in the second half of 2026. Presentation of data evaluating REV102 in a preclinical model of later-onset HPP is expected in the second half of 2025. RLYB332 Program Rallybio continues to evaluate plans for future development of RLYB332, a long-acting, monoclonal anti-matriptase-2 antibody that has the potential to be a best-in-class treatment for diseases of iron overload. Preclinical data has demonstrated superior impact on PD parameters relative to comparator molecules, including serum iron, unsaturated iron binding capacity (UIBC), and transferrin saturation (TSAT). RLYB212 Program In April 2025, Rallybio announced the discontinuation of the RLYB212 program for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT). The Company's decision to discontinue RLYB212 development was based on PK data from the Phase 2 clinical trial demonstrating the inability of the RLYB212 dose regimen to achieve predicted target concentrations, as well as the minimum target concentration required for efficacy. Safety follow-up of the sentinel participant in the Phase 2 trial will continue as specified in the clinical trial protocol. Corporate Update Rallybio is implementing a 40% workforce reduction, representing nine positions, which will be substantially complete by the end of the second quarter of 2025. Rallybio estimates that the workforce reduction will result in aggregate charges of approximately $1.7 million, primarily for one-time employee severance and benefit costs, and excluding share-based compensation expense. First Quarter 2025 Financial Results Revenue: Revenue was $0.2 million for the first quarter of 2025. There was no revenue for the first quarter of 2024. The increase in revenue for the first quarter of 2025 was related to Rallybio's entrance into the collaboration agreement with Johnson & Johnson in the second quarter of 2024 and the recognition of revenue related to the collaboration's performance obligations. Research & Development (R&D) Expenses: R&D expenses were $5.7 million for the first quarter of 2025, compared to $12.9 million for the same period in 2024. The decrease in R&D expenses was primarily due to a decrease in development costs related to RLYB212, RLYB116, and other program candidates, in addition to a decrease in payroll and personnel-related costs, largely related to the workforce reduction in the first quarter of 2024. General & Administrative (G&A) Expenses: G&A expenses were $4.2 million for the first quarter of 2025, compared to $6.9 million for the same period in 2024. The decrease in G&A expenses was primarily due to a decrease in other general and administrative expenses in addition to lower payroll and personnel-related costs, largely related to the workforce reduction in the first quarter of 2024 and lower ongoing headcount in 2025 as compared to 2024. Net Loss and Net Loss Per Common Share: Rallybio reported a net loss of $9.4 million, or $0.21 per common share, for the first quarter of 2025 compared to a net loss of $19.0 million, or $0.47 per common share, for the same period in 2024. Cash Position: As of March 31, 2025, cash, cash equivalents, and marketable securities were $54.5 million. Rallybio expects these funds will be sufficient to support operations into the first half of 2027. 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 pipeline of promising product candidates aimed at addressing diseases with unmet medical need in areas of complement dysregulation, hematology, and metabolic disorders. The Company's lead program, RLYB116, is a differentiated C5 inhibitor with the potential to treat diseases of complement dysregulation. Rallybio also has two programs in preclinical development, including REV102, an ENPP1 inhibitor for the treatment of patients with hypophosphatasia (HPP), and RLYB332, a long-acting matriptase-2 antibody for the treatment of diseases of iron overload. Rallybio is headquartered in New Haven, Connecticut. For more information, please visit 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 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 initiating the RLYB116 confirmatory PK/PD study and the periods in which data is available, including data for Cohorts 1 and 2, whether the PK/PD confirmatory study will demonstrate improved tolerability and complete and sustained inhibition of terminal complement, whether RLYB116 will be effective in treating a broad range of complement-mediated diseases, the potential commercial opportunity for RLYB116, the potential value of Rallybio's pipeline, whether IND enabling studies for REV102 will support initiation of a Phase 1 study, the timing of initiation of a REV102 Phase 1 study, the timing of presenting data evaluating REV102 in a preclinical model of later-onset HPP, expected costs related to the workforce reduction and related charges, including the timing of such charges, and the Company's 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 trials, including the RLYB116 PK/PD confirmatory study, 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 Annual Report on Form 10-K for the period ended December 31, 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. Condensed Consolidated Balance Sheets (Unaudited) (in thousands) MARCH 31, 2025 DECEMBER 31, 2024 Cash, cash equivalents and marketable securities $ 54,495 $ 65,511 Total assets 57,983 68,108 Total liabilities 3,910 6,454 Total stockholders' equity 54,073 61,654 Expand
Yahoo
08-04-2025
- Business
- Yahoo
Rallybio to Discontinue Development of RLYB212 for Prevention of FNAIT
