On August 10, 2021 Achilles Therapeutics plc (NASDAQ: ACHL), a clinical-stage biopharmaceutical company developing precision T cell therapies to treat solid tumors, reported its financial results for the second quarter ended June 30, 2021, and recent business highlights (Press release, Achilles Therapeutics, AUG 10, 2021, View Source [SID1234586201]).

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"Achilles continued to make significant progress in the second quarter of 2021. We presented data at AACR (Free AACR Whitepaper) and ASCO (Free ASCO Whitepaper) demonstrating that we can quantify the active clonal neoantigen-reactive T cell (cNeT) component and cNeT dose of our product. We were also able to track our cNeT post-dosing and describe phenotypic and functional characteristics. We believe that this critical capability will allow us to develop a potency assay which could be a crucial advancement in the TIL therapeutic space. We remain on target to report clinical and characterization data on ten patients that have received cNeT monotherapy across the CHIRON and THETIS trials in the fourth quarter," said Dr Iraj Ali, Chief Executive Officer of Achilles. "We continue to enroll and dose patients and have recruited our first US patient at the Moffitt Cancer Center. Also in the fourth quarter of 2021, we plan to begin enrolling patients using our Process 2 to deliver higher dose cNeT and to open a checkpoint combination cohort in our melanoma (THETIS) study to evaluate the addition of a PD-1 inhibitor to cNeT therapy."

Business Highlights

Closed an initial public offering of 9,750,000 ADRs at a public offering price of $18.00 per share for gross proceeds of $175.5 million.
Enrolled first US Patient in ongoing PI/IIa CHIRON study in non-small cell lung cancer at the Moffitt Cancer Center
Presented a poster at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting describing the ongoing phase I/IIa CHIRON clinical trial evaluating clonal cNeT in patients with advanced non-small cell lung cancer (NSCLC)
Presented data at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting detailing the Company’s comprehensive translational research program and insights into the in vivo dynamics of cNeT post-dosing and the potential to develop a potency-based release assay
Strengthened the Board of Directors and Scientific Advisory Board with the additions of Julie O’Neill and Markwin Velders, Ph.D., respectively
Received a Horizon 2020 grant as part of the Neoantigen Consortium, to collaborate on the development of a tool to predict neoantigen immunogenicity
Financial Highlights

Cash and cash equivalents: Cash and cash equivalents were $299.3 million as of June 30, 2021, as compared to $177.8 million as of December 31, 2020. Current cash and cash equivalents include net proceeds of $160.6 million from an initial public offering that closed in the second quarter. The Company anticipates that its cash and cash equivalents are sufficient to fund its planned operations into the second half of 2023, including full funding of the ongoing Phase I/IIa CHIRON and THETIS clinical trials.
Research and development (R&D) expenses: R&D expenses were $10.8 million for the second quarter ended June 30, 2021, an increase of $6.3 million compared to $4.5 million for the second quarter ended June 30, 2020. R&D expenses were $19.7 million for the six months ended June 30, 2021, an increase of $11.3 million compared to $8.4 million for the six months ended June 30, 2020. The increase was primarily driven by increased activity related to our ongoing clinical trials and overall R&D.
General and administrative (G&A) expenses: G&A expenses were $5.4 million for the second quarter ended June 30, 2021, an increase of $3.0 million compared to $2.4 million for the second quarter ended June 30, 2020. G&A expenses were $10.3 million for the six months ended June 30, 2021, an increase of $6.2 million compared to the $4.1 million for the six months ended June 30, 2020. The increase was primarily driven by fees associated with the Company’s IPO preparation, subsequent public company obligations, and an increase in headcount and related personnel costs.
Net loss: Net loss for the second quarter ended June 30, 2021 was $16.2 million or $0.45 per share compared to $6.9 million or $7.64 per share for the second quarter ended June 30, 2020. Net loss for the six months ended June 30, 2021 was $29.9 million or $1.60 per share compared to $12.1 million or $13.57 per share for the six months ended June 30, 2020. The decrease in loss per share is due in part to the increased number of shares following the conversion and issuance of shares from the IPO.
Upcoming Events
Dr Iraj Ali, Chief Executive Officer, will present at the following investor conferences in September 2021. Additional details will be available at View Source

