Financial Performance - The net loss for Q1 2025 was $5.5 million, compared to a net loss of $7.1 million in Q1 2024, representing a 22.5% improvement [8]. - The basic and diluted net loss per share for Q1 2025 was $0.25, down from $0.86 in Q1 2024 [8]. - Net loss for the three months ended March 31, 2025, was $5,501,000, an improvement from a net loss of $7,113,000 in the same period of 2024 [14]. - Net cash used in operating activities increased to $6,175,000 in Q1 2025, compared to $5,271,000 in Q1 2024 [14]. - Stock-based compensation expense rose to $264,000 in Q1 2025, compared to $150,000 in Q1 2024 [14]. Cash Position - Cash and cash equivalents as of March 31, 2025, were $7.4 million, down from $12.9 million as of December 31, 2024 [8]. - Cash, cash equivalents, and restricted cash at the end of the period totaled $7,428,000, down from $12,067,000 at the end of Q1 2024 [14]. - The company reported a net decrease in cash, cash equivalents, and restricted cash of $5,437,000 during the period [14]. - Cash and cash equivalents at the end of the period were $7,428,000, compared to $12,042,000 in Q1 2024 [14]. Expenses - Research and Development (R&D) expenses for Q1 2025 were $3.1 million, a decrease of 11.6% from $3.5 million in Q1 2024 [8]. - General and Administrative (G&A) expenses for Q1 2025 were $2.5 million, down 33.3% from $3.7 million in Q1 2024 [8]. - Accounts payable decreased to $238,000 in Q1 2025, down from $966,000 in Q1 2024 [14]. - Other assets showed a significant decrease, with a net change of $(764,000) in Q1 2025 compared to an increase of $1,301,000 in Q1 2024 [14]. Corporate Developments - The RENEW Phase 2 trial of LTI-03 in idiopathic pulmonary fibrosis (IPF) has been initiated, with approximately 120 patients expected to be enrolled in two cohorts [3]. - The company plans to share topline interim data from the RENEW trial in the first half of 2026 [2]. - Rein completed warrant exercise and private placement transactions in April 2025, raising approximately $5.28 million [5]. - Two patents covering the novel formulation and administration methods of LTI-03 were granted on April 22, 2025 [5]. - Rein's common stock began trading under the Nasdaq ticker symbol "RNTX" on January 13, 2025, following a rebranding [5]. Stock Transactions - Proceeds from the issuance of common stock, net of offering costs, amounted to $737,000 in Q1 2025 [14]. - There was a conversion of Series X non-voting convertible preferred stock into common stock shares valued at $44,826,000 in the previous period [14].

Financial Performance - The company reported net losses of $5.5 million for Q1 2025, compared to $7.1 million for Q1 2024, with an accumulated deficit of $356.9 million as of March 31, 2025[145]. - The net loss for the three months ended March 31, 2025 was $5.5 million, an improvement of 22.7% compared to a net loss of $7.1 million in the same period of 2024[165]. - During the three months ended March 31, 2025, net cash used in operating activities was $6.2 million, compared to $5.3 million in the same period of 2024, indicating an increase of 17.0%[182]. Cash Position - As of March 31, 2025, the company had cash and cash equivalents of $7.4 million, which is expected to fund operations only until September 2025[147]. - As of March 31, 2025, the company had cash and cash equivalents of $7.4 million, which is expected to fund operations into September 2025[172]. - The company raised $1.7 million from the exercise of PIPE Warrants and Offering Warrants on April 21, 2025[174]. Research and Development - The company initiated the RENEW Phase 2 clinical trial of LTI-03 in May 2025, aiming to enroll approximately 120 patients across up to 50 sites globally[141]. - The RENEW trial will assess the safety and efficacy of LTI-03, with a primary endpoint focused on treatment-emergent adverse events over 24 weeks[142]. - The company plans to continue its research and development of LTI-03 and LTI-01, expecting substantial increases in expenses related to ongoing activities[184]. Operating Expenses - For the three months ended March 31, 2025, total operating expenses decreased to $5.6 million from $7.2 million in the same period of 2024, representing a reduction of approximately 22.1%[165]. - Research and development expenses for the three months ended March 31, 2025 were $3.1 million, down from $3.5 million in the same period of 2024, a decrease of 11.4% primarily due to delays in clinical development[166]. - General and administrative expenses decreased to $2.5 million for the three months ended March 31, 2025, compared to $3.7 million in 2024, reflecting a reduction of 32.4% mainly from decreased professional fees[169]. Financing Activities - The company has financed operations through $145.5 million in net proceeds from common stock sales and $17.7 million from a public offering in May 2024[144]. - The company completed a follow-on public offering in May 2024, raising net proceeds of $17.7 million from the sale of 4,273,505 shares[154]. - The company entered into an "at the market offering" agreement with H.C. Wainwright for an aggregate offering price of up to $13.7 million[148]. - The company anticipates needing additional capital to fund operations, which may be obtained through equity or debt financings, strategic collaborations, or licensing arrangements[171]. Accounting and Compliance - No material changes to critical accounting estimates were reported for the three months ended March 31, 2025[192]. - Recent accounting standards reviewed will not have a material impact on the condensed consolidated financial statements[193]. - The company qualifies as a smaller reporting company and is not required to provide certain market risk disclosures[194]. Future Outlook - The company anticipates continued operating losses due to ongoing research and development activities[146]. - The company has a total potential obligation of approximately $16.9 million under a master services agreement with a third-party CRO for clinical research[189]. - The company has not generated any revenue from product sales and does not expect to do so in the foreseeable future[155].

