On January 6, 2023 Precision BioSciences (Nasdaq: DTIL), a clinical stage gene editing company developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies, reported that the U.S. Food and Drug Administration (FDA) provided supportive Type C feedback on the Company’s CMC processes and analytical methods for azercabtagene zapreleucel (azer-cel; PBCAR0191) (Press release, Precision Biosciences, JAN 6, 2023, View Source [SID1234625977]). Azer-cel is Precision BioSciences lead allogeneic CAR T candidate being evaluated for relapsed or refractory (R/R) non-Hodgkin lymphoma (NHL) in subjects who have relapsed following autologous CAR T treatment.

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"We are pleased with the FDA feedback on key components of our planned CMC package to support azer-cel ongoing development. The responses to questions regarding our analytical methods, proposed potency assays, and manufacturing processes are aligned with our expectations and help guide our plans," said Karl Whitney, Vice President and Head of Regulatory Affairs at Precision BioSciences. "We appreciate the FDA’s input and look forward to continuing regulatory engagement at critical points along our development horizon."

"We look forward to continuing enrollment in the ongoing Phase 1/2a trial and working closely with the FDA as we pursue development of azer-cel. Azer-cel has the potential to become the first allogeneic CAR T therapy to reach the market," said Alan List, M.D., Chief Medical Officer at Precision BioSciences. "There are currently no FDA-approved allogeneic CAR T products to address the high unmet need of the CAR T relapsed or refractory patient population. While autologous CAR T therapy is a promising treatment option for hematological malignancies, up to 60% of high-grade NHL patients relapse after treatment and up to 20% of intended auto-CAR T patients never receive treatment due to inability to manufacture the CAR T product. An effective off-the-shelf allogeneic CAR T treatment has the potential to offer clinical benefit to this growing population of lymphoma patients."

On January 6, 2023 Portage Biotech presented its corporate presentation (Presentation, Portage Biotech, JAN 6, 2023, View Source [SID1234625976]).

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On January 6, 2023 Phio Pharmaceuticals (Nasdaq: PHIO), a clinical stage biotechnology company whose proprietary INTASYL RNAi platform technology is designed to make immune cells more effective in killing tumor cells, reported that its Principal Executive Officer and Executive Chairman, Robert Bitterman, will be a featured speaker at the Entrepreneurial Showcase at the Dermatology Summit in San Francisco on January 8th (Press release, Phio Pharmaceuticals, JAN 6, 2023, View Source [SID1234625974]). The Dermatology Summit is taking place at the Hyatt Regency, alongside the annual JPMorgan Healthcare Conference.

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Mr. Bitterman will discuss innovations in dermatological cancers, highlighting Phio’s proprietary INASTYL platform. INTASYL is the only self-delivering RNAi technology focused on immuno-oncology therapeutics.

Mr. Bitterman was recently appointed to his leadership role at Phio. He previously led Cutanea and Dermik in the development and commercialization of small molecule dermatological products.

On January 6, 2023 Phio Pharmaceuticals Corp. (Nasdaq: PHIO) reported their clinical development partner, AgonOx, Inc. has received confirmation from the FDA that their planned Phase 1 clinical trial in patients with advanced solid tumors may proceed (Press release, Phio Pharmaceuticals, JAN 6, 2023, View Source [SID1234625973]). The initial Phase 1 clinical trial is designed to assess safety and to study the potential for an enhanced therapeutic benefit from the administration of Phio’s PH-762 treated "double positive" (DP) CD8 tumor infiltrating lymphocyte (TIL) in patients with melanoma and other advanced solid tumors.

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Phio is a clinical stage biotechnology company whose proprietary INTASYL RNAi technology is designed to make immune cells more effective in killing tumor cells. Phio’s PH-762 is an INTASYL compound that reduces the expression of PD-1, a protein that inhibits T cells’ ability to kill cancer cells. By decreasing PD-1 expression, the T cells have increased capacity to kill cancer cells.

AgonOx, in collaboration with the Earle A. Chiles Research Institute, a division of Providence Cancer Institute of Oregon, has shown that DP CD8 T cells sorted from human solid tumors (AGX148) are highly enriched for tumor-reactivity leading to increased tumor killing when compared to the other CD8 tumor infiltrating lymphocyte (TIL) subsets.

Preclinical data from in-vitro studies using autologous human tumor cells demonstrated that treating AGX148 with Phio’s PH-762 increased by two-fold their tumor killing activity. The trial will be conducted at Providence under the leadership of Dr. Weinberg and Principal Investigator Brendan Curti, MD, Medical Oncologist and Robert W. Franz Endowed Chair for Clinical Research.

"We’re pleased to have reached this important milestone in our collaboration with AgonOx and Providence Cancer Institute to develop improved cell therapies for cancer patients," said Robert Bitterman, Phio’s Principal Executive Officer and Executive Chairman.

