On March 16, 2023 Jounce Therapeutics, Inc. (Nasdaq: JNCE), a clinical-stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers, reported that patients from the INNATE Phase 2 trial in the ovarian cancer combination cohort with platinum resistant ovarian cancer are experiencing deep and durable responses based on a pre-planned informal data review (Press release, Jounce Therapeutics, MAR 16, 2023, View Source [SID1234628925]).

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The INNATE trial evaluates JTX-8064 as a monotherapy and in combination with the PD-1 inhibitor pimivalimab in patients with advanced solid tumors. JTX-8064, is a humanized IgG4 monoclonal antibody designed to specifically bind to the macrophage receptor Leukocyte Immunoglobulin Like Receptor B2 (LILRB2/ILT4), inhibiting LILRB2 binding with its ligands which may result in reprogramming immune-suppressive macrophages to enhance anti-tumor immunity.

A data review was conducted on March 15, 2023 to assess the potential to achieve proof-of-concept (POC) in the ovarian cancer combination cohort of the INNATE trial. In this cohort, 35 patients with third- and fourth-line platinum resistant ovarian cancer were treated with JTX-8064 and Jounce’s PD-1 inhibitor, pimivalimab. There were five RECIST 1.1 responders, four of which are confirmed, with all five patients remaining on study for over six months with continued tumor reduction over time. The remaining 30 patients have discontinued treatment. Upon confirmation of a fifth confirmed partial response (PR), the ovarian combination cohort would meet Jounce internal criteria for POC, based on a statistically meaningful improvement over the benchmark of pembrolizumab alone in the analogous setting. In patients with one to six prior lines of therapy for ovarian cancer, pembrolizumab monotherapy demonstrated a response rate of 8.5%, with duration of response of 10.2 months. Two of the five responders in INNATE have a PD-L1 score of zero, indicating a low likelihood of response to a PD-1 inhibitor. The combination was well tolerated with <10% of patients reporting Grade 3 or greater related adverse events.

Ovarian Cancer Combination Cohort Responders

Subject PD-L1
(CPS) Response
Assessment When was uPR
demonstrated Current status
1 55% cPR Wk 9 -82.8% (wk 45)
2 15% cPR Wk 18 -66.4% (wk 36)
3 5% cPR Wk 18 -87.6% (wk 27)
4 0% cPR Wk 18 -78.26% (wk 27)
5 0% uPR Wk 27 -40% (wk 27)

Ovarian Combination Cohort Target Lesion % Change from Baseline over Time

"Following this analysis, we are pleased to see these results demonstrating deep and durable responses in patients, including those with a PD-L1 score of 0%, on a very well tolerated regimen. These results lead us to believe that there is a potential for meaningful clinical benefit with this combination in patients with few durable therapeutic options," said Richard Murray, Ph.D., chief executive officer and president of Jounce Therapeutics. "Tumor reduction was observed at 9 weeks in all the responding patients in the ovarian cancer cohort of the INNATE trial, but most did not achieve a PR until week 18, which delayed our ability to assess efficacy in this cohort. Platinum resistant ovarian cancer is a patient population with significant unmet need and progressive disease is often associated with debilitating symptoms."

Enrollment has been completed in all cohorts in the INNATE trial. In addition to the patients with ovarian cancer, both durable responses and stable disease have occurred in patients with renal cell carcinoma, biliary tract cancer, and first-line and second/third-line head and neck squamous cell carcinoma, but the ovarian data is the most encouraging with potential to achieve pre-determined POC criteria.

As previously announced, Jounce is seeking to partner JTX-8064. Jounce continues to believe a company with additional resources and a longer value creation timeline could potentially advance this program for the benefit of cancer patients.

On March 16, 2023 Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer drugs, reported a clinical development and operational update (Press release, Bio-Path Holdings, MAR 16, 2023, View Source [SID1234628923]).

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"Throughout 2022, we laid the foundation to meaningfully advance our DNAbilize antisense RNAi nanoparticle technology in a number of important oncology indications for which there are limited treatment options," said Peter H. Nielsen, President and Chief Executive Officer of Bio-Path. "In addition to our clinical progress, we made significant investment towards shoring up our manufacturing supply and intellectual property armamentarium. Given these advancements and the proven safety and efficacy profile shown to date across our portfolio, we look forward to several near-term clinical milestones."