Summary of RAIN Therapeutics Conference Call Company Overview - RAIN Therapeutics, following a reverse merger with Aleron Therapeutics in October 2023, is focused on developing first-in-class treatments for orphan pulmonary conditions, particularly fibrosis indications [2][4]. Key Products and Clinical Trials - **Lead Asset: LTI-03** - Targeting idiopathic pulmonary fibrosis (IPF), currently moving into a Phase 2 trial [3]. - Demonstrated safety and tolerability in Phase 1 studies, with promising biomarker results [3][24]. - Unique mechanism with both anti-fibrotic and regenerative functions [3][16]. - **Second Asset: LTI-01** - Aimed at treating loculated pleural effusion, a condition affecting approximately 60,000 people in the US annually [4]. - No current FDA-approved pharmacologic solutions exist for this condition [4][33]. Market Potential - The global market for IPF treatments is estimated to reach nearly $12 billion by 2031, with current leading drugs (e.g., Nintedanib) generating around $3.6 billion in 2023 [11][12]. - LTI-01 has a potential market of $400 million in the US and Europe, based on pricing comparable to off-label drugs [33]. Mechanism of Action - LTI-03 operates through a peptide that mimics the Cav1 protein, which is downregulated in fibrotic states [15][37]. - The drug is delivered via dry powder inhalation, targeting lung tissue directly [16][24]. Clinical Study Insights - Phase 1b study showed significant reductions in key biomarker proteins associated with IPF, indicating effective drug delivery to fibrotic lung tissue [25][26]. - The upcoming Phase 2 trial will assess lung function metrics (FVC, FEV1) over 24 weeks, with expectations of positive outcomes based on previous studies [30][31]. Competitive Landscape - Current approved treatments for IPF only slow disease progression and are poorly tolerated, highlighting a significant unmet need in the market [10][12]. - RAIN Therapeutics aims to differentiate itself by offering a safer, more effective treatment option with fewer side effects [18][46]. Future Outlook - Anticipation of Phase 2 trial initiation and data release within the next year, with hopes of demonstrating significant lung function improvements [29][51]. - Ongoing partnership with Taiho Pharmaceutical for commercialization rights in Japan for LTI-01 [33]. Additional Considerations - The regenerative potential of LTI-03 could lead to greater benefits in lung function compared to existing therapies, which primarily focus on slowing decline [44][46]. - The company is optimistic about enrollment for the upcoming trials, aiming for a global reach to capture a diverse patient population [50].

