On March 29, 2022 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that the U.S. Food and Drug Administration (FDA) has cleared UroGen’s Investigational New Drug (IND) application to begin a novel Phase 1 clinical study of the anti-CTLA-4 immunotherapy UGN-301 (zalifrelimab) in patients with recurrent NMIBC (Press release, UroGen Pharma, MAR 29, 2022, View Source [SID1234611132]). The multi-arm Phase 1 study is expected to start in April and support the development of UGN-301 in high-grade (HG) NMIBC.

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UroGen’s pursuit to harness the power of the immune system to fight cancer begins with UGN-301, which UroGen views as a potential cornerstone of a variety of combination therapies targeting recurrent NMIBC and high-grade cancers. UroGen initially plans to combine UGN-301 with UGN-201, the Company’s proprietary formulation of imiquimod a toll-like receptor 7 (TLR7) agonist, which has demonstrated single-agent activity in high-risk bladder cancer patients.

The novel study design will utilize a Master Protocol that UroGen believes is a more efficient and streamlined approach to development. It will provide more flexibility to add study arms as the trial progresses and increase efficiency and reduces costs. UroGen expects the Master Protocol will allow the Company to more quickly evaluate safety, tolerability and dosing of UGN-301 in combination with additional immunomodulators and chemotherapies, with the goal of developing optimized medicines for patients.

"We are pleased that our IND application was cleared to proceed, and we can begin to explore our innovative approach to meeting the high unmet needs in bladder cancer, especially for patients with high-grade disease," says Mark Schoenberg, Chief Medical Officer, UroGen Pharma. "Intravesical delivery of combination therapies is unique with the goal of improving efficacy while avoiding the toxicities associated with systemic treatment of immunotherapies. Our proprietary technology enables local delivery of treatments, which provide opportunities to pursue several promising drug combinations."

Unmet Needs in Bladder Cancer
Bladder cancers are described as muscle invasive or non-muscle invasive based on whether they have invaded the wall of the bladder. HG NMIBC is associated with an increased risk of recurrence and progression. Approximately 25,000 people are diagnosed with HG NMIBC annually. Transurethral resection of bladder tumor (TURBT) followed by intravesical bacillus Calmette-Guérin (BCG) is currently the standard of care for treatment of HG NMIBC.

HG NMIBC patient response to BCG therapy has long been interpreted as evidence that bladder cancer is sensitive to immunotherapy. Unfortunately, many patients with HG disease do not respond to BCG or relapse following therapy. In these cases, patients have limited therapeutic options and often proceed to bladder removal as a means of forestalling disease progression which has a significant association with cancer specific mortality. UroGen’s proprietary RTGel technology provides a novel method for delivering alternatives to BCG including immunomodulatory molecules such as UGN-301 as well as chemotherapeutic agents and other drugs with diverse molecular characteristics and sizes.

On March 29, 2022 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a late clinical stage biopharmaceutical company focused on helping patients fight cancer with oncolytic viral immunotherapies, reported financial results for the fourth quarter and year ended December 31, 2021 and provided a corporate update (Press release, Candel Therapeutics, MAR 29, 2022, View Source [SID1234611131]).

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"2021 was a year of significant progress for Candel, filled with important accomplishments in development of our late-stage pipeline of novel oncolytic viral immunotherapies for cancer treatment. We strengthened our organization for future growth with our successful initial public offering, by meeting key clinical trial accrual milestones and by enhancing our talent pool," said Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer of Candel. "Today, we are well positioned for major catalysts over the next 12 months, namely, multiple data readouts across our product candidates and advancement of our pipeline of promising therapeutics with potential to treat various solid tumors, including lung, brain, pancreatic and prostate cancer. We look forward to delivering on our mission to develop oncolytic viral immunotherapies aimed at tipping the balance in favor of the patient’s immune system to fight cancer."