Important Near-Term Clinical Milestones

BP1001-A Phase 1/1b Clinical Trial in Solid Tumors

● Important trial with advanced or recurrent solid tumors, including ovarian and uterine, pancreatic and breast cancer with initial cohort completion and data readout expected before mid-year.

BP1002 Phase 1/1b Clinical Trial in Relapsed/Refractory AML

● Focus on patients who relapsed on venetoclax treatment with initial cohort completion and readout expected in the second quarter of 2023.

Prexigebersen (BP1001) Phase 2 Clinical Trial in AML

● Two of the three cohorts in the clinical trial already exceed the minimum efficacy required for enrollment expansion.
● Assess safety and efficacy of each cohort treatment combination therapy with potential to qualify for expedited program status after cohort’s initial interim analysis, which are expected to commence by cohort in the second quarter of 2023.

Clinical Program Overview

Bio-Path’s clinical development program consists of one Phase 2 clinical trial and three Phase 1 or 1/1b clinical trials. There is one additional drug candidate that is in preclinical development, which may be submitted to the FDA later in the year in an Investigational New Drug (IND) application.

Phase 2 Clinical Trial – Bio-Path’s Phase 2 clinical trial is treating Acute Myeloid Leukemia (AML) patients. This trial is comprised of three separate cohorts of patients and treatments, each separately approvable by the FDA as a new drug treatment. The first two cohorts are treating patients with the triple combination of prexigebersen (Bio-Path’s drug candidate BP1001), decitabine and venetoclax. The first cohort includes untreated AML patients, and the second cohort includes relapsed/refractory AML patients. Finally, the third cohort is treating relapsed/refractory AML patients, who are venetoclax-resistant or -intolerant, with the two-drug combination of prexigebersen and decitabine. The interim analysis of each cohort to assess the safety and efficacy of treatment combination therapy is expected to commence by cohort in the second quarter of 2023, with the potential to qualify for expedited regulatory status.

Phase 1/1b Clinical Trial in BP1001-A in Advanced Solid Tumors – Phase 1/1b clinical trial of BP1001-A in patients with advanced or recurrent solid tumors, including ovarian and uterine, pancreatic and breast cancer. BP1001-A is a modified

product candidate that incorporates the same drug substance as prexigebersen but has a slightly modified formulation designed to enhance nanoparticle properties. Completion of the first dose escalation cohort is expected in the coming months.

Phase 1/1b Clinical Trial in BP1002 in Relapsed/Refractory AML. Phase 1/1b clinical trial for BP1002 to treat relapsed/refractory AML patients, including venetoclax-resistant patients. BP1002 targets the protein Bcl-2, which is responsible for driving cell survival in up to 60% of all cancers. AML patients that fail frontline venetoclax-based therapy have very poor prognosis with median overall survival of less than three months. Completion of the first dose escalation cohort is expected in the coming months.

Phase 1 Clinical Trial in BP1002 in Refractory/Relapsed Lymphoma and Chronic Lymphocytic Leukemia (CLL). Phase 1 clinical trial for refractory/relapsed lymphoma and CLL. The Phase 1 clinical trial is being conducted at several leading cancer centers, including the Georgia Cancer Center, The University of Texas Southwest and New York Medical College. Completion of the current patient cohort is expected in 2023.

Preclinical Work for BP1003. The Company continues to advance its drug candidate, BP1003, for the treatment of advanced solid tumors, including pancreatic cancer. BP1003 is an antisense RNAi nanoparticle targeting the STAT3 protein. Plans are to conduct a Phase 1 study of BP1003 in patients with refractory, metastatic solid tumors (pancreatic, non-small cell lung cancer).

Manufacturing Supply

The COVID-19 pandemic created manufacturing interruptions over the last several years. The Company experienced manufacturing shutdowns in supply chain manufacturing plants and an increase in lost manufactured product batches that created supply disruptions. In addition, the onset of messenger RNA vaccine development for COVID-19 created increased backlog time at oligonucleotide suppliers. These conditions created drug candidate supply shortfalls that caused enrollment challenges for Bio-Path’s clinical trials.