Financial Performance - The company has incurred net losses of $62.9 million and $15.7 million for the years ended December 31, 2024 and 2023, respectively, with an accumulated deficit of $351.4 million as of December 31, 2024[559]. - The net loss for the year ended December 31, 2024, was $62.9 million, compared to a net loss of $15.7 million in 2023, reflecting an increase in losses of $47.2 million[583]. - Cash used in operating activities was $22.3 million in 2024, compared to $19.8 million in 2023, indicating a higher cash burn rate[602]. - Other income for 2024 was $0.7 million, up from $0.5 million in 2023, primarily due to interest income and investment accretion[589]. Cash and Financing - As of December 31, 2024, the company had cash and cash equivalents of $12.9 million, which is expected to fund operations into August 2025, but is insufficient for completing the planned Phase 2 clinical trial of LTI-03[560]. - The company has financed operations primarily through $145.5 million in net proceeds from sales of common stock and warrants, and $17.7 million from a public offering completed in May 2024[558][565]. - Net cash provided by financing activities for the year ended December 31, 2024, was $17.8 million, an increase from $15.8 million in 2023 due to PIPE Financing[607]. - The company anticipates needing to raise additional capital through public or private equity offerings, debt financings, or collaborations[612]. Research and Development - The company has two product candidates in clinical development: LTI-03 for Idiopathic Pulmonary Fibrosis and LTI-01 for loculated pleural effusion, with LTI-01's clinical development temporarily delayed[556][561]. - Research and development expenses primarily consist of costs related to clinical trials, personnel, and compliance with regulatory requirements[572][576]. - Research and development expenses increased to $14.2 million in 2024 from $4.0 million in 2023, a rise of $10.3 million, primarily due to new clinical programs acquired in the Lung Acquisition[584]. - The company anticipates continued significant expenses and operating losses as it advances clinical development of lead product candidates LTI-03 and LTI-01[594]. - The company plans to continue research and development of LTI-03 and LTI-01, expecting substantial increases in expenses related to ongoing activities[608]. Impairment and Losses - The impairment loss on intangible assets was identified in the fourth quarter of 2024 when the carrying value of LTI-01 exceeded its fair value[577]. - The company incurred an impairment loss on intangible assets of $37.0 million in 2024, linked to the delay in clinical development of LTI-01[587]. - An impairment loss of $37.0 million was recognized for the LTI-01 intangible asset due to a temporary delay in clinical development[626]. General and Administrative Expenses - General and administrative expenses rose to $13.9 million in 2024 from $11.4 million in 2023, an increase of $2.5 million, mainly driven by higher employee-related expenses and costs associated with the Lung Acquisition[586]. Future Outlook - The company anticipates continued operating losses for the foreseeable future due to ongoing research and development costs[559]. - Future capital requirements will depend on various factors, including clinical trial costs and regulatory approval processes[610]. Other Information - The company has federal net operating loss carryforwards of $77.3 million and federal research and development tax credit carryforwards of $2.4 million as of December 31, 2024[590]. - The company does not have any off-balance sheet arrangements as of December 31, 2024, and 2023[614]. - The company is classified as a "smaller reporting company," which may allow it to rely on exemptions from certain disclosure requirements[635]. - Management continues to monitor estimates related to financial conditions and will adjust them as necessary based on changing economic conditions[627].

Core Viewpoint - Rein Therapeutics has rebranded from Aileron Therapeutics to emphasize its commitment to developing therapies for orphan pulmonary and fibrosis indications, with shares set to trade under the new Nasdaq symbol "RNTX" starting January 13, 2025 [1][2] Recent Clinical Achievements - The lead candidate, LTI-03, has shown positive topline data from a Phase 1b clinical trial, indicating safety and tolerability in patients with idiopathic pulmonary fibrosis (IPF), with four biomarkers demonstrating statistical significance [3] - High dose LTI-03 (5 mg BID) maintained a favorable safety profile, while five biomarkers showed dose dependence in the trial [3] Corporate Highlights - In March 2024, Brian Windsor was appointed as CEO, reinforcing the company's focus on first-in-class therapies for orphan pulmonary and fibrosis indications [6] - In May 2024, the company raised approximately $17.7 million through an underwritten registered direct offering of common stock and accompanying warrants [6] Strategic Objectives - A Phase 2 clinical trial for LTI-03 is anticipated to begin in the first half of 2025, targeting idiopathic pulmonary fibrosis [5][6] - The second product candidate, LTI-01, has completed Phase 1b and Phase 2a trials for loculated pleural effusions and has received Orphan Drug Designation and Fast Track Designation in the U.S. [5][6]

Core Insights - Aileron Therapeutics announced promising safety and positive biomarker data from Cohort 2 of the Phase 1b clinical trial of LTI-03 in idiopathic pulmonary fibrosis (IPF) patients, demonstrating dose-dependent effects in five evaluated biomarkers compared to low-dose LTI-03 [1][4] - The combined data set from Cohorts 1 and 2 achieved statistical significance in four out of eight biomarkers evaluated, reinforcing the potential of LTI-03 to improve lung function and reverse disease progression [2][4] - The company is planning a Phase 2 clinical trial following the positive results from the ongoing studies [1][4] Corporate Updates - In October 2024, Aileron entered into an exclusive option agreement with Advancium Health Network for the acquisition of ALRN-6924, a clinical-stage oncology agent, which includes a non-refundable fee and potential milestone payments [3] - The company reported a cash position of $17.7 million as of September 30, 2024, down from $21.9 million as of June 30, 2024, and expects this to fund operations into June 2025 [5] Financial Results - Research and Development (R&D) expenses for Q3 2024 were $3.7 million, a significant increase from less than $0.1 million in Q3 2023, primarily due to clinical programs acquired from the Lung Acquisition [5] - General and Administrative (G&A) expenses for Q3 2024 were $2.3 million, up from $2.0 million in Q3 2023, attributed to increased employee-related expenses and facilities costs [6] - The net loss for Q3 2024 was $5.8 million, compared to $1.8 million in Q3 2023, with a basic and diluted net loss per share of $0.27 [6][10] Pipeline Developments - Aileron's lead product candidate, LTI-03, is a novel synthetic peptide targeting alveolar epithelial cell survival and inhibiting profibrotic signaling, having completed a Phase 1b clinical trial for IPF [7] - The company also has a second product candidate, LTI-01, which has completed Phase 1b and Phase 2a clinical trials for loculated pleural effusions and has received Orphan Drug Designation in the US and EU [7]

