On April 27, 2022 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, reported that it will host a conference call on Thursday, May 5, 2022 at 5:00pm ET to discuss its financial results and corporate update for the quarter ended March 31, 2022 (Press release, Ultragenyx Pharmaceutical, APR 27, 2022, https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-host-conference-call-first-quarter-2022-financial [SID1234613037]).

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The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial
(855) 797-6910 (USA) or (262) 912-6260 (International) and enter the passcode 7951356. The replay of the call will be available for one year.

On April 27, 2022 Novartis reported positive topline results from an interim analysis of the Phase III RATIONALE 306 study, which showed anti-PD-1 immune checkpoint inhibitor tislelizumab plus chemotherapy significantly improved overall survival (OS) compared to chemotherapy in patients with previously untreated unresectable, locally advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC), regardless of PD-L1 expression (Press release, Novartis, APR 27, 2022, View Source [SID1234613053]). Novartis intends to submit these data to regulatory authorities, and will collaborate with BeiGene to present them at an upcoming medical meeting.

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"People living with esophageal cancer experience painful everyday challenges and typically have a poor prognosis, with a five-year survival rate of around five percent for metastatic cases, underscoring the urgency for more immunotherapy options," said Jeff Legos, Executive Vice President, Global Head of Oncology & Hematology Development. "We plan to discuss these data with health authorities, and we will continue to expand our tislelizumab clinical development program in pursuit of novel, synergistic combinations with the ultimate goal of extending survival for more patients."

ESCC is the most common type of esophageal cancer globally, with an estimated 604,000 new cases and 544,000 deaths from esophageal cancer internationally in 2020.2 In the United States, it is estimated there will be more than 20,000 new diagnoses and more than 16,000 deaths from esophageal cancers.3

RATIONALE 306 (NCT03783442) is a multi-regional Phase III, randomized, placebo-controlled, double-blind study of tislelizumab in combination with chemotherapy versus chemotherapy alone in patients with unresectable, locally advanced recurrent or metastatic ESCC. Approximately 649 study participants were randomized 1:1 to receive either tislelizumab plus chemotherapy or chemotherapy alone. The primary endpoint is OS. Secondary endpoints include progression-free survival, objective response rate, duration of response, health-related quality of life measures and safety.

Tislelizumab is currently under review by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for advanced or metastatic ESCC after prior chemotherapy. The EMA is also reviewing tislelizumab for advanced or metastatic non-small cell lung cancer (NSCLC) after prior chemotherapy, and in combination with chemotherapy for previously untreated advanced or metastatic NSCLC.

About Tislelizumab
Novartis is evaluating tislelizumab, a uniquely designed anti-PD-1 monoclonal antibody, in a global clinical development program consisting of 14 pivotal clinical trials across a broad array of solid tumors, with more than 8,800 patients enrolled to date in 35 countries. Novartis four distinct therapeutic platforms (immunotherapy, radioligand therapy, cell and gene therapy, targeted therapy) offer a unique opportunity to study tislelizumab in differentiated, potentially synergistic combinations across our pipeline and portfolio of market compounds.

Novartis has the rights to develop, manufacture and commercialize tislelizumab in North America, Europe and Japan through a collaboration and license agreement with BeiGene.

On April 27, 2022 SQZ Biotechnologies (NYSE: SQZ), focused on unlocking the full potential of cell therapies for multiple therapeutic areas, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the company’s lead cell therapy candidate, SQZ-PBMC-HPV, for the treatment of HPV16+ advanced or metastatic solid tumors (Press release, SQZ Biotech, APR 27, 2022, View Source [SID1234613070]).

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Fast Track Designation is designed to accelerate the development and review of treatments for serious and life-threatening diseases where no treatment currently exists or where the treatment in discovery may be better than what is currently available.

"We are thrilled to receive FDA Fast Track Designation for our SQZ Antigen Presenting Cells product candidate," said Armon Sharei, Ph.D., Chief Executive Officer and Founder at SQZ Biotechnologies. "This designation adds to our exciting clinical data presented at ESMO (Free ESMO Whitepaper)-IO last year where we first demonstrated the potential of our Cell Squeeze technology to drive clinical benefit while maintaining favorable tolerability. The FDA Fast Track program can potentially expedite future review processes and accelerate the registrational path for SQZ-PBMC-HPV."

Data presented at the ESMO (Free ESMO Whitepaper) Immuno-Oncology Congress 2021 has shown that the company’s lead APC cell therapy candidate induced radiographic, symptomatic and immune response as monotherapy in a post-checkpoint HPV16+ solid tumor patient. The company continues to enroll patients in its highest dose monotherapy cohort and is simultaneously enrolling patients for combination therapy in the company’s Phase 1/2 SQZ-PBMC-HPV-101 clinical trial.