The result of these factors led Bio-Path to double the Company’s supply chain, increase capacity, quality and improve scheduling flexibility. These goals were achieved in 2022 and have resulted in increased drug candidate supply, quality and patient enrollment.

Intellectual Property Protection

Bio-Path’s composition of matter patents protect encroachment from third parties on its proprietary products. This technology is solely owned by Bio-Path. These composition patents allow the Company to apply its core technology to new protein targets and receive new 20-year patents. Bio-Path’s patent portfolio is as follows:

● New composition and methods of use patent issued covers DNAbilize technology, solely owned by Bio-Path.
● Five patents issued in the United States; eight foreign patents issued; one additional foreign patent application allowed.
● Five applications pending in the United States along with 60+ applications pending in foreign jurisdictions.

On March 16, 2023 Armata Pharmaceuticals, Inc. (NYSE American: ARMP) ("Armata" or the "Company"), a biotechnology company focused on pathogen-specific bacteriophage therapeutics for antibiotic-resistant and difficult-to-treat bacterial infections, reported financial results for its fourth quarter and full-year 2022, and provided a corporate update (Press release, AmpliPhi Biosciences, MAR 16, 2023, View Source [SID1234628922]).

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Fourth Quarter 2022 and Recent Developments:

·

Announced positive topline results from the SWARM-P.a. Phase 1b/2a clinical trial, which evaluated AP-PA02 in cystic fibrosis patients with chronic pulmonary Pseudomonas aeruginosa infections, supporting progression into Phase 2b;

·

Announced further clinical progression of AP-PA02 with the first patient dosed in the Phase 2 non-cystic fibrosis bronchiectasis (NCFB) study;

·

Advanced AP-SA02 with the completion of first cohort dosing in the Phase 1b/2a Staphylococcus aureus bacteremia study;

·

AP-SA02 prosthetic joint infection study start-up activities continue; and

·

Closed $30 million convertible credit agreement with Innoviva Strategic Opportunities LLC, a wholly owned subsidiary of Innoviva, Inc., Armata’s largest shareholder.

"During the fourth quarter and subsequent period, we made significant progress across multiple programs, highlighted by the announcement of positive topline results from the SWARM-P.a. Phase 1b/2a clinical trial" stated Dr. Brian Varnum, Chief Executive Officer of Armata. "These data demonstrated AP-PA02 to be well tolerated and pharmacokinetics and pharmacodynamics data indicate phage can be dosed in a controllable manner through nebulization. The study design of single ascending and multiple ascending dose exploration served to highlight dose levels and frequency required to achieve target levels of microbial reduction. Exploration of AP-PA02 dose and schedule is continuing in our Phase 2 NCFB study, enabling continuous advancement of AP-PA02 across these related indications. Similarly, in 2023 we anticipate enrolling subjects in our second clinical indication for AP-SA02, namely Staphylococcus aureus prosthetic joint infection, which represents our fourth indication for our two lead programs."

"I am very pleased with what we have accomplished in 2022, and we entered the new year with line-of-sight to data readouts that are essential to advance phage therapy through rigorous clinical trials required to deliver novel treatment options to patients suffering from serious bacterial infections while creating enduring value for our shareholders. Our trials are designed to show the value of phage therapy on top of the standard of care, which if successful, creates the opportunity for broad use of phage therapy. This profile is distinct from the limitations faced by novel antibiotics, which are often reserved for last-line therapy, limiting commercial sales. The Armata team is well-positioned for a successful 2023," concluded Dr. Varnum.

Fourth Quarter 2022 Financial Results

Grant Revenue. The Company recognized grant revenue of approximately $1.1 million for the three months ended December 31, 2022, which represents Medical Technology Enterprise Consortium ("MTEC")’s share of the costs incurred for the Company’s AP-SA02 program for the treatment of Staphylococcus aureus bacteremia. The Company expects to receive $16.3 million in grant funding from MTEC administered by the U.S. Department of Defense and

Graphic

the Defense Health Agency and Joint Warfighter Medical Research Program. The Company recognized approximately $1.0 million of revenue in the comparable period in 2021.

