On November 4, 2021 Replimune Group, Inc. (NASDAQ: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, reported financial results for the fiscal second quarter ended September 30, 2021 and provided a business update (Press release, Replimune, NOV 4, 2021, View Source [SID1234594494]).

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"Replimune continues to build towards a data rich 2022 where we are expecting to reach multiple inflection points culminating in the primary data analysis trigger for our registration-directed randomized controlled clinical trial with RP1 in cutaneous squamous cell carcinoma (CSCC) and the presentation of interim data from our registration directed cohort in anti-PD1 failed melanoma from the IGNYTE clinical trial," said Philip Astley-Sparke, CEO of Replimune. "In the first quarter of 2022 we will be presenting initial data in anti-PD1 failed non-melanoma skin cancers (NMSC) and in NMSC transplant patients with single agent RP1, both including CSCC. Our objective is to establish a broad skin cancer franchise with RP1. In addition to an update on RP2 at SITC (Free SITC Whitepaper) 2021, we look forward to presenting data during 2022 from the expansion of our Phase 1 studies with RP2 and RP3 with a focus on patients with liver metastases from prevalent tumor types. We also look forward to presenting a comprehensive Phase 2 development plan with RP2 and/or 3 in the first quarter of 2022, with study initiation intended around midyear, as we continue to make progress towards positioning our programs as a cornerstone of cancer treatment."

Recent Events and Corporate Updates

Replimune to present three ‘Trial-In-Progress’ posters and a poster including new data with RP2 at the 2021 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 36th Annual Meeting. The Company is scheduled to present four posters at SITC (Free SITC Whitepaper) being held November 10-14, 2021. Included in these posters will be a data update from its 30-patient Phase 1 clinical trial evaluating RP2 alone and combined with Opdivo in difficult-to-treat cancers.
Program Highlights*

CERPASS – Registration directed Phase 2 clinical trial in CSCC

RP1 in combination with Libtayo (cemiplimab) in CSCC: The Company is actively enrolling patients in CERPASS, its registration directed, global, randomized controlled Phase 2 clinical trial of RP1 in combination with Libtayo vs. Libtayo alone in 180 patients with advanced CSCC. The Company expects to complete enrollment such that the primary data analysis is expected to be triggered in late 2022.
IGNYTE – multi cohort Phase 2 clinical trial of RP1 combined with Opdivo

Anti-PD-1 failed melanoma cohort: The Company continues to enroll patients in the 125-patient cohort of the IGNYTE Phase 2 clinical trial in patients with anti-PD1 failed melanoma. The company continues to expect to release interim data from this cohort in late 2022 but, as previously disclosed, in order to document sufficient durability of response, an important secondary endpoint of the study, the primary analysis is expected to be triggered in mid-year 2023.
Non-melanoma skin cancer (NMSC) cohort: The Company has completed enrollment of 30-patients with anti-PD-1 naïve NMSC and continues to enroll patients with anti-PD-1 failed NMSC. The Company remains on track to provide updated data from the anti-PD1 naïve patients and initial data from the anti-PD-1 failed patients in the first quarter of 2022.
Anti-PD(L)-1 failed non-small cell lung cancer (NSCLC) cohort: Dosing is underway in a 30-patient cohort of RP1 in combination with Opdivo in anti-PD(L)-1 failed NSCLC. A recently filed amendment to the IGNYTE protocol includes modifications to the patient eligibility criteria which are expected to enhance enrollment into this cohort of the clinical trial. The Company had planned to provide initial data from this cohort in the first quarter of 2022. However, the changes made to facilitate enrollment are not expected to take effect in time to meet prior guidance and initial data is now expected to be released later in 2022.
ARTACUS – Phase 1b/2 clinical trial of RP1 as monotherapy in solid organ transplant recipients with skin cancers

The Company continues to enroll patients in this 65-patient clinical trial, with potential registrational intent, assessing the safety and efficacy of RP1 in liver and kidney transplant recipients with skin cancers, including CSCC. The Company remains on track to present initial data from this clinical trial in the first quarter of 2022.
RP2 and RP3

