On November 10, 2021 Immunocore Holdings plc (Nasdaq: IMCR), a late-stage biotechnology company pioneering the development of a novel class of T cell receptor (TCR) bispecific immunotherapies designed to treat a broad range of diseases, including cancer, infectious and autoimmune disease, reported its financial results for the quarter and nine months ended September 30, 2021 and provides a business update (Press release, Immunocore, NOV 10, 2021, View Source [SID1234595051]).

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Immunocore’s recent and third quarter highlights include the acceptance of tebentafusp regulatory submissions in the US, EU and UK; the publication of Phase 3 tebentafusp data in the New England Journal Medicine; and the continued dose escalation of MAGE-A4 and PRAME targeting ImmTACs with data to be presented from IMC-C103C targeting MAGE-A4 at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Immuno-Oncology Congress in December of this year (ESMO IO Congress).

Bahija Jallal, Chief Executive Officer of Immunocore, said: "We continue to be encouraged by the interest in our tebentafusp data in metastatic uveal melanoma, including the publication of our Phase 3 data in the New England Journal of Medicine. We have now activated our early access program in fourteen countries and have treated over 150 patients with metastatic uveal melanoma over the last six months. As we advance our ImmTAC programs in other solid tumors, we look forward to continuing to update on our progress at upcoming medical meetings."

Third Quarter 2021 Highlights (including post-period)

Tebentafusp

Earlier this month, the Company presented new clinical data from the metastatic uveal melanoma (mUM) tebentafusp monotherapy program and a Phase 1b study of tebentafusp in combination with durvalumab (anti-PDL1) and/or tremelimumab (anti-CTLA4) in metastatic cutaneous melanoma (mCM) in poster presentations at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 36th Annual Meeting. In a phase 1b trial in mCM of tebentafusp in combination with checkpoint inhibitors, in which the majority of patients had previously received prior anti-PD(L)1 treatments, the maximum target doses of tebentafusp (68 mcg) plus durvalumab (20 mg/kg) with and with/out tremelimumab (1 mg/kg) were tolerated in both doublet and triplet arms of the study. Preliminary evidence of tebentafusp clinical activity in mCM patients who had prior anti-PD(L)1 therapy, currently an unmet medical need, included 1-year overall survival (OS) rate of 76%. In mCM patients who were refractory (defined as best response of progressive disease) to prior anti-PD(L)1, the 1-year OS rate was 61%. In addition, the Company presented a new analysis of baseline gp100 protein tumor expression by immunohistochemistry of tumor biopsies from the phase 2 and phase 3 tebentafusp monotherapy mUM trials, where OS benefit was observed for both high and low gp100 protein tumor expression. Four additional posters depicting new analyses from tebentafusp in metastatic uveal melanoma, as well as the Company’s proprietary soluble TCR bispecific ImmTAC platform were also accepted for presentation at the upcoming SITC (Free SITC Whitepaper) 36th Annual Meeting and will be made available for on-demand viewing throughout the meeting.

In October, the Company announced an exclusive multi-regional agreement for Medison Pharma Ltd. to help seek regulatory authorization and commercialize Immunocore’s tebentafusp (IMCgp100), for the treatment of patients with mUM, in Canada, twenty markets across Central Eastern Europe and Israel. Under the agreement, Medison Pharma would also provide assistance with commercialization activities, assuming regulatory approval is received.

In the third quarter, the Australian Government Department of Health granted tebentafusp Orphan Drug Designation. Additionally, the Australian Government Department of Health has accepted the Marketing Application for tebentafusp in mUM, and the company (through the Adjutor Healthcare Party Ltd.) has also received a Priority Review of its application for approval.

In September, The New England Journal of Medicine (NEJM) published online data from the IMCgp100-202 Phase 3 randomized clinical trial in mUM where the OS Hazard Ratio (HR) in the intent-to-treat population favored tebentafusp, HR=0.51 (95% CI: 0.37, 0.71). The NEJM paper concluded that tebentafusp prolonged OS compared to investigator’s choice in previously untreated mUM.