– RLYB212 Phase 2 PK Results Did Not Achieve Target Concentrations, Including Minimum Target Concentration Required for Efficacy – – RLYB116 Confirmatory PK/PD Study to Initiate in 2Q 2025, with Data in 2H 2025 – NEW HAVEN, Conn., April 08, 2025--(BUSINESS WIRE)--Rallybio Corporation (Nasdaq: RLYB), a clinical-stage biotechnology company translating scientific advances into transformative therapies for patients with devastating rare diseases, today announced the discontinuation of the RLYB212 program for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT). The Company's decision to discontinue RLYB212 development was based on pharmacokinetic (PK) data from the Phase 2 clinical trial demonstrating the inability of the RLYB212 dose regimen to achieve predicted target concentrations, as well as the minimum target concentration required for efficacy. Rallybio remains focused on advancing RLYB116, a once-weekly low volume C5 inhibitor for the treatment of complement-driven diseases, as well as its emerging preclinical programs. "We are disappointed by the PK results of the RLYB212 Phase 2 trial," said Stephen Uden, M.D., Chief Executive Officer of Rallybio. "Given that the results significantly deviated from the predicted range and the absence of empiric data to further inform dose adjustment, the risk/benefit no longer supports continued dosing, and we will discontinue RLYB212 development. We are grateful to the participants, investigators, and study staff for their partnership and dedication to this program." Dr. Uden continued, "Rallybio remains steadfast in our mission to develop transformative therapies. We are focused on creating shareholder value by advancing our portfolio of potentially best-in-class assets for patients with rare diseases, which includes RLYB116 and REV102, an ENPP1 inhibitor for patients with hypophosphatasia, as well as RLYB332, a long-acting matriptase-2 antibody for diseases of iron overload." RLYB212 Phase 2 Trial The single-arm Phase 2 dose confirmation trial was designed to assess the PK and safety of RLYB212 in pregnant women at higher risk for HPA-1a alloimmunization and FNAIT. Secondary objectives included the assessment of pregnancy and neonatal/infant outcomes, and the occurrence of emergent HPA-1a alloimmunization. Second trimester PK results from the sentinel participant demonstrated an inability of RLYB212 to achieve predicted target concentrations of 6 ng/mL to 10 ng/mL, as well as the minimum target concentration required for efficacy of 3 ng/mL, with values near or below the assay's lower limit of quantitation. Dose adjustment is not deemed feasible given that PK levels are meaningfully outside the predicted range and the absence of empiric data to inform an adjustment. It is hypothesized that HPA-1a antigen expression on the placenta may be impacting plasma concentrations of RLYB212. No further enrollment in the trial is planned and all screening of participants has been stopped. The Company will continue safety follow-up of the sentinel participant as specified in the clinical trial protocol. Promising Rare Disease Pipeline to Deliver Data in 2H 2025 RLYB116 Program Rallybio remains on track to initiate dosing in the RLYB116 confirmatory clinical pharmacokinetic/ pharmacodynamic (PK/PD) study in the second quarter of 2025, with data readouts from Cohorts 1 and 2 expected in the third and fourth quarter of 2025, respectively. The study is expected to demonstrate complete and sustained complement inhibition with improved tolerability of RLYB116. RLYB116 is a novel antibody mimetic fusion protein designed to inhibit C5 and to provide a once-weekly, small volume, subcutaneously injected therapy to meet patient demand for a convenient, self-administered at-home solution. RLYB116 has the potential to address significant unmet need for patients across a number of complement mediated diseases, including paroxysmal nocturnal hemoglobinuria (PNH), antiphospholipid syndrome (APS), and generalized myasthenia gravis (gMG), which together represent a commercial opportunity of more than $6 billion. Importantly, this strategic focus on complement-mediated diseases presents a unique opportunity for Rallybio to utilize its team's deep domain experience in successfully designing, developing, and delivering complement inhibitors for patients with rare diseases. Preclinical Programs REV102, an ENPP1 inhibitor for the treatment of patients with HPP under development through a joint venture with Recursion Pharmaceuticals, entered investigational new drug application (IND)-enabling studies in the first quarter of 2025 to support the initiation of a Phase 1 study in 2026. Data evaluating REV102 in a preclinical model of later-onset HPP is expected in the second half of 2025. Rallybio's portfolio also includes RLYB332, a long-acting, monoclonal anti-matriptase-2 antibody that has the potential to be a best-in-class treatment for diseases of iron overload. Preclinical data has demonstrated superior impact on PD parameters relative to comparator molecules, including on serum iron, unsaturated iron binding capacity (UIBC), and transferrin saturation (TSAT). The Company is preparing plans for future development of RLYB332. About RallybioRallybio (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 pipeline of promising product candidates aimed at addressing diseases with unmet medical need in areas of complement dysregulation, hematology, and metabolic disorders. The Company's lead program, RLYB116, is a differentiated C5 inhibitor with the potential to treat diseases of complement dysregulation. Rallybio also has two programs in preclinical development, including REV102, an ENPP1 inhibitor for the treatment of patients with hypophosphatasia (HPP), and RLYB332, a long-acting matriptase-2 antibody for the treatment of diseases of iron overload. Rallybio is headquartered in New Haven, Connecticut. For more information, please visit and follow us on LinkedIn and Twitter. Forward-Looking StatementsThis press release contains forward-looking statements that are based on our management's beliefs and assumptions and 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 initiating the RLYB116 confirmatory PK/PD study and the date when data is available, including data for Cohorts 1 and 2, whether the PK/PD confirmatory study will demonstrate improved tolerability and sustained inhibition of terminal complement, whether RLYB116 will be effective in treating a broad range of complement-mediated diseases, the potential commercial opportunity for RLYB116, and the timing of initiation of a REV102 Phase 1 study. 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 RLYB116 PK/PD confirmatory study, 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, 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 Annual Report on Form 10-K for the period ended December 31, 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. View source version on Contacts Investor ContactsSamantha TracyRallybio Corporation(475) 47-RALLY (Ext. 282)investors@ Kevin LuiPrecision AQ(212) Media Contactmedia@ Sign in to access your portfolio
