Replimune Reports Fiscal Fourth Quarter and Year-Ended 2021 Financial Results and Provides Corporate Update

On May 20, 2021 Replimune Group, Inc. (NASDAQ: REPL), a biotechnology company developing a series of oncolytic immuno-gene therapies derived from its Immulytic platform, reported financial results for the fiscal fourth quarter and year ended March 31, 2021 and provided a business update (Press release, Replimune, MAY 20, 2021, View Source [SID1234580379]).

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"We have made good progress advancing our programs over the quarter and look forward to providing a data update with RP1 and RP2 at our virtual investor event being held in June as we continue to enroll into our registration-directed clinical trials in CSCC and anti-PD1 failed melanoma, with expected readouts in 2022," said Philip Astley-Sparke, CEO of Replimune. "Our vision extends well beyond our initial lead skin cancer indications, with the broader aim of becoming a cornerstone for immuno-oncology treatments across a wide spectrum of tumor types. With this in mind, and as we move closer to market, we recently expanded our senior management team with two key hires with a considerable track record of success in bringing multiple high profile programs to BLA approval and in overseeing the launch of new drugs in the immuno-oncology space."

Corporate Updates

Expanded management team with key hires to prepare for the transition to a commercial company. The Company hired Sushil Patel, Ph.D. as Chief Commercial Officer, arriving from Genentech where he was the head of their global oncology franchise for lung cancer, skin cancer and rare / agnostic tumor types, and previously the lifecycle leader in lung cancer for the multi-billion-dollar checkpoint blockade drug Tecentriq. In addition to hiring Dr. Patel, the Company also appointed Tanya Lewis as Chief Development Operations Officer. Ms. Lewis’s past accomplishments include successful negotiations related to registration trial designs, approval, and/or commercialization of XPOVIO, VELCADE, VARUBI, INTEGRILIN and ZEJULA.

Announced plans to amend the CERPASS clinical trial protocol to add complete response (CR) rate as an additional independent primary endpoint. The amendment is based on the depth and durability of responses and the manageable safety profile seen in patients with non-melanoma skin cancers treated with RP1 in combination with Opdivo to date. Under the modified clinical trial protocol for CERPASS, Replimune plans to add CR rate as an additional independent primary endpoint, in addition to overall response rate (ORR), and to reduce target enrollment from 240 patients to 180 patients. Secondary endpoints will continue to include duration of response, progression-free survival (PFS), and overall survival (OS). Replimune plans to submit the amended protocol to the FDA by the end of this quarter and is maintaining its guidance to expect primary data read out in 2022.

Held Type B meeting with the FDA to discuss the regulatory pathway for RP1 in combination with Opdivo (nivolumab) in anti-PD1 failed melanoma. Replimune recently held a Type B meeting with the FDA to discuss the design of the currently enrolling 125-patient registration-directed cohort of patients with anti-PD1 failed melanoma in the IGNYTE clinical trial. The FDA expressed that while a randomized controlled clinical trial would always be preferred for registration, if the clinical data is sufficiently compelling in this patient population with no clear standard of care, then the data could be submitted to the FDA for review under the accelerated approval pathway. The FDA also indicated that a randomized confirmatory trial would be needed as is required under the accelerated approval process.

Presented new biomarker and pre-clinical data for RP1 and RP2, at the 2021 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The data presented continues to confirm potent anti-tumor activity and activation of robust systemic immune activation by RP1 and RP2.

Virtual Investor Event to be held on Thursday, June 3, 2021. The Company will host a virtual investor event to present updated data from its Phase 2 skin cancer cohorts combining RP1 with Opdivo and data from its Phase 1 study of RP2 alone and in combination with Opdivo. The event will include presentations by the management team and Mark Middleton, Professor of Experimental Cancer Medicine in the Department of Oncology, consultant Medical Oncologist at the Oxford Cancer and Hematology Centre and Head of the Department of Oncology at the University of Oxford.

First RP1 batches produced and filled at state-of-the-art manufacturing facility. Release testing is underway for the first GMP RP1 batches produced at its 63,000-square-foot manufacturing facility in Framingham, MA, that was built to support the commercialization of all of its products. Technology transfer for RP2 has also commenced and is expected to complete in the next quarter.
Program Highlights and Upcoming Milestones

RP1 in combination with Libtayo (cemiplimab) in CSCC: CERPASS, the Company’s Phase 2, global, randomized, controlled, registration-directed clinical trial continues to actively enroll patients and remains on track for a primary data read out in 2022.

RP1 in combination with Opdivo in anti-PD-1 failed melanoma: The Company’s 125-patient cohort in the IGNYTE Phase 2 clinical trial of RP1 in combination with Opdivo, continues to actively enroll patients and remains on track to report the primary data read out in 2022.