In September, the Company presented new data and analysis from tebentafusp at the ESMO (Free ESMO Whitepaper) Congress. The findings presented in an oral presentation, by Alexander N. Shoushtari MD, medical oncologist at Memorial Sloan Kettering Cancer Center, demonstrated that 70% of evaluable patients had a reduction in circulating tumor DNA (ctDNA) by Week 9 and the degree of reduction was strongly associated with OS.

In September, the Company announced the United Kingdom’s Medicines and Healthcare products Regulatory Agency has accepted a MAA seeking the approval of tebentafusp for the treatment of patients with mUM.

In August, the U.S. Food and Drug Administration (FDA) accepted for review Immunocore’s BLA for tebentafusp. The FDA has granted Priority Review to the Company’s BLA submission, a designation for drugs which, if approved, may provide significant improvements in the safety and effectiveness of the treatment of serious conditions. Priority Review designation shortens the review period from the standard ten months to six months from the filing acceptance of the BLA, and therefore, there is a PDUFA target action date of February 23, 2022.

The FDA will review the BLA for tebentafusp under the Real-Time Oncology Review (RTOR) pilot program, an initiative of the FDA’s Oncology Center of Excellence designed to expedite the delivery of safe and effective cancer treatments to patients. Tebentafusp is also being reviewed under the FDA’s Project Orbis initiative, which enables concurrent review by the health authorities in partner countries that have requested participation. Previously, the FDA granted Breakthrough Therapy Designation to tebentafusp for the treatment of HLA-A*02:01-positive adult patients with unresectable or mUM. Over 150 patients have accessed tebentafusp through the global early access program across 14 countries.

In August, the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP), accepted the Company’s MAA. In July, the EMA agreed to the Company’s request for accelerated assessment of its MAA based on the determination that tebentafusp is a product of major interest for public health and therapeutic innovation. Accelerated assessment potentially reduces the time frame for the CHMP and Committee for Advanced Therapies to review the Company’s submitted MAA for advanced therapies. While the CHMP review period of a MAA can take up to 210 days, the accelerated assessment reduces the timeframe for review of the MAA to 150 days (excluding clock-stops).

IMC-C103C targeting MAGE-A4

In the third quarter, the Company continued to dose escalate IMC-C103C, an ImmTAC molecule targeting an HLA-A*02:01 MAGE-A4 antigen, in a first-in-human, Phase 1/2 dose escalation trial in patients with solid tumor cancers including non-small-cell lung cancer (NSCLC), gastric, head and neck, and ovarian. As of June 30, 2021, the Company has enrolled 39 patients in the Phase 1 study. Early pharmacodynamic data indicate that IMC-C103C monotherapy is demonstrating biological activity at the doses currently under evaluation. The Company plans to report the initial Phase 1 data at the ESMO (Free ESMO Whitepaper) IO Congress in December. Immunocore will also host an investor call on December 6th that will be accessible via the ‘Investor Relations’ section of the Company’s website.

IMC-F106C targeting PRAME

In the third quarter, the Company continued to dose escalate IMC-F106C, an ImmTAC molecule targeting an HLA-A*02:01 PRAME antigen, in a first-in-human, Phase 1/2 dose escalation trial in patients with multiple solid tumor cancers. PRAME is overexpressed in many solid tumors including NSCLC, SCLC, endometrial, ovarian, melanoma and breast cancers. As of June 30, 2021, the company has enrolled 23 patients in the Phase 1 study. Early pharmacodynamic data indicate that IMC-F106C monotherapy is demonstrating biological activity at the doses currently under evaluation. The Company plans to report the initial Phase 1 data in mid-2022.

IMC-I109V targeting HBV

In the third quarter, the Company continued to enroll patients in the IMC-I109V global Phase 1 single ascending dose trial. IMC-I109V is the first candidate in development using the Company’s immune‐mobilising monoclonal T cell receptors against virus (ImmTAV) platform to enter clinical trials. IMC-I109V targets a conserved Hepatitis B virus (HBV) envelope antigen and is being developed as a potential functional cure.