Wells Fargo Virtual Healthcare Conference: Friday, September 10, 2021
H.C. Wainwright 23rd Annual Global Investment Conference: September 13-15, 2021
Oppenheimer Fall Healthcare Life Sciences & Med Tech Summit: September 20-23, 2021

On August 10, 2021 Equillium, Inc. (Nasdaq: EQ), a clinical-stage biotechnology company developing itolizumab to treat severe autoimmune and inflammatory disorders with high unmet medical need, reported financial results for the second quarter 2021, and provided an update on its clinical programs (Press release, Equillium, AUG 10, 2021, View Source [SID1234586229]).

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"The second quarter of the year was highlighted by positive data from our Phase 1b EQUATE study in acute graft-versus-host disease," said Bruce Steel, chief executive officer at Equillium. "These data were critical for achieving a positive outcome from our End-of-Phase 1 meeting with the FDA and accelerating our plans to immediately advance to a single, pivotal Phase 3 clinical study. This strategy, if successful, may position itolizumab to become the first approved therapy to treat patients with acute graft-versus-host disease in the first-line setting."

Corporate & Clinical Highlights Since Beginning of Q2 2021:

Announced positive topline results from the EQUATE study in first-line treatment of acute graft-versus-host disease presented at the 2021 Virtual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper), and plans to initiate a Phase 3 pivotal study following an End-of-Phase 1 meeting with the FDA
Itolizumab continues to demonstrate favorable safety and efficacy profile
Rapid and durable complete responses resulted in clinically meaningful reduction in corticosteroid use
Data support clinical advancement of itolizumab in first-line treatment of aGVHD
Presented multiple posters at the 104th annual meeting of the American Association of Immunologists; research highlighted:
Itolizumab’s novel mechanism of action and its effect on modulating T cell responses through inhibition of the CD6-ALCAM pathway
Development of a pharmacodynamic biomarker assay to monitor target engagement and fate of CD6 on T cells in patients treated with itolizumab
Upcoming Catalysts:

EQUALISE Phase 1b study: interim data from Type B patients (lupus nephritis) expected 2H 2021
EQUIP Phase 1b study: topline data in uncontrolled asthma expected 2H 2021
Initiate pivotal study in first-line aGVHD expected Q4 2021
Second Quarter 2021 Financial Results

Research and development (R&D) expenses for the second quarter of 2021 were $6.0 million, compared with $3.9 million for the same period in 2020. The increase was driven by greater employee compensation and benefit expenses primarily related to increased headcount, an increase in clinical development expenses primarily related to the EQUATE and EQUALISE studies, as well as greater consulting, overhead, and non-clinical research expenses.

General and administrative (G&A) expenses for the second quarter of 2021 were $2.9 million, compared with $2.7 million for the same period in 2020. The increase was driven by greater employee compensation and benefits primarily related to increased headcount, partially offset by a decrease in non-cash stock-based compensation mainly due to retention option grants issued to certain officers and directors in the second quarter of 2020.

Net loss for the second quarter of 2021 was $9.2 million, or $(0.31) per basic and diluted share, compared with a net loss of $6.5 million, or $(0.37) per basic and diluted share for the same period in 2020. The increase in net loss was largely attributable to greater operating expenses.

Cash used in operations for the second quarter of 2021 was $7.0 million compared to $7.9 million in the first quarter of 2021.

Cash, cash equivalents and short-term investments totaled $97.6 million as of June 30, 2021, compared to $104.1 million as of March 31, 2021. Equillium believes that its cash and investments will be sufficient to fund its currently planned operations into 2023.

About Itolizumab

Itolizumab is a clinical-stage, first-in-class anti-CD6 monoclonal antibody that selectively targets the CD6-ALCAM pathway. This pathway plays a central role in modulating the activity and trafficking of T cells that drive a number of immuno-inflammatory diseases. Equillium acquired rights to itolizumab through an exclusive partnership with Biocon Limited.

On August 10, 2021 Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a precision oncology company developing next-generation therapies that target genetic drivers of cancer, reported financial results and operational updates for the second quarter ended June 30, 2021 (Press release, Turning Point Therapeutics, AUG 10, 2021, View Source [SID1234586251]).

