On May 13, 2019 Neon Therapeutics, Inc. (Nasdaq: NTGN), a clinical-stage immuno-oncology company developing neoantigen-based therapeutics, reported financial results for the first quarter ended March 31, 2019 and provided a business update (Press release, Neon Therapeutics, MAY 13, 2019, View Source [SID1234536227]).

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"The field of cancer immunotherapy has advanced considerably in recent years, but there remains a significant need for new approaches that could extend the benefits of immunotherapy and lead to improved patient outcomes. Neoantigen-based therapies hold the promise to transform the treatment of cancer and we are proud of our leadership in pursuing these untapped targets," said Hugh O’Dowd, Neon’s Chief Executive Officer. "We continue to make important progress across the Company, including our efforts to establish RECON as the best-in-class neoantigen bioinformatic prediction engine with our machine learning and proteomics expertise. We intend to further strengthen our position as a class-leader in neoantigen selection by continuing to leverage proprietary immunogenicity data from our ongoing clinical studies. We remain excited about numerous upcoming clinical milestones, including the top-line results from our NT-001 study of NEO-PV-01, which we expect to be the first clinical dataset ever reported with 12-month follow-up for a personal neoantigen vaccine in metastatic melanoma, non-small cell lung and bladder cancers."

Pipeline Updates
Neon is developing neoantigen-targeting therapies across multiple treatment modalities, including vaccines, adoptive T cell therapies and TCR-based T cell therapies to bring benefit to a broad set of patients across tumor types and stages of disease. This approach requires effectively identifying and selecting the optimal neoantigen targets for the unique mutational fingerprint of each individual patient’s tumor. Neon is pioneering a proprietary neoantigen platform to identify and harness the most therapeutically relevant neoantigens, including through its RECON bioinformatic neoantigen prediction engine, deep capabilities in peptide chemistry and manufacturing, and NEO-STIM T cell biology and immune-monitoring expertise.
Neon is using two distinct neoantigen therapeutic approaches across this platform: personal therapies that target individualized mutations (NEON / ONE), and off-the-shelf precision therapies (NEON / SELECT) that target shared mutations present in sub-populations of cancer patients.
"Neon is pursuing novel therapeutic approaches at the frontiers of immuno-oncology. We designed our R&D strategy to enable us to determine how our neoantigen-targeted platform and product candidates could improve patient outcomes beyond the limitations of current standard of care treatment. Our near-term R&D strategy is focused on leveraging Phase 1b clinical trials to determine the optimal patient selection, rational combinations and trial designs to inform subsequent Phase 2 and later stage clinical trials," said Richard Gaynor, M.D., Neon’s President of Research and Development.
NEON / ONE is Neon’s personal medicine approach to neoantigen-targeted therapies that are tailored for the individual profile of each patient’s tumor. Neon’s initial clinical strategy has focused on solid tumor indications where checkpoint inhibitors have been approved, either in combination or in refractory settings.

NEO-PV-01: Personal Neoantigen Vaccine

NEO-PV-01: Neon is evaluating its lead product candidate, a personal neoantigen vaccine, in a variety of treatment settings in Phase 1b development to determine the optimal late-stage clinical trials.

NT-001 Trial: Neon’s ongoing Phase 1b open-label clinical trial is evaluating a combination of NEO-PV-01 with OPDIVO (nivolumab) in patients with metastatic melanoma, non-small cell lung cancer (NSCLC) or bladder cancer.

Neon recently presented updated data from NT-001 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, describing the immune and pathologic markers associated with study subjects remaining progression-free at nine months. The analysis both confirmed the mechanism of action of NEO-PV-01 and highlighted new data on the association between histology and epitope spread in patients who had not progressed at nine months in the melanoma cohort. In addition, multiple molecular markers were determined to correlate with lack of clinical progression, which may prove useful for patient selection in late-stage clinical trials.

Neon expects to report top-line clinical results, including 12-month follow-up, from NT-001 in July 2019. The results are expected to include data from an intention-to-treat (ITT) analysis from 82 enrolled patients, including median progression-free survival (PFS) and objective response rates (ORR), safety and baseline patient characteristics in each of the three tumor cohorts.

NT-002 Trial: Neon recently completed enrollment in NT-002, its Phase 1b clinical trial evaluating NEO-PV-01 in combination with the current standard of care, KEYTRUDA (pembrolizumab) and chemotherapy, in first-line patients with untreated advanced or metastatic NSCLC. This trial is evaluating the safety, tolerability and efficacy of NEO-PV-01 in the metastatic setting. Neon expects to report immune monitoring and clinical outcome data from this trial by the end of Q3 2020.