Financial Performance - Aileron Therapeutics reported a net loss of $5.8 million for Q3 2024, compared to a net loss of $1.8 million in Q3 2023, with a net loss per share of $0.27 compared to $0.40 in the prior year[6]. - Total operating expenses for Q3 2024 were $6.1 million, compared to $2.0 million in Q3 2023, reflecting increased R&D and G&A expenses[10]. - General and Administrative (G&A) expenses for Q3 2024 were $2.3 million, up from $2.0 million in Q3 2023, primarily due to increased employee-related expenses[6]. - Aileron's accumulated deficit increased to $310.4 million as of September 30, 2024, compared to $288.5 million at the end of 2023[9]. Research and Development - Research and Development (R&D) expenses for Q3 2024 were $3.7 million, a significant increase from less than $0.1 million in Q3 2023, primarily due to clinical programs acquired from the Lung Acquisition[6]. - Aileron announced positive data from Cohort 2 of the Phase 1b clinical trial of LTI-03, showing dose-dependent effects in five biomarkers, with four biomarkers achieving statistical significance when combined with Cohort 1 data[2][4]. - Aileron plans to initiate a Phase 2 clinical trial for LTI-03, building on the promising results from the Phase 1b trial[1][8]. Cash and Assets - Cash and cash equivalents as of September 30, 2024, were $17.7 million, down from $21.9 million as of June 30, 2024, with expectations to fund operations into June 2025[6]. - The company reported total assets of $104.2 million as of September 30, 2024, down from $106.0 million at the end of 2023[9]. Strategic Agreements - The company entered into an exclusive option agreement with Advancium Health Network for the acquisition of ALRN-6924, which includes potential milestone payments and sales royalties[3].

Clinical Development - Company is focused on developing novel therapies for orphan pulmonary and fibrosis indications, with two product candidates in clinical development: LTI-03 and LTI-01 [71] - LTI-03 demonstrated statistically significant reductions in profibrotic proteins in Cohort 1 of the Phase 1b trial, with GAL-7 (p=0.0014), TSLP (p=0.0223), and Col-1α1 (p=0.0489) showing significant decreases [73] - In Cohort 2 of the Phase 1b trial, LTI-03 showed dose-dependent trends in five biomarkers, including COL1A1 and CXCL7, indicating active pharmacodynamics [73] - Company plans to initiate a Phase 2 clinical trial for LTI-03 following positive results from the Phase 1b trial [73] Financial Performance - Revenue for the three months ended September 30, 2024 was $0, with a net loss of $5.8 million, compared to a net loss of $1.8 million for the same period in 2023, representing an increase in loss of $4.0 million [85] - Research and development expenses for the three months ended September 30, 2024 were $3.7 million, a significant increase from less than $0.1 million in the same period in 2023, primarily due to clinical programs acquired in the Lung Acquisition [86] - General and administrative expenses increased to $2.3 million for the three months ended September 30, 2024, up from $2.0 million in the same period in 2023, driven by increased employee-related expenses [87] - Total operating expenses for the three months ended September 30, 2024 were $6.1 million, compared to $2.0 million for the same period in 2023, reflecting an increase of $4.1 million [85] - For the nine months ended September 30, 2024, research and development expenses were $10.9 million, compared to $2.0 million for the same period in 2023, an increase of $8.9 million due to the Lung Acquisition [92] - General and administrative expenses for the nine months ended September 30, 2024 were $11.4 million, up from $6.0 million in the same period in 2023, reflecting an increase of $5.4 million [93] - The net cash used in operating activities for the nine months ended September 30, 2024 was $17.5 million, primarily due to a net loss of $21.9 million [98] - Cash used in operating activities for the nine months ended September 30, 2024, was $(17.5) million, compared to $(9.4) million for the same period in 2023 [100] Cash Position and Funding Needs - As of September 30, 2024, the company reported cash and cash equivalents of $17.7 million, which may not be sufficient to fund operations for the next twelve months, raising concerns about going concern [76] - The company expects to continue incurring significant expenses and operating losses as it advances clinical development of its lead product candidates, LTI-03 and LTI-01 [96] - The company anticipates needing additional capital to fund operations, which may be obtained through equity or debt financings, strategic collaborations, or licensing arrangements [96] - Net cash provided by financing activities for the nine months ended September 30, 2024, was $17.8 million, primarily due to an offering in May 2024, while there were no financing activities in the same period of 2023 [101] - As of September 30, 2024, the company had cash and cash equivalents of $17.7 million, which is expected to fund operations into June 2025 [102] - The company may need to obtain substantial additional funding to support ongoing operations and product development, with potential impacts from global economic conditions [104] - The company anticipates that additional financing may be required, which could lead to dilution for existing stockholders or increased fixed payment obligations [104] Acquisition and Strategic Focus - Company acquired Lung Therapeutics, Inc. on October 31, 2023, issuing 344,345 shares of common stock and 19,903 shares of Series X non-voting convertible preferred stock [74] - Following the Lung Acquisition, the company shifted its focus to developing first-in-class medicines for significant unmet medical needs in orphan pulmonary and fibrosis indications [74] - The company is focused on expanding its research pipeline and internal R&D capabilities, which may require significant capital investment [102] Accounting and Reporting - There were no material changes to critical accounting estimates during the three and nine months ended September 30, 2024, compared to the previous fiscal year [105] - The company is classified as a "smaller reporting company," which allows it to rely on certain exemptions from disclosure requirements [107] - The company has no cash provided by investing activities for the nine months ended September 30, 2024, compared to $16.3 million in the same period of 2023 [101]