RP2 alone and in combination with Opdivo in difficult-to-treat cancers: The Company remains on track to provide a data update from the Phase 1 clinical trial in patients treated as monotherapy with RP2 and RP2 combined with Opdivo at SITC (Free SITC Whitepaper) in the fourth quarter of 2021. The Company has filed an amendment to expand this trial to, in particular, provide data in patients with liver metastases from various prevalent tumor types with a focus on patients with lung, breast and gastrointestinal cancers including colorectal cancer.
RP3 alone and in combination with anti-PD-1 therapy: The Company is enrolling patients in its Phase 1 clinical trial with RP3, with initial monotherapy data expected to be presented in the first quarter of 2022. The Company expects to start enrolling patients to be treated with RP3 in combination with anti-PD-1 therapy early in the new year, including patients with liver metastases from various prevalent tumor types such as lung, breast, head and neck cancer and gastrointestinal cancers including colorectal cancer.
RP2 and/or RP3 next stage development with focus on patients with liver metastases from a range of tumor types: The Company remains on track to initiate a broad clinical development program with RP2 and/or RP3, intended to include a range of prevalent tumor types with focus on patients with liver metastases, around mid-year 2022. Details of this plan are intended to be presented in the first quarter of 2022.

*Program Highlight dates are on a calendar-year basis.
Financial Highlights

Cash Position: As of September 30, 2021, cash, cash equivalents and short-term investments were $435.8 million, as compared to $476.3 million as of March 31, 2021. This decrease was primarily related to cash utilized in operating activities in advancing the Company’s 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, excluding any confirmatory trial required by the FDA or other regulatory body.
R&D Expenses: Research and development expenses were $19.9 million for the second quarter ended September 30, 2021, as compared to $14.1 million for the second quarter ended September 30, 2020. This increase was primarily due to increased clinical and manufacturing expenses driven by the Company’s lead programs and increased personnel expenses. Research and development expenses included $2.2 million in stock-based compensation expenses for the second quarter ended September 30, 2021.

G&A Expenses: General and administrative expenses were $9.3 million for the second quarter ended September 30, 2021, as compared to $5.6 million for the second quarter ended September 30, 2020. The increase was primarily driven by personnel-related costs, professional fees, and facility expansion. General and administrative expenses included $4.1 million in stock-based compensation expenses for the second quarter ended September 30, 2021.

Net Loss: Net loss was $29.4 million for the second quarter ended September 30, 2021, as compared to a net loss of $20.1 million for the second quarter ended September 30, 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-PD-1 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 conducted 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 cohort 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 cohorts are in non-melanoma skin cancers which includes both naïve and anti-PD-1 failed CSCC, in anti-PD1 failed microsatellite instability high, or MSI-H/dMMR tumors and anti-PD(L)-1 failed non-small cell lung cancer, or NSCLC. This trial is being conducted under a collaboration and supply agreement with Bristol-Myers Squibb Company.
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.

On November 4, 2021Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported financial results for the third quarter ended September 30, 2021 and provided a business update (Press release, Protara Therapeutics, NOV 4, 2021, View Source [SID1234594493]).

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"We have made significant progress advancing our TARA-002 clinical programs in the third quarter, most notably the completion of our confirmatory large-scale GMP comparability work and the clearance of our Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for our non-muscle invasive bladder cancer (NMIBC) clinical development program. NMIBC is one of the most recurrent and difficult to treat cancers with limited treatment options, and we look forward to initiating our Phase 1 clinical trial of TARA-002 in adults with high-grade NMIBC by the end of the year," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We are also working to align with the FDA on the design of a clinical trial of TARA-002 in patients with lymphatic malformations (LMs), a rare pediatric indication for which there are currently no U.S. FDA-approved therapies."