IMC-M113V targeting HIV

In the third quarter, the Company continued to advance IMC-M113V, an ImmTAV molecule target an HIV gag antigen bispecific TCR molecule. The Company’s HIV programs are funded by the Bill & Melinda Gates Foundation, and regulatory submission to enable clinical testing is anticipated in the second half of 2021.

Financial Results

Basic and diluted loss per share was £0.69 or $0.93 for the three months ended September 30, 2021 compared to an adjusted £0.72 for the three months ended September 30, 2020. Basic and diluted loss per share was £2.19 or $2.95 for the nine months ended September 30, 2021 compared to an adjusted £2.02 for the nine months ended September 30, 2020. Total operating loss for the three months ended September 30, 2021 was £31.0 million or $41.7 million compared to £23.4 million for the same period last year. Total operating loss for the nine months ended September 30, 2021 was £97.3 million or $131.1 million compared to £66.0 million for the same period in the prior year. The increases in operating loss were driven by increases in employee costs associated with a non-cash share-based payment charge.

Revenue for the three and nine months ended September 30, 2021 was £5.9 million or $8.0 million and £19.9 million or $26.8 million, respectively, as compared to £6.7 million and £22.7 million, respectively, for the three and nine months ended September 30, 2020. The decrease in revenue was primarily due to a reduction in activity under our collaboration agreements.

For the three and nine months ended September 30, 2021, our research and development ("R&D") expenses were £16.8 million or $22.6 million and £53.2 million or $71.6 million, respectively, as compared to £20.4 million and £57.6 million, respectively, for the three and nine months ended September 30, 2020. The reduction in R&D expenses was largely attributable to a reduction in clinical trial activity for tebentafusp as we seek regulatory approval and prepare for commercial launch.

For the three and nine months ended September 30, 2021, our administrative expenses were £20.0 million or $27.0 million and £64.0 million or $86.3 million, respectively, compared to £9.7 million and £31.6 million respectively, for the three and nine months ended September 30, 2020. The overall increase was driven by a £6.4 million and £19.3 million increase, respectively, in the non-cash share-based payment charge. In addition, pre-commercial expenditure relating to tebentafusp increased by £4.8 million and £10.4 million, respectively, in the three and nine months ended September 30, 2021.

Cash and cash equivalents were £256.6 million or approximately $345.6 million as of September 30, 2021 compared to £129.7 million as of December 31, 2020.

About Tebentafusp

Tebentafusp is a novel bispecific protein comprised of a soluble T cell receptor fused to an anti-CD3 immune-effector function. Tebentafusp specifically targets gp100, a lineage antigen expressed in melanocytes and melanoma, and is the first molecule developed using Immunocore’s ImmTAC technology platform designed to redirect and activate T cells to recognise and kill tumour cells. Tebentafusp has been granted Priority Review; Real Time Oncology Review; Breakthrough Therapy Designation, Fast Track designation and orphan drug designation by the FDA in the United States; orphan drug status in the European Union; and Promising Innovative Medicine (PIM) designation under the UK Early Access to Medicines Scheme for metastatic uveal melanoma. The European Medicine Agency (EMA) has granted the tebentafusp Marketing Authorization Application (MAA) for an Accelerated Assessment procedure based on the Committee for Medicinal Products for Human Use (CHMP) agreement that tebentafusp is a product of major interest for public health and therapeutic innovation. Tebentafusp is also being reviewed under the FDA’s Project Orbis initiative, which enables concurrent review by the health authorities in partner countries that have requested participation. For more information about enrolling in tebentafusp clinical trials for metastatic uveal melanoma, please visit ClinicalTrials.gov (NCT03070392).

On November 10, 2021 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported financial results for the third quarter ended September 30, 2021 and provided corporate updates (Press release, Cogent Biosciences, NOV 10, 2021, View Source [SID1234595050]).