RP1 in combination with Opdivo in melanoma and non-melanoma skin cancers (NMSC): Following a positive Phase 2 data update in October 2020 and enrollment of the initial melanoma cohort (including anti-PD1 naïve and anti-PD1 failed patients) being complete, the Company continues to enroll its 45-patient cohort evaluating RP1 in combination with Opdivo in NMSC. The Company plans to provide an update on these programs at its June 3rd investor event.

RP1 in anti-PD1 failed NSCLC: The first patient in the cohort of 30 anti-PD1 failed NSCLC patients treated with RP1 combined with Opdivo has been dosed, and the Company expects to report initial data in the second half of 2021.

RP1 as monotherapy in solid organ transplant recipients with CSCC: The Company is currently enrolling a 30 patient Phase 1b clinical trial (ARTACUS) assessing the safety and efficacy of RP1 in liver and kidney transplant recipients with CSCC. Enrollment in this immuno-compromised population has been hampered by COVID 19, but as the effects of the pandemic reduce the Company expects recruitment to increase. Early data from this clinical trial is intended to be presented in the second half of 2021.

RP1 in combination with Opdivo in MSI-H/dMMR tumors: The Company continues to expect to be able to decide whether to pursue RP1 for MSI-H/dMMR tumors into registration-directed development by the end of 2021.

RP2 alone and in combination with Opdivo: Following positive data with RP2 given as monotherapy that were presented in October 2020, the Company is actively enrolling a 30-patient cohort evaluating RP2 in combination with Opdivo. Updated data from this clinical trial, including an update on patients treated with RP2 monotherapy and initial data with RP2 in combination with Opdivo will be presented at the upcoming June 3rd investor event.

RP3 alone and in combination with anti-PD-1 therapy: The Phase 1 clinical trial evaluating RP3 alone and in combination with anti-PD1 therapy in solid tumor patients is actively enrolling and the Company remains on track to report initial data in the second half of 2021.

Target evaluation for new indications is currently underway: The Company remains on track to disclose part of its initial development plans for RP2 and/or RP3 in less immune responsive tumor types at the upcoming June 3rd investor event.
Financial Fiscal Quarter Four and Year End Highlights:

Cash Position: As of March 31, 2021, cash, cash equivalents and short-term investments were $476.3 million, as compared to $168.6 million as of March 31, 2020. This increase was primarily related to $372.5 million in net proceeds from financing activities offset by an increase in cash utilized in operating activities in advancing our expanded clinical development plan.

Based on the current operating plan, Replimune believes that existing cash and cash equivalents and short-term investments will fund operating expenses and capital expenditure requirements into the second half of 2024.
R&D Expenses: Research and development expenses were $16.2 million for the fourth quarter and $56.8 million for the fiscal year ended March 31, 2021, as compared to $11.2 million for the fourth quarter and $38.8 million for the fiscal year ended March 31, 2020. This increase was primarily due to clinical expenses driven by the company’s lead programs, expansion into additional studies, operating our dedicated manufacturing facility and related increased personnel costs. Research and development expenses included $2.0 million in stock-based compensation expenses for the fourth quarter and $5.7 million in stock-based compensation expenses for the fiscal year ended March 31, 2021.

G&A Expenses: General and administrative expenses were $6.0 million for the fourth quarter and $23.2 million for the fiscal year ended March 31, 2021, as compared to $5.2 million for the fourth quarter and $17.4 million for the year ended March 31, 2020. The increase was primarily driven by personnel related costs, professional fees, and facility expansion. General and administrative expenses included $1.5 million in stock-based compensation expenses for the fourth quarter and $6.0 million in stock-based compensation expenses for the fiscal year ended March 31, 2021.

Net Loss: Net loss was $21.5 million for the fourth quarter and $80.9 million for the fiscal year ended March 31, 2021, as compared to a net loss of $15.8 million for the fourth quarter and $52.6 million for the fiscal year ended March 31, 2020.
About CERPASS
CERPASS is Replimune’s registration-directed randomized, global Phase 2 clinical study to compare the effects of Libtayo alone versus a combination of Libtayo and Replimune’s investigational oncolytic immunotherapy RP1. The clinical trial will enroll 180 patients with locally advanced or metastatic cutaneous squamous cell carcinoma (CSCC) who are naïve to anti-PD1 therapy. The trial will evaluate complete response (CR) rate and overall response rate (ORR) as its two primary efficacy endpoints as assessed by independent review, as well as duration of response, progression-free survival (PFS), and overall survival (OS) as its secondary endpoints. The study is being run under a clinical trial collaboration agreement with Regeneron in which the costs of the trial are shared and full commercial rights retained by Replimune. Libtayo is being jointly developed by Regeneron and Sanofi.
Libtayo is a registered trademark of Regeneron.