Core Insights - Aileron Therapeutics announced positive topline data from Cohort 2 of its Phase 1b clinical trial for LTI-03, indicating potential therapeutic effects in idiopathic pulmonary fibrosis (IPF) patients [1][2][3] Group 1: Clinical Trial Results - High-dose LTI-03 (5 mg BID) reduced expression of multiple profibrotic proteins in both pathological basal-like cells and fibroblasts, suggesting a therapeutic effect [1][4] - A positive trend was observed in seven out of eight IPF biomarkers, with four biomarkers showing statistically significant changes in the combined data set of Cohort 1 and Cohort 2 [2][4] - Five biomarkers exhibited dose-dependent movement compared to low-dose LTI-03, indicating active pharmacodynamics [1][5] Group 2: Safety and Tolerability - High-dose LTI-03 was well-tolerated, with no safety signals or drug-related adverse events leading to trial discontinuation [1][5] - LTI-03 did not induce inflammation in peripheral blood mononuclear cells, measured by pAKT, a safety marker for inflammation [5] Group 3: Future Plans - The company is planning a Phase 2 clinical trial to further evaluate LTI-03's potential in treating IPF [1][3] - Aileron will host a conference call to discuss the topline results from Cohort 2 of the Phase 1b clinical trial [6] Group 4: Background Information - LTI-03 is a novel, Caveolin-1-related peptide designed to address significant unmet medical needs in orphan pulmonary and fibrosis indications [1][10] - IPF is a chronic lung disease characterized by progressive tissue scarring, affecting approximately 100,000 people in the United States [8]

Core Insights - Advancium Health Network has entered into an exclusive option agreement with Aileron Therapeutics for the acquisition of ALRN-6924, a clinical-stage oncology agent aimed at treating retinoblastoma, a rare pediatric eye cancer [1][3][4] - ALRN-6924 selectively targets MDM2 and MDMX to activate p53-mediated tumor suppression, representing a novel approach compared to traditional chemotherapy [2][4] - The agreement marks Advancium's first drug opportunity in its mission to develop therapies for children with cancer, highlighting the collaboration's significance for both organizations [4] Company Overview - Aileron Therapeutics is focused on developing first-in-class medicines for unmet medical needs in orphan pulmonary and fibrosis indications, with its lead product candidate being LTI-03 [6][7] - Advancium Health Network is a public charity that combines for-profit infrastructure with non-profit ideals to address healthcare barriers for underserved populations [8] Market Context - Retinoblastoma affects approximately 300 cases annually in the U.S. and 9,000 cases worldwide, predominantly in children, indicating a significant unmet need for effective treatments [2] - The traditional treatment for retinoblastoma often involves non-selective chemotherapy and may result in the removal of affected eye(s), underscoring the potential impact of ALRN-6924 as a targeted therapy [2]