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"We are pleased to announce that we have started the SUMMIT trial, a Phase 2 study of bezuclastinib in patients with nonadvanced systemic mastocytosis," said Andrew Robbins, President and CEO of Cogent Biosciences. "Based on recently presented preclinical data, we believe that bezuclastinib has best-in-class potential as a highly potent and selective KIT mutant inhibitor and look forward to initiating the PEAK trial for GIST patients in the coming weeks."

Recent Program and Corporate Highlights

SUMMIT trial initiated in NonAdvSM patients
Cogent initiated SUMMIT, a randomized, double-blind, placebo-controlled, global, multicenter, Phase 2 clinical trial. The study is designed to explore the safety and efficacy of bezuclastinib in patients with moderate to severe Indolent Systemic Mastocytosis (ISM) or Smoldering Systemic Mastocytosis (SSM).
SUMMIT is designed in three parts. Part 1 will enroll approximately 48 patients across 3 dose cohorts, plus one placebo arm, and is designed to confirm the optimal bezuclastinib dose. In addition, Part 1 will serve to validate a patient-reported outcomes (PRO) tool for use in assessing efficacy during Part 2 of the trial. Part 2 of SUMMIT will be randomized, double-blind, and placebo-controlled at a single dose level and will include a primary endpoint of disease improvement using the PRO tool from Part 1. After participation in Part 1 or Part 2, all patients may receive bezuclastinib in a long-term extension.
Learn more about the SUMMIT trial at cogentclinicaltrials.com
APEX trial on track for preliminary clinical data readout in the first half of 2022
Cogent is currently enrolling APEX, a Phase 2 clinical trial of bezuclastinib in patients with Advanced Systemic Mastocytosis (AdvSM) and expects to report preliminary clinical data at a scientific conference during the first half of 2022, including levels of serum tryptase, a validated biomarker of mast cell activity.
Learn more about the APEX trial at cogentclinicaltrials.com.
PEAK trial of bezuclastinib and sunitinib for GIST patients to start in 2021
Following recent positive interactions with the FDA, Cogent remains on track to initiate PEAK, a Phase 3 clinical trial of bezuclastinib in combination with sunitinib in imatinib-resistant GIST patients, during 2021.
Announces updated bezuclastinib formulation in partnership with Serán Biosciences
Leveraging Serán’s expertise in formulation and process optimization, an updated formulation of bezuclastinib has been developed. This formulation is expected to reduce the number of daily tablets, improving the overall patient experience.
Updated formulation will be used in PEAK trial beginning in 2021.
Presented new preclinical data supporting bezuclastinib as potential best-in-class KIT inhibitor
Preclinical data presented at the 2021 AACR (Free AACR Whitepaper)-NCI-EORTC Virtual 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) showed further evidence of bezuclastinib as a differentiated, potent, and selective KIT inhibitor.
In head-to-head studies comparing several commercial and development-stage KIT mutant inhibitors, bezuclastinib demonstrated minimal activity against closely related kinases, including PDGFR.
In a nonclinical safety pharmacology study in rodents, bezuclastinib demonstrated minimal brain penetration with a low brain-to-plasma ratio.
Appointed Dana Martin as Chief Patient Officer & Senior Vice President, Medical Affairs
Dr. Martin joins with over 20 years of experience in the biopharmaceutical industry. Prior to joining Cogent, he held several roles of increasing leadership responsibility in the areas of clinical pharmacy, medical affairs, and patient advocacy at Genzyme Corporation, Synageva BioPharma, Sarepta Therapeutics, and Kiniksa Pharmaceuticals. Dr. Martin has contributed to the clinical development and/or product launch of multiple rare disease therapeutics, including first-to-market treatments for Fabry disease, Pompe disease, lysosomal acid lipase deficiency, and Duchenne muscular dystrophy. He holds a Bachelor of Science in pharmacy and a Doctor of Pharmacy from Massachusetts College of Pharmacy-Boston.
Appointed Courtney Watson as Vice President of Clinical Development Operations
Mrs. Watson joins Cogent with nearly 15 years of experience in the biopharmaceutical industry. Prior to joining Cogent, Mrs. Watson was the Head of Clinical Operations at Fusion Pharmaceuticals. Previously, she served in various Clinical Operations roles of increasing responsibility at Forma Therapeutics, Synageva BioPharma, and Ziopharm Oncology. Mrs. Watson holds a Bachelor of Arts from the University of Southern Maine.
Third Quarter 2021 Summarized Financial Results

