On August 14, 2024 Aileron Therapeutics, Inc. ("Aileron" or the "Company") (NASDAQ: ALRN), a biopharmaceutical company advancing a pipeline of potential first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, reported financial results for the second quarter ended June 30, 2024, and provided a business update (Press release, Aileron Therapeutics, AUG 14, 2024, View Source [SID1234645885]).

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"Throughout the first half of the year, we focused on strengthening our balance sheet and advancing the development of inhaled LTI-03 in IPF," said Brian Windsor, Ph.D., President and Chief Executive Officer of Aileron. "We are extremely pleased with the positive data from Cohort 1 of the ongoing Phase 1b clinical trial announced in May, particularly the achievement of statistical significance in three out of eight biomarkers which is a testament to the potential of LTI-03 to inhibit fibrosis and improve lung function. Additionally, in May, we raised approximately $18.2 million in net proceeds in an underwritten registered direct offering, which provides us with the resources to further validate LTI-03 in the ongoing Phase 1b trial. We look forward to reporting topline results from the high-dose cohort in the third quarter of this year."

Second Quarter 2024 Highlights and Recent Updates

Corporate Updates

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In May 2024, the Company completed an underwritten registered direct offering of 4,273,505 shares of its common stock and accompanying warrants to purchase an aggregate of 4,273,505 shares of its common stock. Net proceeds from the offering were approximately $18.2 million, after deducting underwriting discounts and commissions and other offering expenses. The Company has the potential to receive approximately $20.0 million in additional proceeds from the exercise of the warrants issued in the offering.

Pipeline

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Announced positive Cohort 1 data from the ongoing Phase 1b clinical trial evaluating the safety and tolerability of inhaled LTI-03 in patients diagnosed with IPF.

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Following inhaled administration of low dose LTI-03 (2.5 mg BID, or twice daily) in twelve patients, a positive trend was observed in seven out of eight biomarkers. The findings included:

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Evidence of LTI-03 reducing expression of multiple profibrotic proteins produced in both basal-like cells and fibroblasts that contribute to the progression of IPF, with statistically significant decreases in three biomarkers, reinforcing the potential of LTI-03 to inhibit fibrosis, inflammation and associated changes in the lungs.

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LTI-03 stimulated production of solRAGE, a factor indicative of type I epithelial cell health, a critically important aspect of IPF that has gone largely unaddressed.

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LTI-03 was generally well-tolerated with no serious adverse events ("SAEs") reported.

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The Phase 1b trial is ongoing, with topline results from the high-dose Cohort 2 expected in the third quarter of 2024.

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On May 1, 2024, the Company hosted a pulmonary care expert call to discuss the Cohort 1 Phase 1b results of LTI-03, featuring pulmonary care expert Andreas Günther, M.D., Head of the Center for Interstitial and Rare Lung Diseases at the Justus Liebig University in Giessen, Germany. A replay of the event can be accessed at View Source
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LTI-01 is in development for loculated pleural effusion ("LPE"), a serious consequence of pneumonia with significant unmet medical need.

Second Quarter 2024 Financial Results
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Cash Position: Cash and cash equivalents as of June 30, 2024, were $21.9 million, compared to $12.0 million as of March 31, 2024. After including the net proceeds raised from the May 2024 offering and based on its current operating plan, the Company expects its existing cash and cash equivalents to be sufficient to fund the completion of the Phase 1b clinical trial and its operations into the second half of 2025.

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Research and Development ("R&D") Expenses: R&D expenses for the quarter ended June 30, 2024, were $3.7 million, compared to $0.2 million for the quarter ended June 30, 2023. The increase of $3.5 million was primarily a result of the clinical programs acquired as part of the Company’s acquisition of Lung Therapeutics, Inc. in October 2023 (the "Lung Acquisition"). During the quarter ended June 30, 2024, Aileron incurred expenses of $1.1 million on clinical trials, $2.0 million on manufacturing, and $0.1 million on regulatory and development consulting as well as $0.5 million on employee and related expenses associated with clinical programs acquired in the Lung Acquisition.

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General and Administrative ("G&A") Expenses: G&A expenses for the quarter ended June 30, 2024, were $5.3 million, compared to $1.9 million for the quarter ended June 30, 2023. The increase of $3.4 million was primarily due to increased professional fees of $1.0 million and increased employee and related expenses of $1.8 million as a result of increased business activity and headcount associated with the Lung Acquisition, and increased facilities and other expenses of $0.5 million during the quarter ended June 30, 2024 as compared to the quarter ended June 30, 2023.

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Net Loss: Net loss for the quarter ended June 30, 2024, was $8.9 million, compared to $1.8 million for the quarter ended June 30, 2023. The basic and diluted net loss per share for the quarter ended June 30, 2024 was $0.45 compared to $0.39 for the quarter ended June 30, 2023.