"This bench to bedside research has the potential to help patients with solid malignancies and this is the first step to bringing this therapy to patients," said Andrew Weinberg, PhD, Founder and Chief Scientific Officer at AgonOx, Inc. and Research Scientist/Judy Hartmann Endowed Chair at Providence.

On January 6, 2023 Mersana Therapeutics, Inc. (NASDAQ: MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, reported a business update and announced strategic objectives and expected milestones for 2023 (Press release, Mersana Therapeutics, JAN 6, 2023, View Source [SID1234625972]).

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"Following a year of tremendous accomplishment in 2022, we are now approaching top-line data from our first registrational trial of UpRi, which we believe will provide an opportunity to further demonstrate Mersana’s increasing role as an ADC leader," said Anna Protopapas, President and Chief Executive Officer of Mersana Therapeutics. "The data we expect to report from our UPLIFT clinical trial in mid-2023 will represent the most significant milestone to date in our effort to establish UpRi as a foundational medicine for patients with ovarian cancer. Assuming positive data, we plan to target the submission of a BLA around the end of 2023 and prepare for a potential U.S. commercial launch in 2024. We will also continue to advance our UP-NEXT and UPGRADE-A trials of UpRi in earlier lines of treatment."

"Beyond UpRi, our efforts to expand the reach of all three of our fully-scaled ADC platforms, each supported by substantial data, will remain a central theme in 2023," continued Ms. Protopapas. "We will work aggressively to progress our two next-generation ADCs, XMT-1660 and XMT-2056, in Phase 1 trials and establish proof-of concept. Additionally, exploring new collaborations will remain a core component of our strategy as we seek to build upon Mersana’s recent business development successes."

Strategic Objective: Establish UpRi as a Foundational Medicine in Ovarian Cancer

2022 Accomplishments

Completed enrollment in UPLIFT, the company’s single-arm registrational trial of UpRi in platinum-resistant ovarian cancer
Initiated Phase 3 UP-NEXT clinical trial of UpRi as a maintenance monotherapy in recurrent platinum-sensitive ovarian cancer
Neared completion of dose escalation in Phase 1 UPGRADE-A trial of UpRi in combination with carboplatin in platinum-sensitive ovarian cancer
Announced that the European Commission has designated UpRi as an orphan medicinal product for the treatment of ovarian cancer

Expected Milestones

Report top-line data from UPLIFT in mid-2023
Assuming positive data, submit a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) around the end of 2023
Prepare for potential U.S. accelerated approval and commercial launch in 2024
Significantly advance enrollment of UP-NEXT in 2023
Initiate dose expansion portion of UPGRADE-A in the first quarter of 2023 and report interim data from UPGRADE-A in the second half of 2023

Strategic Objective: Advance Clinical-Stage Pipeline

2022 Accomplishments

XMT-1660: Initiated multicenter Phase 1 clinical trial in patients with previously treated breast, endometrial and ovarian cancers
XMT-1660: Announced Fast Track designation for the treatment of adult patients with advanced or metastatic triple-negative breast cancer
XMT-2056: Announced FDA orphan drug designation for the treatment of gastric cancer

Expected Milestones

XMT-1660: Complete dose escalation portion of Phase 1 clinical trial in 2023
XMT-2056: Initiate Phase 1 clinical trial in the first quarter of 2023
Strategic Objective: Position Mersana as the ADC Partner-of-Choice

2022 Accomplishments

Entered into the following agreements that collectively provided Mersana with $170 million in upfront payments and an opportunity for more than $3 billion in milestones, plus royalties:
An Immunosynthen research collaboration and license agreement with Merck KGaA, Darmstadt, Germany ​for two targets, which includes a $30 million upfront payment to Mersana and the potential for up to $800 million in total potential milestones, plus tiered royalties up to the low double-digits on net sales
A collaboration, option and license agreement with GlaxoSmithKline plc (GSK) for the co-development and commercialization of XMT-2056, which provided Mersana with a $100 million upfront option purchase fee and the potential to receive up to $1.36 billion in the form of an additional option exercise fee and milestone payments, plus an option for Mersana to retain a U.S. profit share and tiered royalties on net sales outside of the United States or to receive tiered royalties up to the mid-twenties on global net sales
A Dolasynthen research collaboration and license agreement with Janssen Biotech, Inc. for three targets, which provided Mersana with a $40 million upfront payment and the potential to receive over $1 billion in total potential milestones, plus tiered royalties up to the low double-digits on net sales

Expected Milestones

Pursue impactful new collaborations
Execute against existing collaboration agreements

Financial Update

Mersana estimates that its cash, cash equivalents and marketable securities as of December 31, 2022 were approximately $280 million. This figure is preliminary and unaudited. The company expects to report its audited cash, cash equivalents and marketable securities, as well as other information necessary for a complete understanding of its financial position, in its Annual Report on Form 10-K for the year ended December 31, 2022. The company expects that its available funds, together with the $30 million upfront payment due from Merck KGaA, Darmstadt, Germany ​under the collaboration and license agreement referenced above, will be sufficient to support its operating plan commitments into the second half of 2024.