R&D Expenses: Research and development expenses were $14.8 million for the third quarter of 2021 as compared to $5.0 million for the third quarter of 2020. Research and development expenses include non-cash stock compensation expense of $1.4 million for the third quarter of 2021 compared to $1.9 million for the third quarter of 2020.
G&A Expenses: General and administrative expenses were $5.0 million for the third quarter of 2021 as compared to $5.6 million for the third quarter of 2020. General and administrative expenses include non-cash stock compensation expense of $2.0 million for the third quarter of 2021 compared to $1.6 million for the third quarter of 2020.
Net Loss: Net loss was $19.1 million for the third quarter of 2021 as compared to a net loss of $50.0 million for the third quarter of 2020, which included $46.9 million resulting from the accounting treatment related to the asset acquisition of Kiq LLC. During the third quarter of 2021, the company spent $15.2 million of its cash and cash equivalents.
Cash and Cash Equivalents: As of September 30, 2021, Cogent had cash and cash equivalents of $202.9 million. The company believes that its cash and cash equivalents will be sufficient to fund its operating expenses and capital expenditure requirements into 2024.

On November 10, 2021 Greenwich LifeSciences, Inc. (Nasdaq: GLSI) (the "Company"), a clinical-stage biopharmaceutical company focused on the development of GP2, an immunotherapy to prevent breast cancer recurrences in patients who have previously undergone surgery, reported an update on the upcoming Phase III clinical trial FLAMINGO-01 (Press release, Greenwich LifeSciences, NOV 10, 2021, View Source [SID1234595049]).

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CEO Snehal Patel commented, "Our objective is to make GLSI-100 available to as many patients as possible as soon as possible. This will include opening FLAMINGO-01 in many clinical sites that are geographically situated to maximize access for patients. We continue to hear from patients interested in participating in our GLSI-100 trial and expect to be able to refer them to participating sites as appropriate. We recommend that patients or their physicians contact a participating clinical trial site near them once these sites have been opened and we communicate the participating sites to the public. We have been pleasantly surprised by the level of interest in our trial by major teaching hospitals led by breast cancer KOLs. While these sites may lead to strong enrollment, they may also lead to additional collaboration designed to optimize treatment with GLSI-100 in subsequent trials. We will be meeting with these KOLs at the upcoming in-person San Antonio Breast Cancer Symposium in December 2021 and look forward to opening the first sites and the trial soon thereafter."

The trial is titled:

"A Randomized, Multicenter, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of HER2/neu Peptide GLSI-100 (GP2 + GM-CSF) in HER2/neu Positive Subjects with Residual Disease or High-Risk PCR after both Neoadjuvant and Postoperative Adjuvant Trastuzumab-based Therapy (FLAMINGO-01)".

Vice President of Clinical and Regulatory Affairs, Dr. Jaye Thompson further commented, "The trial has been designed with an open label arm designed to provide a continual flow of data that can be publicly released, while preserving the blinding and randomization of the pivotal arms of the trial. Thus, we plan to continue to publish Phase IIb and Phase III trial data at conferences throughout the conduct of the Phase III study. The projected timeline to report the interim analysis data will depend on the rate of recurrences in both arms of the trial, but is estimated to be approximately 3 years from the time of first patient treatment. We are also making a major investment in commercial manufacturing now, which may allow for submission of a Biologics Licensing Application (BLA) to the FDA for conditional marketing approval of GLSI-100 based on the results of the interim analysis."

Design features of the FLAMINGO-01 Phase III trial include:

– The Company has added more frequent sampling and testing of patients over longer time frames and plans to utilize improved technologies to analyze immune response.