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"Our team made strong progress advancing our 4 clinical drug candidates in 5 ongoing clinical trials as well as our research programs with the first 2 development candidates targeted in the second half of 2022," said Athena Countouriotis, M.D., president and CEO. "In our clinical programs, we are pleased with ongoing enrollment in our pivotal TRIDENT-1 study of repotrectinib. In addition, we recently initiated the Phase 1 expansion cohorts in our SHIELD-1 study of TPX-0022 and just last week were granted Fast Track designation for TPX-0022 in certain gastric cancer indications. We look forward to multiple data updates from our clinical studies at medical conferences during the fourth quarter."

Second quarter and recent highlights include:

REPOTRECTINIB, ROS1/TRK Inhibitor

Enrollment of approximately 300 patients in the Phase 1 and 2 portions of the TRIDENT-1 study, including more than 50 patients in the ROS1-positive TKI-naïve advanced non-small cell lung cancer (NSCLC) patient cohort (EXP-1). Enrollment in the EXP-1 cohort is ongoing to provide continued access to new patients.

Acceptance of TRIDENT-1 clinical data for presentation at the 2021 AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in October.

Acceptance of initial clinical data from the ongoing Phase 1/2 CARE study in pediatric and young adult patients with advanced solid tumors harboring ALK, ROS1 or NTRK alterations for an oral presentation at the 53rd Congress of the International Society of Paediatric Oncology (SIOP) in October.

First patients dosed in China in the TRIDENT-1 study as part of the company’s partnership with Zai Lab to develop repotrectinib in greater China. Achievement of the milestones resulted in revenue of $5 million to Turning Point under its collaboration agreement with Zai Lab.
TPX-0022, MET/ SRC/CSF1R Inhibitor

Selection of the likely recommended Phase 2 dose (RP2D) in the ongoing Phase 1 SHIELD-1 study of TPX-0022, Turning Point’s MET, SRC and CSF1R inhibitor, and initiation of Phase 1 expansion cohorts. Subject to feedback from the U.S. Food and Drug Administration (FDA) at an end of Phase 1 meeting in the third quarter, including agreement on the RP2D, the company plans to revise the study into a potentially registrational Phase 1/2 and proceed into the Phase 2 portion.

Acceptance of SHIELD-1 clinical data for presentation at the 2021 AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in October.

Orphan Drug Designation granted by the FDA for the treatment of patients with gastric cancer, including gastroesophageal junction adenocarcinoma (GEJ).

Fast Track Designation granted by the FDA for the treatment of patients with MET amplified advanced or metastatic gastric cancer or GEJ adenocarcinoma after prior chemotherapy.
TPX-0046, RET Inhibitor

Progress in the ongoing dose-finding portion of the Phase 1/2 SWORD-1 study, where the company continues to evaluate multiple doses and schedules to further characterize the pharmacokinetics, safety, and efficacy profile before determining the RP2D.
TPX-0131, ALK Inhibitor

Ongoing patient dosing in the Phase 1/2 FORGE-1 study of TPX-0131 in locally advanced or metastatic TKI-pretreated ALK-positive NSCLC. The study endpoints include safety and tolerability, determination of the recommended Phase 2 dose, pharmacokinetics, and any early signals of efficacy.

Publication of preclinical data in the AACR (Free AACR Whitepaper) Journal of Molecular Cancer Therapeutics showing TPX-0131 to be potent against a wide range of ALK resistant mutations, including G1202R, L1196M and multiple compound mutations.
Discovery

Advancing four internal discovery programs targeting aberrant GTPase signaling known to drive genomically defined cancers with significant unmet medical need. The most advanced programs target KRAS G12D and the p21 activated kinase, or "PAK" family. Turning Point is targeting 2 development candidates in the second half of 2022 with a goal to achieve at least one new IND per year beginning in 2023.
Second Quarter Financial Results

Revenue: Revenue of $5.2 million recognized during the quarter was driven primarily by milestones earned from Zai Lab (Shanghai) Co. Ltd. under the company’s license agreement for repotrectinib in Greater China. Revenue for the first half of 2021 totaled $30.4 million.