On August 14, 2024 Achilles Therapeutics plc (NASDAQ: ACHL), a clinical-stage biopharmaceutical company developing AI-powered precision T cell therapies targeting clonal neoantigens to treat solid tumors, reported its financial results for the second quarter ended June 30, 2024, and recent business highlights (Press release, Achilles Therapeutics, AUG 14, 2024, View Source [SID1234645884]).

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"During the second quarter we shared interim Phase I/IIa data from our ongoing CHIRON and THETIS TIL-based cNeT clinical trials and established an important research collaboration with Arcturus Therapeutics to explore the use of clonal neoantigens in second-generation personalized mRNA cancer vaccines", said Dr Iraj Ali, Chief Executive Officer of Achilles Therapeutics. "Our insights into the factors that drive durable engraftment and immune evasion led us to add an additional cohort in the CHIRON and THETIS trials to evaluate cNeT persistence and clinical activity in patients with enhanced host conditioning (EHC). These findings, along with the cancer vaccine research collaboration, continue to illustrate the potential value of our platform, including the unparalleled capability of PELEUS to select tumor targets with the highest potential for immune system recognition in a variety of modalities including TIL, neoantigen vaccines, ADCs, and TCR-T therapies.

Our financial position remains strong with more than $95 million in cash, which we expect to support operations through 2025, including the completion of the ongoing Phase I/IIa trials and the Arcturus collaboration."

Clinical and Business Updates
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Announced research collaboration with Arcturus Therapeutics to explore second-generation mRNA cancer vaccines

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Combines Achilles’ AI-powered, tumor-targeting technology with Arcturus’ self-amplifying mRNA platform
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Combined technologies have the potential to generate potent and durable T cell responses in pre-clinical IND-enabling studies 
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Provided an interim Phase I/IIa update on the use of clonal neoantigen reactive T cells (cNeT) in advanced NSCLC and melanoma
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Dosed first EHC patients in CHIRON and THETIS, with the first three EHC patients showing improved cNeT persistence and engraftment
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Delivered 10 products containing over 100 million cNeT and five containing over one billion cNeT
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Matilde Saggese, MD, has been appointed as Interim Chief Medical Officer. Dr. Saggese has served as Vice President and Medical Director of Achilles Therapeutics since March 2021.

Financial Highlights
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Cash and cash equivalents: Cash and cash equivalents were $95.1 million as of June 30, 2024, as compared to $131.5 million as of December 31, 2023. The Company believes that its cash and cash equivalents are sufficient to fund its planned operations through 2025.
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Research and development (R&D) expenses: R&D expenses were $13.6 million for the second quarter ended June 30, 2024, compared to $13.8 million for the second quarter ended June 30, 2023. The decrease was primarily driven by decreased activity in THETIS and lower personnel costs, partially offset by increased activity in CHIRON.
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General and administrative (G&A) expenses: G&A expenses were $4.2 million for the second quarter ended June 30, 2024, compared to $4.3 million for second quarter ended June 30, 2023. This decrease was primarily driven by lower personnel costs and lower insurance premiums.
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Net loss: Net loss for the second quarter ended June 30, 2024 was $16.4 million or $0.41 per share compared to $16.8 million or $0.42 per share for the second quarter ended June 30, 2023.

2024 Focus

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Clinical Data: Report clinical activity and translational science data from patients in CHIRON and THETIS, evaluating the benefit of EHC, with a meaningful data update expected in the second half of 2024
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Arcturus Collaboration: Ongoing evaluation of best-in-class, self-amplifying mRNA (sa-mRNA) personalized cancer vaccines (PCVs) targeting clonal neoantigens with the potential to generate IND-enabling data

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Translational Science: Leverage the Company’s unique bioinformatics platform to better understand the drivers associated with clinical responses
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Manufacturing Development: Continue VELOS and PELEUS development to optimize cNeT dose and functionality

On August 14, 2024 Nykode Therapeutics ASA (OSE: NYKD), a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel immunotherapies, reported that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 12,059,459, entitled, "Therapeutic Anticancer Neoepitope Vaccine" (Press release, Nykode Therapeutics, AUG 14, 2024, View Source [SID1234645817]).

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The newly issued patent describes Nykode’s fully individualized neoantigen based vaccine, VB10.NEO, which is in development for the treatment of locally advanced or metastatic solid tumours. The 20 year expiration date of this patent is January 5, 2037. Related patents were previously granted to the company in Russia, India, and Australia.

Michael Engsig, CEO of Nykode, said, ‘We are very pleased with the granting of this important patent in the U.S., a key market for Nykode. Continuously expanding our patent protection is a core strategy for us. The granting of this patent is a testament to the world-class innovation taking place in Nykode’s labs. We remain dedicated to pushing the boundaries of what’s possible in medicine, bringing hope to patients and their families around the globe."