SQZ-PBMC-HPV-101 Trial Design

SQZ-PBMC-HPV is being evaluated in a Phase 1/2 clinical trial for the treatment of HPV16+ advanced or metastatic solid tumors. Patients must be positive for the human leukocyte antigen serotype HLA-A*02. The investigational candidate, which targets E6 and E7 oncoproteins, is being studied as a monotherapy and in combination with immuno-oncology agents. The study’s primary outcome measures in the monotherapy and combination phases of the trial include safety and tolerability. Antitumor activity is a secondary outcome measure in both the monotherapy and combination stages of the trial, and manufacturing feasibility is a secondary outcome measure in the monotherapy phase of the trial. The monotherapy phase of the study includes escalating dose cohorts with a dose-limiting toxicity (DLT) window of 28 days and is designed to identify a recommended phase 2 dose. The planned combination phase of the study will include SQZ-PBMC-HPV and checkpoint inhibitors. DLT will be measured over 42 days.

About Human Papillomavirus Positive Cancers

Human papillomavirus (HPV) is one of the most common viruses worldwide and certain strains persist for many years, often leading to cancer. According to the Centers for Disease Control (CDC), in the United States HPV+ tumors represent 3% of all cancers in women and 2% of all cancers in men, resulting in over 39,000 new cases of HPV+ tumors every year. HPV infection is larger outside of the U.S., and according to the International Journal of Cancer, HPV+ tumors account for 4.5% of all cancers worldwide resulting in approximately 630,000 new cases every year. According to the CDC, HPV infection plays a significant role in the formation of more than 90% of anal and cervical cancers, and most cases of vaginal (75%), oropharyngeal (70%), vulval (70%) and penile (60%) cancers.

On April 27, 2022 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported the following executive management changes: Dr. Claus Moller has stepped down from his positions as Chief Executive Officer and Board Member effective immediately (Press release, Y-mAbs Therapeutics, APR 27, 2022, View Source [SID1234613087]). Thomas Gad, the Company’s Founder, Chairman and President has assumed the role of Interim CEO and Board Member. In addition, Mr. Gad has stepped down as Chairman and Dr. Jim Healy, current Board Member and Chair of the Compensation Committee, has been appointed as Chairman of the Board. The Board has begun a search for Dr. Moller’s successor.

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Dr. Healy said, "On behalf of the Board, we thank Claus for his service and many accomplishments during his tenure as CEO. The Company has made great strategic, commercial, and operational progress. We wish Claus the very best. Claus has assembled a talented and highly capable leadership team, which will ensure continuity while we conduct a search for a permanent replacement."

Thomas Gad further notes, "I want to personally thank Claus for his contribution to building Y-mAbs. At the same time, I am excited about Y-mAbs future. DANYELZA sales are increasing, and we just ended the first quarter of 2022 with net revenues of $10.5 million, which provides us the visibility to issue full year revenue guidance of $45-50 million. The omburtamab BLA was resubmitted on March 31, 2022 and is currently under review with the FDA. Financially, as of March 31, 2022, we believe we are well-positioned with $156.7 million in cash that provides a runway to mid-2024. Upon the potential approval of omburtamab, the Company will be entitled to receive a Priority Review Voucher ("PRV"). Proceeds from monetization of any such PRV and potential omburtamab revenues are currently not included in this guidance. At this point in time, most of the pivotal trials, post marketing commitments and regulatory work for omburtamab are behind us, and we have further adjusted our operating expenditures for 2022, corresponding to operating expenses of $162-167 million and a net cash burn of $78-83 million, to ensure we can deliver on our future milestones."

Preliminary Financial Results

The preliminary financial results set forth above are unaudited and based on management’s initial review of the Company’s results as of and for the quarter ended March 31, 2022, and are subject to revision based upon the Company’s quarter-end closing procedures and the completion of the review by the Company’s external auditors of the Company’s quarter-end financial statements. Actual results may differ materially from these preliminary results as a result of the completion of quarter-end closing procedures, final adjustments, and other developments arising between now and the time that the Company’s financial results are finalized. In addition, these preliminary results are not a comprehensive statement of the Company’s financial results for the quarter ended March 31, 2022, should not be viewed as a substitute for complete financial statements prepared in accordance with U.S. generally accepted accounting principles, and are not necessarily indicative of the Company’s results for any future period.

On April 27, 2022 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products for cancer, reported that it will report first quarter 2022 financial results on Wednesday, May 4, 2022 after the close of the market (Press release, Allogene, APR 27, 2022, View Source [SID1234616274]). The announcement will be followed by a live audio webcast and conference call at 2:00 PM Pacific Time/5:00 PM Eastern Time.

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Audio Webcast
The webcast will be made available on the Company’s website at www.allogene.com under the Investors tab in the News and Events section. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.