R&D Expenses: Research and development expenses were $44.7 million in the quarter, compared to $24.2 million in the second quarter of 2020. The $20.5 million increase was primarily driven by the year-over-year increase in investments to develop repotrectinib, TPX-0022, TPX-0046 and TPX-0131, discovery efforts and personnel expenses. R&D expenses for the first half of 2021 totaled $85.9 million.

G&A Expenses: General and administrative expenses were $17.2 million compared to $8.6 million in the second quarter of 2020, primarily related to higher personnel expenses from an increase in head count and professional services. G&A expenses for the first half of 2021 totaled $37.2 million.

Net Income/Loss: Net loss was $56.3 million compared to a net loss of $31.5 million for the second quarter of 2020. Net loss for the first half of 2021 was $91.8 million.

Cash position: Cash, cash equivalents and marketable securities at June 30, 2021 totaled approximately $1.1 billion. Net cash used during the first half of 2021 was $44.7 million. Turning Point projects its cash position funds current operations into 2024.
Upcoming Milestones
Key milestones anticipated in the second half of 2021 include:

Repotrectinib

Initiate the first cohort of a multi-arm Phase 1b/2 TRIDENT-2 combination study in patients with KRAS mutant G12D advanced solid tumors in the third quarter

Provide a clinical data update by physician assessment from multiple ROS1 and NTRK patient cohorts of the Phase 2 TRIDENT-1 study at the AACR (Free AACR Whitepaper)-NCI-EORTC conference in October

Report initial clinical data from the ongoing Phase 1/2 CARE study in pediatric and young adult patients in an oral presentation at the 53rd SIOP Congress in October
TPX-0022

Provide a clinical data update across multiple tumor types and MET genetic alterations from the Phase 1 dose finding portion of the SHIELD-1 study at the AACR (Free AACR Whitepaper)-NCI-EORTC conference in October

Initiate the Phase 2 portion of the SHIELD-1 study, pending FDA feedback, in the fourth quarter

Initiate the Phase 1b/2 SHIELD-2 study of TPX-0022 in combination with an epidermal growth factor receptor (EGFR) targeted therapy in the fourth quarter
Webcast, Conference Call, Upcoming Investor Conferences
Turning Point will webcast its Quarterly Update Conference Call today, Aug. 9 at 4:30 p.m. ET/1:30 p.m. PT. Dr. Countouriotis will host the call, which will be accessible through the "Investors" section of tptherapeutics.com or by dialing (877) 388-2118 (in the United States) or (470) 495-9489 (outside the U.S.) using conference ID 4029189. A replay will be available through the "Investors" section of www.tptherapeutics.com.

Dr. Countouriotis will also participate in a targeted oncology panel discussion at the 2021 Wedbush Pacgrow Virtual Healthcare Conference on Aug. 10 at 10:55 a.m. ET, and a "fireside chat" question-and-answer session at the 41st Annual Canaccord Genuity Growth Conference on Aug. 11 at 4 p.m. ET. Both sessions will be accessible through the "Investors" section of www.tptherapeutics.com.

On August 10, 2021 Nouscom, a clinical stage immuno-oncology company developing off-the-shelf and personalized cancer neoantigen vaccines, reported the online publication of new research describing its novel and proprietary algorithm for the identification and prioritization of patient-specific tumor neoantigens, VENUS (Press release, NousCom, AUG 10, 2021, View Source [SID1234586268]).

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The VENUS algorithm predicts tumor specific mutated peptides that have the highest likelihood of inducing an immune response and act as neoantigens. Each neopeptide is assigned a score that combines its abundance and expression across tumor cells, with the likelihood of being presented on the tumor cell surface.

The paper entitled: "VENUS, a Novel Selection Approach to Improve the Accuracy of Neoantigens’ Prediction" (G. Leoni, A.M. D’Alise et al, reference below) has been published in the special issue of the peer-reviewed journal Vaccines focused on cancer neo-antigen vaccines.

The publication describes the predictive power of VENUS both in silico and in vivo when incorporated into Nouscom’s heterologous prime boost viral vector platform. In silico, the authors demonstrated VENUS’ ability to select 19 out of 20 neo-antigens inducing spontaneous CD8 or CD4 T cell mediated immune responses, from the top 60 ranked mutated peptides from 9 cancer patients.