Recent Highlights and Upcoming Milestones

TARA-002 in NMIBC

In October 2021, Protara announced that the FDA cleared the Company’s IND application for TARA-002, an investigational cell-based therapy in development for the treatment of NMIBC. The confirmatory, GMP-scale comparability data for TARA-002 in relation to OK-432, the originator therapy for TARA-002, have been completed and were reviewed by the FDA as part of the clearance of the IND.
The Company plans to commence a Phase 1 clinical trial by the end of 2021 to assess the safety, tolerability, and preliminary signs of anti-tumor activity of TARA-002 in adults with high-grade NMIBC.
TARA-002 in LMs

In October 2021, the Company updated its IND submission for TARA-002 for the treatment of LMs with completed confirmatory, GMP-scale comparability data, and plans to engage the FDA on the design, and subsequently initiate a clinical trial in pediatric LM patients.
IV Choline Chloride in Intestinal Failure Associated Liver Disease (IFALD)

In September 2021, the Company announced the completion of a retrospective prevalence study designed to enhance understanding of the incidence of IFALD in patients dependent on parenteral nutrition (PN). The study found that approximately 30% of patients who are dependent on PN have cholestasis, despite the use of medical management in these patients.
The Company plans to use the results from the completed retrospective study and ongoing prospective study to inform next steps for the IV Choline Chloride development program.
Corporate Update

In October 2021, the Company hired Mary Grendell as its new General Counsel. Ms. Grendell has an extensive track record as a legal executive, most recently serving as Vice President, Deputy General Counsel and Corporate Secretary, at Intercept Pharmaceuticals. Previously, she held positions at Mylan (now part of Viatris) and Amgen following her early legal career at Sullivan & Cromwell and Covington & Burling. Ms. Grendell received her J.D. from the University of Pennsylvania Law School and her B.A. from Yale University.
Third Quarter 2021 Financial Results

As of September 30, 2021, cash, cash equivalents and marketable debt securities totaled $138.4 million.

Research and development expenses for the third quarter of 2021 increased to $4.1 million from $2.8 million during the third quarter of 2020. The increased R&D expenses were primarily due to increases in non-clinical, clinical and regulatory expenses associated with TARA-002, headcount and stock-based compensation, and other employee-related expenses.

General and administrative expenses for the third quarter of 2021 increased to $6.7 million from $5.3 million during the third quarter of 2020. The increase was primarily due to increases in headcount and employee-related expenses, development of commercial capabilities, and costs associated with the new corporate office in New York, NY.

For the third quarter of 2021, Protara reported a net loss of $10.8 million, or $0.96 per share, compared with a net loss of $8.0 million, or $1.26 per share, for the third quarter of 2020. Net loss for the third quarter of 2021 included approximately $2.7 million of stock-based compensation expenses.
About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and LMs for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan and Taiwan by Chugai Pharmaceutical Co., Ltd. Protara has successfully demonstrated manufacturing comparability between TARA-002 and OK-432.

When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a strong immune cascade. Neutrophils, monocytes and lymphocytes infiltrate the abnormal cells and various cytokines, including interleukins IL-6, IL-8, IL-12, interferon (IFN)-gamma, tumor necrosis factor (TNF)-alpha, and vascular endothelial growth factor (VEGF) are secreted by immune cells to induce a strong local inflammatory reaction and destroy the abnormal cells.

About Non-Muscle Invasive Bladder Cancer

Bladder cancer is the 6th most common cancer in the United States, with NMIBC representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle.

About Lymphatic Malformations

LMs are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Most LMs are present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of 3 years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels, lymphatics; recurrent infection, and cosmetic and other functional disabilities.

About IV Choline Chloride and Intestinal Failure-associated Liver Disease (IFALD)

IV Choline Chloride is an investigational, intravenous (IV) phospholipid substrate replacement therapy initially in development for patients receiving PN who have IFALD. Choline is a known important substrate for phospholipids that are critical for healthy liver function. Because PN patients cannot sufficiently absorb adequate levels of choline and no available PN formulations contain sufficient amounts of choline to correct this deficiency, PN patients often experience a prolonged progression to hepatic failure and death, with the only known intervention being a dual small bowel/liver transplant. If approved, IV Choline Chloride would be the first approved therapy for IFALD. It has been granted Orphan Drug Designations (ODDs) by the FDA for the treatment of IFALD and the prevention of choline deficiency in PN patients.