– A third open-label arm treating up to 100 patients has been added to the Phase III trial to test GLSI-100 in HLA types other than HLA-A*02 and to assess immune response and clinical outcome. This third arm will function similar to a Phase II trial, thus creating potential for early immune response data analysis and proof of concept in other HLA types, which would expand GLSI-100’s market by HLA type from 50% up to 80% or more.

– The recurrence rate data from the third arm, along with injection site reaction and immune response data from any arm across all HLA types will be available for analysis throughout the study and may provide meaningful data until the interim analysis of the recurrence rate data from the blinded HLA-A*02 arms of the Phase III trial is completed.

– In both of the blinded, randomized, placebo-controlled HLA-A*02 arms of the Phase III trial, the approximately 500 patient trial design will include an event-driven interim analysis for superiority or futility. This analysis will be conducted when approximately half of the expected breast cancer recurrences or 14 events have occurred. While a hazard ratio of HR = 0 was observed in the Phase IIb trial, a more conservative HR = 0.3 was selected for the sizing of the Phase III trial with plans in place to adaptively adjust the size of the trial as necessary.

About FLAMINGO-01 and GLSI-100

The Phase III clinical trial will be called FLAMINGO-01 and the combination of GP2 + GM-CSF will be called GLSI-100. The Phase III trial is comprised of 2 blinded, randomized, placebo-controlled arms for approximately 500 HLA-A*02 patients and 1 open label arm of up to 100 patients for all other HLA types. An interim analysis has been designed to detect a hazard ratio of 0.3 in IDFS, where 28 events will be required. An interim analysis for superiority and futility will be conducted when at least half of those events, 14, have occurred. This sample size provides 80% power if the annual rate of events in placebo-treated subjects is 2.4% or greater. The trial is currently being registered on clinicaltrials.gov and the link and trial identifier will be published shortly. For future updates about FLAMINGO-01 please visit the Company’s clinical trial tab at View Source

About Breast Cancer and HER2/neu Positivity

One in eight U.S. women will develop invasive breast cancer over her lifetime, with approximately 282,000 new breast cancer patients and 3.8 million breast cancer survivors in 2021. HER2/neu (human epidermal growth factor receptor 2) protein is a cell surface receptor protein that is expressed in a variety of common cancers, including in 75% of breast cancers at low (1+), intermediate (2+), and high (3+ or over-expressor) levels.

On November 10, 2021 Nuvalent, Inc., (Nasdaq: NUVL), a biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, reported recent pipeline progress and third quarter 2021 financial results (Press release, Nuvalent, NOV 10, 2021, View Source [SID1234595048]).

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"Throughout the third quarter of 2021, we continued to progress our novel portfolio of precisely targeted therapies for patients with cancer. Notably, our first clinical trial of NVL-520, the ‘ARROS-1’ study, is now open for enrollment of patients with advanced ROS1-positive NSCLC and other solid tumors," said James Porter, Ph.D., Chief Executive Officer at Nuvalent. "We anticipate a robust set of upcoming operational milestones, including the dosing of the first patient in our ARROS-1 study in 2021, the advancement of our parallel lead program NVL-655 into clinical development for ALK-positive cancers, and the expansion of our portfolio with additional internally developed product candidates. With a dedicated, expert team and a strong balance sheet in place, we believe we are well-positioned to achieve the milestones ahead."