Fourth Quarter 2021 & Recent Highlights

Presented Candel’s novel oncolytic viral immunotherapies at several preeminent industry conferences:
October 2021: Cambridge Healthtech Institute’s 9th Annual Immuno-Oncology Summit
October 2021: Hanson Wade 6th Annual Oncolytic Virotherapy Summit
October 2021: 28th Annual Prostate Cancer Foundation Scientific Retreat
November 2021: 13th International Oncolytic Virus Conference Meeting
November 2021: Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 36th Annual Meeting
Patrick Y. Wen, MD, principal investigator, presented initial safety data on CAN-2409 in combination with nivolumab from Candel’s Phase 1 clinical trial in high-grade glioma at the 26th Annual Meeting of the Society for Neuro-Oncology in November 2021.
Established a collaboration with Partnership for Accelerating Cancer Therapies and the Cancer Immune Monitoring and Analysis Centers – Cancer Immunologic Data Commons to profile the biomarker response to a combination of CAN-2409 and valacyclovir, in combination with anti-PD-1 and PD-L1 immune checkpoint inhibitors in patients with non-small cell lung cancer (NSCLC) in December 2021.
Enhanced executive leadership with the promotion of Francesca Barone, MD, PhD to Chief Scientific Officer and appointment of Seshu Tyagarajan, PhD, RAC as Chief Technical and Development Officer in 2022.
Strengthened cash position with non-dilutive debt financing with Silicon Valley Bank (SVB) in February 2022.
Appointed Mace Rothenberg, MD, prior Chief Medical Officer of Pfizer, as Senior Advisor to the CEO.
Key Upcoming Milestones

The Company expects to present initial data from an ongoing Phase 2 clinical trial of CAN-2409 and valacyclovir combined with PD-1 or PD-L1 targeting agents in patients with NSCLC in June 2022.
In the fourth quarter of 2022, the Company expects to present data from two high grade glioma clinical trials – a Phase 1b clinical trial of CAN-2409 in combination with nivolumab (Opdivo) combined with standard of care first line treatment and a Phase 1 clinical trial of CAN-3110 in recurrent high-grade glioma.
Financial Results for the Year and Fourth Quarter Ended December 31, 2021

Cash Position: Cash and cash equivalents as of December 31, 2021 were $82.6 million, compared to $35.1 million as of December 31, 2020. The increase was due to receipt of $71.3 million of net proceeds from the Company’s successful initial public offering (IPO) of common stock in August 2021. In addition, the Company utilized $22.2 million in the year ended December 31, 2021 to fund operating activities. Subsequent to December 31, 2021, the Company entered into a term loan agreement with SVB and borrowed $20.0 million. Based on current plans and assumptions, the Company expects that its existing cash and cash equivalents, including the $20 million in term loan proceeds, will be sufficient to fund its operations into the fourth quarter of 2023.

Research & Development Expenses: Research and development expenses were $3.9 million and $15.2 million for the three- and twelve-month periods ended December 31, 2021, respectively, as compared to $3.5 million and $8.8 million for the comparable periods of 2020. The increases were primarily due to increased personnel-related costs, including stock-based compensation, for additional headcount to support the ongoing clinical trials for Candel’s product candidates as well as increased clinical development costs related to our clinical trial sites and the cost of treatment and follow-up on patients. Excluding stock-based compensation expense of ($810,000) and $1.2 million for the three- and twelve-month periods ended December 31, 2021, respectively, research and development expenses for the three- and twelve-month periods ended December 31, 2021, were $4.7 million and $14.0 million, respectively.

General and Administrative Expenses: General and administrative expenses were $3.9 million and $10.7 million for the three- and twelve-month periods ended December 31, 2021, respectively, as compared to $2.6 million and $5.2 million for the comparable periods of 2020. The increases were primarily due to increased personnel-related costs, including stock-based compensation, for additional headcount required to support the growth of the Company and the transition to a public company, an increase in professional fees associated with Candel’s preparation for the IPO completed in August 2021 and costs associated with operating as a public company. Excluding stock-based compensation expense of $364,000 and $1.7 million for the three- and twelve-month periods ended December 31, 2021, respectively, general and administrative expenses for the three- and twelve-month periods ended December 31, 2021, were $3.5 million and $9.0 million, respectively.