NT-003 Trial: This Phase 1b clinical trial of NEO-PV-01 in metastatic melanoma combinations is currently enrolling. NT-003 will evaluate NEO-PV-01 and OPDIVO in combination with other agents, including Apexigen’s CD40 agonist (APX005M) or a CTLA-4 antagonist, to further enhance any NEO-PV-01-induced neoantigen immune response and improve clinical outcomes. This trial will also evaluate alternative NEO-PV-01 dosing schedules. Neon plans to announce immune monitoring data from this study in 2H 2020.

NT-004 Trial: Neon is planning to explore NEO-PV-01 in a monotherapy approach in an earlier disease setting in this Phase 1b trial.

NEO-PTC-01: Personal Neoantigen T cell Therapy

NEO-PTC-01: Neon’s personal adoptive T cell therapy consists of multiple T cell populations targeting neoantigens that are predicted to be the most therapeutically relevant from each patient’s tumor. NEO-PTC-01 uses T cells from the periphery of each patient that are then primed, activated and expanded to generate a therapy that specifically targets that patient’s personal neoantigens, with the potential to drive a robust and persistent anti-tumor response.

CTA Filing: Building on success to date in generating both memory and de novo immune responses, Neon is completing process development, which supports its plan to file a Clinical Trial Application (CTA) in Europe in the second half of 2019 to evaluate NEO-PTC-01 in refractory solid tumor settings. This work is being performed in collaboration with the Netherlands Cancer Institute (NKI), a leading academic research and treatment center with expertise in T cell biology and treatments.
NEON / SELECT is Neon’s off-the-shelf precision approach to neoantigen-targeted therapies that seek to target prevalent neoantigens that are shared across subsets of patients or tumor types.

NEO-SV-01: Breast Neoantigen Vaccine

NEO-SV-01: Neon is planning to develop its off-the-shelf neoantigen vaccine for the treatment of a genetically-defined subset of hormone receptor-positive (HR+) breast cancer, potentially across disease stages in combination with hormonal, chemotherapy or targeted therapies.

IND Filing: Following the completion of target validation and preclinical product development work, Neon expects to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration for a Phase 1b clinical trial of NEO-SV-01 in Q2 2019.

TCR-based T cell Therapies

TCRs: Neon continues to advance its precision T cell approach and has assembled libraries of high-quality T-cell receptors (TCRs) against various shared neoantigens. Neon is leveraging its proprietary NEO-STIM

induction protocol to rapidly generate T cells specific to these targets where multiple TCRs can be sequenced and characterized.
Expected Milestones
Clinical Trial Results:

NEO-PV-01: Top-line clinical results, including 12-month follow-up, from NT-001 Phase 1b trial in metastatic melanoma, NSCLC and bladder cancers (July 2019)

NEO-PV-01: Clinical results and correlative immune data from NT-001 Phase 1b trial planned for presentation at a medical congress (2H 2019)

NEO-PV-01: Clinical results and correlative immune data, including 12-month follow-up, from NT-002 Phase 1b trial in first-line metastatic NSCLC (Q3 2020)

NEO-PV-01: Immune data from NT-003 Phase 1b trial in metastatic melanoma combinations (2H 2020)
Clinical Trial Applications/Initiations:

NEO-SV-01: U.S. IND filing to evaluate NEO-SV-01 in a genetically defined subset of HR+ breast cancer (Q2 2019)

NEO-PTC-01: European CTA filing to evaluate NEO-PTC-01 in a refractory solid tumor setting (2H 2019)
First Quarter 2019 Financial Results and Financial Guidance:

R&D Expenses: Research and development expenses were $16.2 million for the first quarter of 2019, compared to $13.2 million for the same period last year. The increase was primarily due to costs related to research and development of NEO-PV-01, NEO-PTC-01 and NEO-SV-01, as well as investments in R&D headcount to support the advancement of Neon’s pipeline.

G&A Expenses: General and administrative expenses were $5.4 million for the first quarter of 2019, compared to $3.6 million for the same period last year. The increase was primarily due to personnel-related expenses, professional fees to support expanding operations and costs associated with being a public company.

Net Loss: Net loss attributable to common stockholders was $21.0 million for the first quarter of 2019, compared to $19.7 million for the same period last year.

Cash Position: As of March 31, 2019, cash, cash equivalents and marketable securities were $81.3 million, as compared to cash, cash equivalents and marketable securities of $103.3 million as of December 31, 2018.

Financial Guidance: Based on its current operating plan, Neon expects that its existing cash, cash equivalents and marketable securities will enable the Company to fund its anticipated operating expenses and capital expenditure requirements into at least Q2 2020.
OPDIVO is a registered trademark of Bristol-Myers Squibb Company. KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.