On November 4, 2021 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, reported that the Company intends to release its third quarter 2021 financial results on Thursday, November 11, 2021 (Press release, Onconova, NOV 4, 2021, View Source [SID1234594492]). Management plans to host a conference call and live webcast at 4:30 p.m. ET on that day to discuss these results and provide an update on its pipeline programs.

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Conference Call and Webcast Information

Interested parties who wish to participate in the conference call may do so by dialing (855) 428-5741 for domestic and (210) 229-8823 for international callers and using conference ID 5367655.

Those interested in listening to the conference call via the internet may do so by visiting the investors and media page on the company’s website at www.onconova.com and clicking on the webcast link. In addition to the live webcast, a replay will be available on the Onconova website for 90 days following the call.

On November 4, 2021 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported that management will participate in three virtual investor conferences including the Credit Suisse 30th Annual Healthcare Conference taking place November 8-11, 2021, the Evercore ISI 4th Annual HealthCONx Conference taking place November 30 – December 2, 2021 and the Piper Sandler 33rd Annual Virtual Healthcare Conference taking place November 30 – December 2, 2021 (Press release, CymaBay Therapeutics, NOV 4, 2021, View Source [SID1234594491]).

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Credit Suisse 30th Annual Healthcare Conference
Date: Monday, November 8
Time: 11:20am Eastern Time Fireside Chat
Webcast: View Source

Evercore ISI 4th Annual HealthCONx Conference
Date: Tuesday, November 30
Time: 9:15am Eastern Time Fireside Chat
Webcast: View Source

Piper Sandler 33rd Annual Virtual Healthcare Conference
Date: Thursday, December 2
Time: 10:00am Eastern Time Fireside Chat
Webcast: View Source

On November 4, 2021 XOMA Corporation (Nasdaq: XOMA) reported NIS793, an anti-TGFβ monoclonal antibody licensed from the Company, has advanced to the Phase 3 development stage, triggering a $35 million milestone payment from Novartis (Press release, Xoma, NOV 4, 2021, View Source [SID1234594490]). The Phase 3 trial (NCT04935359) is designed to assess the efficacy and safety of NIS793 in combination with gemcitabine/nab-paclitaxel versus gemcitabine/nab-paclitaxel and placebo, in first-line metastatic pancreatic ductal adenocarcinoma (mPDAC). In July, Novartis announced the U.S. Food and Drug Administration has granted Orphan Drug Designation to NIS793 in combination with standard of care chemotherapy for the treatment of pancreatic cancer.

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"Pancreatic cancer claims far too many lives every year, and we appreciate that Novartis chose to pursue it as the first indication for late-stage development with NIS793. We are grateful to the patients and their families who are participating in all of the NIS793 clinical studies," stated Jim Neal, Chief Executive Officer of XOMA. "NIS793 represents one of several important assets in our partner-funded portfolio. This $35 million milestone gives us additional capital to acquire the rights to potential future milestone and royalty economics from biotech companies who can then use the capital to pursue their goal of curing a disease or condition by advancing their clinical development activities."

More information about the NIS793 Phase 3 clinical study, NCT04935359, titled "Study of Efficacy and Safety of NIS793 in Combination With Standard of Care (SOC) Chemotherapy in First-line Metastatic Pancreatic Ductal Adenocarcinoma (mPDAC)" can be found at ClinicalTrials.gov.

Under the terms of the 2015 anti-TGFβ development and commercialization agreement with Novartis, XOMA has the potential to earn up to $410 million in additional milestone payments. Should Novartis receive regulatory approval to commercialize NIS793, XOMA will receive tiered royalties on net product sales that range from mid-single digit to low double digits.

NIS793 is an investigational compound. Efficacy and safety have not been established. There is no guarantee that NIS793 will become commercially available.