Total Operating Expenses: Total operating expenses were $7.8 million and $25.9 million for the three- and twelve-month periods ended December 31, 2021, respectively, as compared to $6.2 million and $13.9 million for the comparable periods of 2020. The increases were primarily due to increased personnel-related costs, including stock-based compensation, for additional headcount required to support the growth of the Company and the transition to a public company, an increase in professional fees associated with Candel’s preparation for the IPO completed in August 2021, an increase in costs associated with operating as a public company and increased clinical development costs. Excluding stock-based compensation expense of ($446,000) and $2.9 million for the three- and twelve-month periods ended December 31, 2021, respectively, total operating expenses for the three- and twelve-month periods ended December 31, 2021, were $8.2 million and $23.0 million, respectively.

Net Loss: Net (income) loss was ($1.6 million) and $36.1 million for the three- and twelve-month periods ended December 31, 2021, respectively, as compared to a net loss of $10.5 million and $17.7 million for the comparable periods of 2020. The net (income) loss for the three- and twelve-month periods ended December 31, 2021, includes a noncash credit and charge of ($9.2 million) and $11.4 million, respectively, for the change in the fair value of the Company’s warrant liability and stock-based compensation expense of ($446,000) and $3.0 million, respectively. Excluding the noncash charges for the change in the warrant liability and the stock-based compensation, the net loss for the three- and twelve-month periods ended December 31, 2021, was $8.0 million and $21.7 million, respectively.

On March 29, 2022 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported that management will participate in three upcoming investor conferences (Press release, Autolus, MAR 29, 2022, View Source [SID1234611130]). An audio webcast of any presentations will be available live. You can access the webcasts via the Events section of the Autolus website. An archived version will also be available through the Company’s website for a limited time following the conference.

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On March 29, 2022 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported the appointment of Reshma Rangwala, MD, PhD, as the Company’s Chief Medical Officer (Press release, Karyopharm, MAR 29, 2022, View Source,-MBA-and-Michael-Kauffman,-MD,-PhD-and-appointment-of-Reshma-Rangwala,-MD,-PhD-as-Chief-Medical-Officer [SID1234611124]). Dr. Rangwala will join the Company in mid-April 2022 and will be responsible for leading the Company’s clinical development programs and strategy.

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"We are delighted to have Dr. Rangwala join our team at this transformative point in our Company’s evolution," said Richard Paulson, President and Chief Executive Officer of Karyopharm. "Her extensive experience developing and executing clinical strategies for novel oncology therapeutics, as well as engaging regulators and the medical community at all stages of development, will be of immeasurable value as we accelerate our four core programs in clinical development."

"I am thrilled to join the Karyopharm team at this exciting and pivotal time in the company’s growth," said Dr. Rangwala. "Given their unique mechanism of action, both XPOVIO (selinexor) and eltanexor have significant potential for patients battling an array of cancer types and I look forward to leading the future clinical development and advancing both assets through the clinic. I am impressed with both the team and the science of XPO1 inhibition and I am excited to realize the potential of these important therapies."

Dr. Rangwala brings to Karyopharm more than a decade of experience in oncology and drug development. She was most recently the Chief Medical Officer at Aravive where she led the clinical development of batiraxcept across multiple tumor types. Prior to Aravive, she served as Vice President, Medical, at Genmab where she led the clinical development program for a first-in-class antibody drug conjugate and managed clinical strategy, protocol development, data monitoring, data analysis, study report authoring, and biologic licensing application preparations. Prior to Genmab, she served as Executive Clinical Director at Merck & Co., where she was involved in the clinical development of KEYTRUDA in non-small cell lung cancer and gynecologic malignancies. She received her B.S. in Biology from Duke University and her M.D./Ph.D. from the University of Cincinnati College of Medicine. Dr. Rangwala completed her internal medicine residency at Barnes Jewish Hospital in St. Louis, MO and her medical oncology fellowship at the Hospital of the University of Pennsylvania.