Recent Program Highlights

ARROS-1 Phase 1/2 Clinical Trial of NVL-520 Open for Enrollment:
Nuvalent has activated multiple U.S. sites to begin enrollment in its ARROS-1 clinical trial, a Phase 1/2, multicenter, open-label, dose-escalation and expansion study evaluating NVL-520 as an oral monotherapy in patients with advanced ROS1-positive non-small cell lung cancer (NSCLC) and other solid tumors. NVL-520 is a novel ROS1-selective inhibitor designed to address the clinical challenges of emergent treatment resistance, central nervous system (CNS)-related adverse events, and brain metastases that may limit the use of currently available ROS1 tyrosine kinase inhibitors (TKIs). The Phase 2 portion of the ARROS-1 study is designed to support potential registration of NVL-520 in both ROS1-positive patients with NSCLC who are kinase inhibitor-naïve and who have been previously treated with ROS1 kinase inhibitors. Clinical site expansion in the US and EU is ongoing.
Preclinical Data Supporting Lead Programs for ROS1-Positive and ALK-Positive NSCLC Presented at 2021 AACR (Free AACR Whitepaper)-NCI-EORTC Molecular Targets and Cancer Therapeutics Conference: Nuvalent presented new preclinical data at the 2021 AACR (Free AACR Whitepaper)-NCI-EORTC Molecular Targets and Cancer Therapeutics Conference further demonstrating that NVL-520 and NVL-655, its parallel lead product candidates, were active against both wild-type and various known resistance variants of ROS1 or ALK, respectively; were brain-penetrant with the potential to address brain metastases; and selectively inhibited their respective targets compared to the structurally related tropomyosin receptor kinase B (TRKB), thereby minimizing the potential for off-target TRKB-related CNS adverse events. The ARROS-1 Phase 1/2 clinical trial of NVL-520 is open for enrollment, and Nuvalent plans to initiate a Phase 1/2 clinical trial of NVL-655 in advanced ALK-positive NSCLC and other cancers during the first half of 2022.
Upcoming Investor Conference Presentations

33rd Annual Virtual Piper Healthcare Conference: Dr. Porter will participate in a pre-recorded fireside chat during the 33rd Annual Virtual Piper Healthcare Conference, being held November 30 – December 2, 2021. The fireside chat will be available to registered participants beginning on November 22, 2021, and the webcast will be available in the investor section of the company’s website at www.nuvalent.com for 30 days following the presentation.
Third Quarter 2021 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $302.4 million as of September 30, 2021, including net proceeds from the initial public offering completed on August 2, 2021, compared to $10.3 million as of December 31, 2020.
Research & Development (R&D) expenses: R&D expenses were $9.1 million for the third quarter of 2021, compared to $3.7 million for the third quarter of 2020.
General & Administrative (G&A) expenses: G&A expenses were $3.4 million for the third quarter of 2021, compared to $0.3 million for the third quarter of 2020.
Net Loss: Net loss for the third quarter of 2021 was $12.4 million, or $0.39 per share, compared to $4.8 million, or $1.57 for the third quarter of 2020.

On November 10, 2021 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported financial results and business highlights for the third quarter of 2021 (Press release, Hookipa Pharma, NOV 10, 2021, View Source [SID1234595047]).

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"Our team remains focused on optimizing the potential of our versatile arenaviral platform to redefine success in cancer immunotherapy by driving strong tumor-specific T cell responses. Third quarter highlights include our clinical collaboration with Merck & Co., Inc., Kenilworth, NJ, USA, on our HB-200 program in head and neck cancer and the publication of our pre-clinical data in melanoma in Nature Communications, both of which offered external validation of the promise of our technology," said Joern Aldag, Chief Executive Officer at HOOKIPA. "Given our recent HB-200 data update, we’re even more energized about the start of our Phase 2 combination study of HB-201 with pembrolizumab as a 1st- or 2nd-line treatment in head and neck cancers, as well as progressing and expanding our oncology pipeline with candidates for prostate and KRAS-mutated cancers."

Quarter Highlights

In August, pre-clinical data on HOOKIPA’s arenaviral immunotherapeutic in melanoma was published in the peer-reviewed journal, Nature Communications. The data showed that HOOKIPA’s replicating Lymphocytic choriomeningitis (LCMV)-based vector, designed to target melanoma, modulated the tumor micro-environment and induced potent, antigen-specific T cell responses, resulting in tumor regression and tumor eradication in the pre-clinical setting. The data reinforce the potential of HOOKIPA’s arenaviral immunotherapeutic technology to activate and mobilize anti-tumor T cells for effective control and eradication of established tumors.