About VB10.NEO

VB10.NEO is a proprietary individualized neoantigen vaccine in development for the treatment of locally advanced or metastatic solid tumors under an exclusive, worldwide clinical collaboration with Genentech, a member of the Roche Group. The vaccine is designed to be produced on-demand according to the neoantigen profile of an individual patient. Neoantigens are proteins generated by tumor-specific mutations not present in normal tissues and are thus an attractive target for cancer immunotherapy as they may be recognized as foreign by the immune system.

Nykode is currently conducting a clinical study evaluating VB10.NEO: VB N-02, an open-label Phase 1b, dose-escalation study of the safety and antigen-specific immune responses elicited by VB10.NEO in combination with Roche’s checkpoint inhibitor atezolizumab in patients with locally advanced and metastatic tumors (NCT05018273).

On August 13, 2024 CSL reported its results presentation for the full year ended 30 June 2024 (Presentation, CSL, AUG 13, 2024, View Source [SID1234647101]).

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On August 13, 2024 AN2 Therapeutics, Inc. (Nasdaq: ANTX), a biopharmaceutical company focused on discovering and developing novel small molecule therapeutics derived from its boron chemistry platform, reported financial results for the quarter ended June 30, 2024 (Press release, AN2 Therapeutics, AUG 13, 2024, View Source [SID1234646195]).

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"We reported results from the EBO-301 Phase 2/3 study in patients with treatment-refractory MAC lung disease last week and over the coming months will further analyze the data to determine next steps in NTM lung disease," said Eric Easom, Co-Founder, Chairman, President and CEO. "Despite this setback, we remain well positioned as a company – we have a boron chemistry platform with two development programs that are expected to advance into clinical trials in 2025. AN2-502998 is expected to enter Phase 1 with an aim to cure chronic Chagas disease, a potentially life-threatening illness that causes severe cardiac disease and where there are limited to no treatment options. We also plan to initiate a Phase 2 trial with epetraborole for the treatment of melioidosis, a deadly bacterial infection and global bioterrorism threat. Additionally, we have a pipeline of internally developed boron-based compounds in research targeting high unmet needs in infectious disease and oncology and we have the financial runway to allow us to achieve multiple inflection points over the next three years."

Second Quarter & Recent Business Updates:

Termination of Epetraborole Pivotal Phase 2/3 Clinical Study in TR-MAC Lung Disease
AN2 recently announced topline results from the Phase 2 part of the EBO-301 Phase 2/3 study evaluating epetraborole on top of an optimized background regimen in treatment-refractory MAC lung disease. The Phase 2 part of the study met its primary objective of demonstrating the potential validation of a novel patient-reported outcome (PRO) tool and a higher PRO-based clinical response rate in the epetraborole + OBR arm vs. placebo + OBR. However, sputum culture conversion at Month 6, a key secondary endpoint, was similar between treatment arms. Based on the topline data, the Company has terminated the Phase 2 and Phase 3 parts of the EBO-301 trial. Oral epetraborole 500 mg daily was generally well-tolerated and the study was not terminated due to safety concerns. The Company plans to further analyze the EBO-301 data to better understand the results and their impact on the ongoing development of epetraborole for nontuberculous mycobacteria (NTM) lung disease.

Published New Epetraborole Data in Antimicrobial Agents in Chemotherapy
In July, the company published a study in the journal Antimicrobial Agents in Chemotherapy, which highlighted the efficacy of epetraborole against M. abscessus in a mouse lung infection model. The study suggests that epetraborole could become an important therapy to address the high unmet need for effective oral treatment options for M. abscessus lung disease.

Selected Second Quarter Financial Results

Research and Development (R&D) Expenses: R&D expenses for the second quarter of 2024 were $12.1 million compared to $13.5 million for the same period during 2023 due to decreased chemistry manufacturing and controls activity and decreased expenses for completed Phase 1 clinical trials, partially offset by increased Phase 2/3 clinical trial costs, increased consulting and outside service costs and increased personnel-related expenses.
General and Administrative (G&A) Expenses: G&A expenses for the second quarter of 2024 were $3.7 million compared to $3.1 million for the same period during 2023 due to an increase in personnel-related expenses and professional and outside services.
Other Income, Net: Other income, net for the second quarter of 2024 was $1.4 million compared to $0.8 million for the same period during 2023 due to increased interest income based on higher interest rates and higher cash, cash equivalents and investment balances.
Net loss: Net loss for the second quarter of 2024 was $14.4 million, compared to $15.8 million for the same period during 2023.
Cash Position: The Company had cash, cash equivalents, and investments of $104.5 million at June 30, 2024, which is expected to fund operations through 2027.