About IGNYTE
IGNYTE is Replimune’s multi-cohort Phase 1/2 trial of RP1 plus Opdivo. There are 4 tumor specific cohorts currently enrolling in this trial including a 125-patient extension cohort of RP1 combined with Opdivo in anti-PD-1 failed cutaneous melanoma. This cohort was initiated after completing enrollment in a prior Phase 2 cohort in the same trial of approximately 30 patients with melanoma. The additional thirty patient cohorts are studying RP1 in combination with Opdivo in non-melanoma skin cancers which includes both naïve and anti-PD-1 failed CSCC, in microsatellite instability high, or MSI-H/dMMR tumor types and anti-PD-1 failed non-small cell lung cancer, or NSCLC. This trial is being done under a collaboration and supply agreement with Bristol Myer Squibb.
Opdivo is a registered trademark of Bristol-Myers Squibb Company.

About RP1
RP1 is Replimune’s lead Immulytic product candidate and is based on a proprietary new strain of herpes simplex virus engineered to maximize tumor killing potency, the immunogenicity of tumor cell death and the activation of a systemic anti-tumor immune response.

About RP2 & RP3
RP2 and RP3 are derivatives of RP1 that express additional proteins. RP2 expresses an anti-CTLA-4 antibody-like molecule and RP3 additionally expresses the immune co-stimulatory pathway activating proteins CD40L and 4-1BBL. RP2 and RP3 are intended to provide targeted and potent delivery to the sites of immune response initiation in the tumor and draining lymph nodes, with the goal of focusing systemic immune-based efficacy on tumors and limiting off-target toxicity.

Pieris Pharmaceuticals to Present at the Jefferies 2021 Virtual Healthcare Conference

On May 20, 2021 Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer, and other indications, reported that management is scheduled to participate in a fireside chat at the Jefferies 2021 Virtual Healthcare Conference on Friday, June 4, 2021 at 1:00 PM EDT (Press release, Pieris Pharmaceuticals, MAY 20, 2021, View Source [SID1234580378]). A webcast of the company’s presentation will be available at this link.

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NeuBase Therapeutics Appoints Dr. Sandra Rojas-Caro Chief Medical Officer

On May 20, 2021 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company accelerating the genetic revolution using a new class of precision genetic medicines, reported the appointment of Sandra Rojas-Caro, M.D., as Chief Medical Officer, effective May 24, 2021 (Press release, NeuBase Therapeutics, MAY 20, 2021, View Source [SID1234580377]). With more than 20 years of drug development experience across all phases of drug development, Dr. Rojas-Caro will oversee the preclinical and clinical development, medical, and regulatory strategy of NeuBase’s pipeline to address disease at its base cause.

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"Sandra is a dynamic and experienced R&D leader, whose expertise covers all aspects of clinical development, including the successful advancement of a novel therapeutic from IND to FDA approval," said Dietrich A. Stephan, Ph.D., Founder, CEO and Chairman of NeuBase. "Furthermore, she’s been responsible for dozens of clinical studies in multiple therapeutic modalities and indications from rare to common diseases. Now is an important time to welcome Sandra to the executive leadership team as we look to advance our first drug candidate into clinical trials next year and expand our pipeline to address historically undruggable oncogenic driver mutations."

"I’m impressed by NeuBase’s unique, targeted therapeutic approach to precision genetic medicines, which is designed to fundamentally address disease-causing mutations at the DNA or RNA level," said Dr. Rojas-Caro. "I am excited to join NeuBase’s exceptional leadership and scientific team, which is comprised of a number of founders and leading innovators in precision medicine. I look forward to applying NeuBase’s PATrOLTM platform, which has broad potential to address a wide range of rare and common genetically defined diseases."

Dr. Rojas-Caro has broad R&D leadership, executive management and team-building experience in private and public biotech companies and large pharma. She has been directly involved in successful global regulatory submissions, including an FDA and EMA approval and more than 10 investigational new drug (IND) applications. Most recently, she was Chief Medical Officer for Gemini Therapeutics, a company focused on redefining age-related macular degeneration (AMD) and linked disorders with precision medicine. At Gemini, she led development through several milestones, including the company’s first IND and the first cohorts of genetically selected patients dosed with the company’s leading biologic therapeutic. Prior to Gemini, Sandra served as Chief Medical Officer for Aeglea BioTherapeutics, a biotechnology company developing a new generation of enzyme-based therapeutics to treat inborn errors of metabolism (IEM). Prior to Aeglea, she served as Group Vice President of Clinical Research and Development at Synageva BioPharma where she was instrumental in leading the clinical development team that secured the FDA and EMA approval of Kanuma (sebelipase alfa) for the treatment of lysosomal acid lipase deficiency, as well as advancing the clinical development of other IEM programs. Following the acquisition of Synageva by Alexion, Dr. Rojas-Caro served as Vice President and R&D Project Team Leader for the Metabolic Rare Diseases Unit, and she supported the post-merger integration. Earlier in her career, she held roles in clinical and translational research with increasing levels of responsibility at Roche, Array BioPharma and Pfizer, where she was responsible for the design and implementation of early development clinical strategy across a broad range of indications.