In September, HOOKIPA announced a clinical collaboration and supply agreement with Merck & Co, Inc., Kenilworth, NJ, USA (known as MSD outside of the United States and Canada) to evaluate the combination of HB-200, a novel arenaviral immunotherapeutic, and KEYTRUDA (pembrolizumab) as 1st-line treatment for patients with advanced head and neck cancer.
Business updates

On November 9, 2021 HOOKIPA provided a data update across its clinical development program. HOOKIPA announced it is advancing HB-201 to Phase 2, to be evaluated in combination with pembrolizumab as 1st- or 2nd-line treatment for Human Papillomavirus 16 Positive (HPV16+) squamous cell head and neck cancers (HNSCC). Interim Phase 1 data in heavily pre-treated patients continue to show HB-200 monotherapy (both HB-201 alone and HB-202/HB-201) is highly effective at expanding T cells, has a favorable tolerability profile and promising, early anti-tumor activity. As of November 1, 2021, among 28 patients dosed intravenously, HB-200 resulted in a 75 percent disease control rate and shrinkage of target lesions in 53 percent of patients. In these patients, HOOKIPA has observed three partial responses (including one confirmed and one unconfirmed in an ongoing patient) and one ongoing patient with a near partial response (29 percent tumor shrinkage). Based on the strength of the HB-200 data, HOOKIPA has prioritized its oncology portfolio, including HB-300 for prostate cancer and HB-700 for KRAS-mutated cancers, and plans further development of its infectious disease programs to be done in partnership with other companies.

Upcoming Milestones

Phase 1 HB-200 HPV16+ HNSCC additional data: mid-year 2022
Phase 2 HB-201 + pembrolizumab HPV16+ HNSCC 1st-line initial data: second half of 2022
Phase 2 HB-201 + pembrolizumab HPV16+ HNSCC 2nd-line initial data: second half of 2022
Randomized Phase 2 HB-200 + pembrolizumab HPV16+ HNSCC 1st-line trial initiation: first half of 2023
Investigational New Drug application for HB-300 in metastatic prostate cancer: third quarter 2022
Third Quarter 2021 Financial Results

Cash Position: HOOKIPA’s cash, cash equivalents and restricted cash as of September 30, 2021 was $82.7 million compared to $143.2 million as of December 31, 2020. The decrease was primarily attributable to cash used in operating activities.

Revenue was $3.9 million for the three months ended September 30, 2021, and $4.0 million for the three months ended September 30, 2020. Revenues did not materially change compared to the three months ended September 30, 2020 though they included lower deferred revenues from upfront and milestone payments which were partially offset by higher cost reimbursements.

Research and Development Expenses: HOOKIPA’s research and development expenses were $20.7 million for the three months ended September 30, 2021, compared to $16.0 million for the three months ended September 30, 2020.

The primary drivers of the increase in direct research and development expenses were an increase in manufacturing and quality control expenses of $2.1 million, along with a general increase in other direct research and development expenses and laboratory expenses of $1.6 million, partially offset by a decrease in expenses for clinical studies of $0.6 million. The increase in manufacturing and quality control expenses as well as other direct research and development expenses was mainly due to the progress in HOOKIPA’s HB-201 and HB-202 clinical trial, in particular for monitoring and testing activities, and manufacturing and quality control work in preparation of a further extension of the trial. Clinical study expenses decreased primarily due to the completion of patient enrollment of the Phase 2 study for our CMV vaccine candidate HB-101.

Internal research and development expenses increased by $1.6 million, mainly due to HOOKIPA’s increased research and development headcount.

General and Administrative Expenses: General and administrative expenses for the three months ended September 30, 2021 were $4.3 million, compared to $4.4 million for the three months ended September 30, 2020. The decrease was primarily due to a decrease in personnel-related expenses and a decrease in professional and consulting fees. The decrease in personnel-related expenses resulted from decreased stock compensation expenses, partially offset by a growth in headcount along with increased salaries in our general and administrative functions.

Net Loss: HOOKIPA’s net loss was $20.0 million for the three months ended September 30, 2021, compared to a net loss of $13.6 million for the three months ended September 30, 2020. The increase was primarily due to an increase in HOOKIPA’s research and development activities.