In addition, an adenoviral vector vaccine encoding the top 60 ranked mutations, demonstrated efficacy and a potent T cell response, when used in combination with an anti-PD1 checkpoint inhibitor, in a murine cancer model.

The VENUS algorithm, in combination with Nouscom’s heterologous prime/boost viral vector platform, comprising a proprietary non-human adenoviral vector (GAd) and Modified Vaccinia Ankara vector (MVA), has been used to develop NOUS-PEV, a novel personalized cancer immunotherapy. NOUS-PEV is being evaluated in a Phase 1b clinical trial in combination with the anti-PD-1 checkpoint inhibitor pembrolizumab in patients with either locally advanced 1L melanoma or 1L non-small cell lung cancer (NSCLC) expressing more than 50% PD-L1. The trial (NCT04990479) is currently enrolling patients across multiple sites in Europe.

Dr. Elisa Scarselli, Chief Scientific Officer and Co-Founder of Nouscom, said: "The data published in Vaccines highlights the significance of using VENUS to enhance the accuracy of neoantigen prediction for cancer vaccine development. Combining VENUS with Nouscom’s viral vector platform allows the targeting of up to 60 tumor mutations in each patient that can generate broad and deep immune responses, potentially overcoming issues of tumor heterogeneity and escape through immunoediting. This could be an important advantage for Nouscom’s approach to personalized cancer immunotherapy."

"We look forward to announcing the first patient being dosed with Nouscom’s personalized immunotherapy, NOUS-PEV.”

References

G. Leoni, A.M. D’Alise et al. VENUS, a Novel Selection Approach to Improve the Accuracy of Neoantigens’ Prediction Vaccines (MDPI).

Online publication: View Source

About NOUS-PEV

NOUS-PEV is a personalized cancer immunotherapy designed for each patient based on selection and prioritization of mutations unique to that patient’s tumor. The strategy is based on Nouscom’s heterologous prime boost platform already clinically validated by its lead off-the-shelf clinical development program NOUS-209, composed of a proprietary non-human adenoviral vector (GAd) and Modified Vaccinia Ankara vector (MVA). Each of the two viral vector systems encodes multiple personalized neoantigens selected with a proprietary algorithm (VENUS), which prioritizes up to 60 mutations that represent the most immunogenic neoantigens.

NOUS-PEV is being evaluated in a Phase 1b clinical trial in combination with the anti-PD-1 checkpoint inhibitor pembrolizumab in patients with either locally advanced 1L melanoma or 1L non-small cell lung cancer (NSCLC) expressing more than 50% PD-L1. The trial (NCT04990479) commenced in 2021 and is currently enrolling patients across multiple clinical sites in Europe.

On August 10, 2021 Mabwell reported that NMPA has accepted the company’s IND application for an anti-Nectin-4 ADC(R&D code: 9MW2821) (Press release, Mabwell Biotech, AUG 10, 2021, View Source [SID1234617925]). This is China’s first drug candidate that has been accepted by the NMPA for IND application review among its domestic counterparts using the same target.

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9MW2821, categorized as a Class I innovative drug, is a novel anti-Nectin-4 ADC drug candidate developed by world-class ADC drug development platform and automated high-throughput antibody discovery platform of Mabwell. This drug candidate has achieved site-specific modification based on the ADC conjugate technology jointly developed by Mabwell and Shanghai Institute of Materia Medica.

9MW2821 boasts the advantages of homogenous structure, high purity and being easy production. It has also demonstrated favorable druggability properties in binding affinity, endocytosis, preliminary in vivo and in vitro pharmacodynamic activities, drug metabolism properties and preliminary safety. Results from preclinical pharmacological and toxicological studies showed that this drug candidate has favorable anti-tumor effects in a variety of animal tumor models. In addition, its safety in cynomolgus monkeys and mice indicated a superior safety margin.

9MW2821 will be evaluated in advanced and metastatic urothelial carcinoma in a Phase 1 trial, and Mabwell plans to explore its potential in other solid tumors in future trials.