Purple Biotech to Present New Clinical Data from NT219 at the 2021 ASCO Annual Meeting

On May 20, 2021 Purple Biotech Ltd. ("Purple Biotech", or the "Company") (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class, effective and durable therapies by overcoming tumor immune evasion and drug resistance, reported that it will present new data from the ongoing Phase 1/2 clinical trial of NT219 on Friday, June 4, 2021, at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting, which will be held virtually from June 4-8 (Press release, Purple Biotech, MAY 20, 2021, View Source [SID1234580376]).

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NT219 is a dual inhibitor, novel small molecule that simultaneously targets IRS1/2 and STAT3. The Phase 1/2 trial is evaluating NT219 as monotherapy treatment of advanced solid tumors, as well as in combination with cetuximab, an epithelial growth factor receptor (EGFR) blocking monoclonal antibody, for the treatment of recurrent and/or metastatic solid tumors and head and neck cancer or colorectal adenocarcinoma.

"We are excited to present interim data from our ongoing Phase 1/2 clinical trial of NT219 as monotherapy for the treatment of solid tumors that has the potential to help people living with hard-to-treat cancers," said Bertrand Liang, M.D., Ph.D., Chief Medical Officer of Purple Biotech. "We are excited to have clinical data for our NT219 program and we continue to expect the availability of further top-line data from the first part of this study in the second half of this year."

Details of the presentation are as follows:

Title: A Phase 1/2 study with open-label, dose escalation phase followed by single-arm expansion at the maximum tolerated dose to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of NT219 injection alone and in combination with Cetuximab in Adults with Advanced Solid Tumors and Head and Neck Cancer.

Session: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology

Location: ASCO (Free ASCO Whitepaper) Meeting Library

VBL Therapeutics to Provide an Update on the OVAL Phase 3 Registration Enabling Study of VB-111 in Ovarian Cancer at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting

On May 20, 2021 VBL Therapeutics (Nasdaq: VBLT) reported that a poster providing an update on the OVAL Phase 3 registration enabling clinical trial of VB-111 in ovarian cancer will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, VBL Therapeutics, MAY 20, 2021, View Source [SID1234580375]).

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"We are encouraged by the continued progress of the OVAL clinical trial, which remains on track," said Dror Harats, M.D., CEO of VBL Therapeutics. "The poster will provide an important update on the trial."

Title: Clinical Trial in Progress: Pivotal Study of VB-111 Combined with Paclitaxel vs. Paclitaxel for Treatment of Platinum-Resistant Ovarian Cancer (OVAL, VB-111-701/GOG-3018)
Authors: Arend, R.C., et al.
Session: Gynecologic Cancer
Session type: Poster Session
Abstract: 5599

About the OVAL study (NCT03398655)
OVAL is an international Phase 3 randomized pivotal registration enabling clinical trial that compares a combination of VB-111 and paclitaxel to placebo plus paclitaxel, in patients with platinum resistant ovarian cancer. The study is planned to enroll approximately 400 patients. OVAL is conducted in collaboration with the GOG Foundation, Inc., an independent international non-profit organization with the purpose of promoting excellence in the field of gynecologic malignancies.

About VB-111 (ofranergene obadenovec)
VB-111 is an anti-cancer gene-therapy agent that is being developed to treat a wide range of solid tumors. VB-111 is a unique biologic agent that uses a dual mechanism to target solid tumors. Its mechanism combines blockade of tumor vasculature with an anti-tumor immune response. VB-111 is administered as an IV infusion once every 6-8 weeks. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in an "all comers" Phase 1 trial as well as in three tumor-specific Phase 2 studies. VB-111 has received an Orphan Designation for the treatment of ovarian cancer from the European Commission. VB-111 has also received orphan drug designation in both the US and Europe, and fast track designation in the US, for prolongation of survival in patients with recurrent glioblastoma. VB-111 demonstrated proof-of-concept and survival benefit in Phase 2 clinical trials in radioiodine-refractory thyroid cancer and recurrent platinum-resistant ovarian cancer (NCT01711970).