Actinium Pharmaceuticals(ATNM) - 2023 Q2 - Quarterly Report

Drug Development and Clinical Trials - Actinium Pharmaceuticals aims to launch two radiotherapy drugs, Iomab-B and Actimab-A, over the next several years to improve outcomes for patients with relapsed or refractory acute myeloid leukemia (r/r AML) [77] - Iomab-B met the primary endpoint of durable Complete Remission (dCR) with a statistical significance of p<0.0001 in the Phase 3 SIERRA trial, demonstrating double the 1-year and median overall survival compared to control arm patients [81] - Actimab-A has shown a survival benefit of 53% and 32% at one and two years in patients typically expected to live two to four months, validating its therapeutic approach [86] - Actinium is working towards submitting a Biologics License Application (BLA) for Iomab-B to the FDA by the end of 2023, aiming for commercialization in the U.S. [83] - The SIERRA trial for Iomab-B demonstrated unprecedented access to bone marrow transplants (BMT) and improved outcomes, establishing operations at 24 leading BMT centers in the U.S. and Canada, representing about 30% of transplant volume [100] - Actimab-A showed a median overall survival (OS) of 59% at one year and 32% at two years in patients who had failed venetoclax treatment, indicating its potential as a backbone therapy for r/r AML [102] - Iomab-B is positioned to become the standard of care for r/r AML patients not considered transplantable, with a Biologics License Application (BLA) expected to be filed in the second half of 2023 [100] - Actimab-A + CLAG-M combination therapy showed a 1-year survival rate of 59% in patients who failed prior venetoclax-based therapy, significantly improving traditional outcomes [146] - Actimab-A + venetoclax combination trial has shown promising results with complete response (CR) and partial response in early cohorts, and proof-of-concept data is expected in the second half of 2023 [147] - Iomab-B achieved a 75% initial remission rate at 30 days post-BMT compared to 6.3% in the control arm, with a p-value of <0.0001 [123] - 22% of patients receiving Iomab-B maintained durable complete remission (dCR) for 6 months or more, while none in the control arm achieved dCR [123] - The 1-year overall survival (OS) rate for patients achieving 6-month dCR was 92.3%, and the 2-year OS rate was 59.9% [126] - Median OS for Iomab-B patients was 6.4 months, double the 3.2 months for non-crossover control patients, and 7.1 months for crossover patients [129] - Iomab-B demonstrated a 78% reduction in the probability of an event in event-free survival (EFS), with EFS at 6 months being 28% for Iomab-B versus 0.2% for the control arm [130] - The incidence of sepsis was significantly lower in the Iomab-B arm at 6.1% compared to 28.6% in the control arm [134] Market Opportunity and Strategy - The market opportunity for Iomab-B and Actimab-A in AML is significant, with approximately 21,000 new cases annually in the U.S. and over 50% of patients developing relapsed or refractory disease [92] - Currently, less than 20% of AML patients can access a bone marrow transplant (BMT), but Iomab-B could potentially expand this market from 400 r/r AML patients currently transplanted to approximately 8,000 unfit patients [93] - The company plans to expand the Iomab-B label into five additional hematologic indications, including MDS, ALL, HL, NHL, and MM, to increase market potential [103] - Iomab-B is being expanded to address over 165,000 patients diagnosed with hematologic cancers who could benefit from transplant but currently lack access [152] - Actinium's strategy focuses on building a specialty radiotherapeutics company targeting the top 100 cancer hospitals, emphasizing treatment for relapsed or refractory (r/r) diseases with high unmet medical needs [96] Financial Overview - Actinium has approximately $91.2 million in cash on hand as of June 30, 2023, expected to fund operations through 2025 [90] - Total revenue for the three months ended June 30, 2023 was $0, compared to $45 thousand for the same period in 2022 [172] - Research and development expenses for the three months ended June 30, 2023 were $11.1 million, an increase of $6.4 million from $4.7 million in the same period of 2022 [178] - General and administrative expenses for the three months ended June 30, 2023 were $4.6 million, up $1.4 million from $3.2 million in the same period of 2022 [179] - Net loss for the three months ended June 30, 2023 was $15.2 million, an increase of $7.4 million from $7.8 million in the same period of 2022 [181] - Total operating expenses for the six months ended June 30, 2023 were $27.2 million, compared to $14.0 million for the same period in 2022 [184] - Cash used in operating activities for the six months ended June 30, 2023 was $28.7 million, an increase of $50.8 million from $22.1 million in the prior-year period [194] - Net cash provided by financing activities for the six months ended June 30, 2023 was $10.8 million, compared to $16.6 million for the same period in 2022 [195] - Research and development expenses for the six months ended June 30, 2023 were $18.9 million, an increase of $9.9 million from $9.0 million in the same period of 2022 [188] - Long-term license revenue deferred was $35.0 million at June 30, 2023, resulting from the receipt from Immedica, to be recognized upon regulatory approval of Iomab-B [177] Intellectual Property and Partnerships - Actinium's intellectual property portfolio includes over 200 issued patents and pending applications worldwide, supporting its innovative pipeline [90] - The company has over 200 issued and pending patent applications worldwide, ensuring strong intellectual property protection for its products, including Iomab-B until 2037 [105] - The company has a patent portfolio of over 200 issued patents and pending applications, with effective lives ranging from 2024 to 2043, supporting its proprietary technology [168] - A CRADA with the NCI was established to accelerate Actimab-A development, providing access to over 2,000 clinical trial sites and support for various combination therapies [149] - Actinium has licensed EUMENA commercial rights for Iomab-B to Immedica, receiving an upfront payment of $35 million and potential additional milestones of $417 million [140] Research and Development Focus - The company is exploring the use of Actimab-A in solid tumor indications, with promising preclinical data presented at the AACR Annual Meeting [87] - Actinium aims to leverage its R&D capabilities and partnerships to advance solid tumor programs and expand its commercial footprint into the top 100 hospitals [106] - Actinium's HER3-targeted radiotherapy showed significant tumor growth suppression (p<0.0001) in preclinical models of ovarian and colorectal cancers, highlighting its therapeutic potential [160] - The company is leveraging its technology platform to develop novel radiotherapeutic assets, with promising preclinical results in various tumor models [158] - Iomab-ACT is being developed for use in conditioning prior to CAR-T and gene therapies, with a clinical trial funded by the NIH expected to present proof-of-concept data in the second half of 2023 [104] - Iomab-ACT is being developed for conditioning prior to CAR-T therapies, with initial cohorts showing promise in reducing common toxicities associated with CAR-T treatments [156] Economic and Market Conditions - As of June 30, 2023, the company's cash equivalents primarily consisted of short-term money market funds, with no significant market risk related to interest rates [219] - An immediate 10% change in interest rates would not materially affect the fair market value of the company's financial position or results of operations [219] - Inflation has not had a material effect on the company's business or financial condition during the six months ended June 30, 2023 [220]