Further Transition of Co-Founders

Karyopharm also announced today that co-founders Sharon Shacham, PhD, MBA, and Michael Kauffman, MD, PhD will step down from their respective roles as Chief Scientific Officer and Senior Clinical Advisor as of May 31, 2022. Dr. Kauffman has stepped down from his role as a member of the Board of Directors. Dr. Shacham will continue to serve on Karyopharm’s Scientific Advisory Board and will serve in an advisory capacity.

"Through their unwavering dedication and passion, Drs. Shacham and Kauffman built Karyopharm from the ground up, bringing hope to countless cancer patients and their families. It has been an honor to work alongside them, and I’m truly grateful for their partnership over the last year. This executive transition has allowed us to glean as much knowledge and insight from our founders as possible, while also bolstering our leadership team in clinical and program management. We believe that we are well positioned for future success, with a promising and focused pipeline of assets as well as a strong commercial strategy," concluded Mr. Paulson.

"I want to thank all of my colleagues at Karyopharm for their partnership over the years as we built the XPOVIO and eltanexor franchises and advanced our development programs targeting both hematologic and solid tumor malignancies," said Dr. Shacham. "I’m confident the team will continue to achieve its objectives and deliver value to investors, healthcare providers and most importantly, to patients."

"It has been a pleasure working alongside the management team at Karyopharm as we grew the hypothesis about nuclear export and its role in cancer from an idea into a robust commercial enterprise with several programs in development that have significant potential for patients," said Dr. Kauffman. "As the Company enters this next stage of its evolution and growth, I believe Karyopharm is well-positioned to deliver on its mission to bring differentiated medicines to patients battling cancer."

On March 29, 2022 IGM Biosciences, Inc. (NASDAQ: IGMS) reported that it intends to offer and sell $200.0 million of shares of its common stock and, in lieu of common stock to certain investors that so choose, non-voting common stock, in an underwritten public offering (Press release, IGM Biosciences, MAR 29, 2022, View Source [SID1234611123]). In addition, IGM intends to grant the underwriters a 30-day option to purchase up to an additional $30.0 million of shares at the public offering price, less underwriting discounts and commissions. All of the securities offered in the offering will be sold by IGM. The proposed offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

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J.P. Morgan, BofA Securities, Stifel, and Guggenheim Securities are acting as joint book-running managers for the offering.

The securities in the offering will be offered by IGM pursuant to a Registration Statement on Form S-3, filed with the Securities and Exchange Commission (SEC) on August 9, 2021 and declared effective on August 19, 2021. IGM will file a preliminary prospectus supplement and accompanying prospectus relating to the proposed offering with the SEC, copies of which can be accessed for free through the SEC’s website at www.sec.gov. The final terms of the offering will be disclosed in a final prospectus supplement to be filed with the SEC. When available, copies of the preliminary prospectus supplement, the final prospectus supplement and the accompanying prospectuses relating to this offering may also be obtained from: J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204, or by email at [email protected]; BofA Securities, Attention: Prospectus Department, NC1-004-03-43, 200 North College Street, 3rd Floor, Charlotte, North Carolina 28255, or via email: [email protected]; Stifel, Nicolaus & Company, Incorporated, One Montgomery Street, Suite 3700, San Francisco, CA 94104, Attn: Syndicate, or by phone at (415) 364-2720, or by email at [email protected]; or Guggenheim Securities, LLC, Attention: Equity Syndicate Department, 330 Madison Avenue, New York, NY 10017, by telephone at (212) 518-9544, or by email at [email protected].

This press release does not constitute an offer to sell or a solicitation of an offer to buy, nor will there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation, or sale would be unlawful before registration or qualification under the securities laws of that state or jurisdiction.