Research and Development. Research and development expenses for the three months ended December 31, 2022, were approximately $9.6 million as compared to approximately $4.8 million for the comparable period in 2021. The company continues to invest in clinical trial and personnel related expenses associated with its primary development programs.

General and Administrative. General and administrative expenses for the three months ended December 31, 2022, were approximately $1.8 million as compared to approximately $2.2 million for the comparable period in 2021.

Loss from Operations. Loss from operations for the three months ended December 31, 2022, was $(10.3) million as compared to a loss from operations of approximately $(6.0) million for the comparable period in 2021.

Cash and Equivalents. As of December 31, 2022, Armata held approximately $14.9 million of unrestricted cash and cash equivalents, as compared to $10.3 million as of December 31, 2021.

As of March 13, 2023, there were approximately 36.1 million shares of common stock outstanding.

On March 16, 2023 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a biopharmaceutical company focused on advancing innovative immune-oncology technologies addressing hard to treat cancers, reported that it will participate in the Virtual Investor NETs in Cancer Spotlight Event on Wednesday, March 29, 2023 at 11:00 AM ET (Press release, Xenetic Biosciences, MAR 16, 2023, View Source [SID1234628915]).

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For the roundtable discussion, Jeffrey F. Eisenberg, Chief Executive Officer and Curtis A. Lockshin, PhD, Chief Scientific Officer of Xenetic Biosciences will be joined by Jonathan Spicer, MD, PhD, Scientific Advisor to the Company and seasoned surgeon scientist that currently serves as an Associate Professor of Surgery at McGill University and Medical Director of the McGill University Health Center (MUHC) Thoracic Oncology Network. Dr. Spicer is recognized as a leader in understanding how neutrophils impact cancer progression, in particular, the role of NETs in cancer biology, and has developed one of the most active research programs in the area of neoadjuvant immunotherapy for operable lung cancer. As part of the event, the Company and Dr. Spicer will discuss Xenetic’s DNase-based oncology platform, the role of Neutrophil Extracellular Traps ("NETs") in cancer and the use of DNase in targeting NETs, and the potential broad utility of the platform for the treatment of high-value oncology indications where there remains significant unmet need.

In addition to the moderated portion of the event, investors and interested parties will have the opportunity to submit questions live during the event. The Company will answer as many questions as possible during the event.

A live video webcast of the Virtual Investor NETs in Cancer Spotlight Event will be available on the Events page in the Investors section of the Company’s website (xeneticbio.com) and archived for 90 days.

On March 16, 2023 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported financial results for the fourth quarter and full year ended December 31, 2022, and overview of recent developments (Press release, UroGen Pharma, MAR 16, 2023, View Source [SID1234628914]).

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"I’m proud of our progress in 2022 and encouraged by our pipeline advancement and expanded access to JELMYTO as we work to fundamentally change the treatment paradigm of urologic cancers," said Liz Barrett, President and Chief Executive Officer of UroGen. "Last year was a marked year for our exciting investigational product, UGN-102, with the potential to transform the treatment of bladder cancer as the only non-surgical therapeutic for LG-IR-NMIBC. As we have shared many times, we believe UGN-102 could be a transformational product, and one that represents a significant opportunity to address the estimated 80,000 patients suffering from LG-IR-NMIBC in the U.S. each year. In 2022, we initiated and completed enrollment of ENVISION, our Phase 3 pivotal study of UGN-102, and more recently shared long-term follow up data from the previously completed OPTIMA II study, which showed a 24.4-month median time to recurrence in evaluable patients who completed the study. Topline data from the ATLAS and ENVISION trials are expected mid-year 2023, and assuming positive results, we remain on track to submit a New Drug Application (NDA) in 2024. Supported by a growing body of real-world outcomes data, we delivered our strongest ever JELMYTO net sales in Q4 helping propel ~34% full-year growth for fiscal 2022. Looking ahead, we remain confident in the long-term success of JELMYTO in low-grade upper tract urothelial cancer (LG-UTUC) and the significant market opportunity of UGN-102, if approved, to drive growth in the years ahead."

Business Highlights:

UGN-102 (mitomycin) for intravesical solution:

Announced full enrollment in Phase 3 ENVISION pivotal study of UGN-102 for the treatment of LG-IR-NMIBC, which targeted enrollment of ~220 patients across 90 sites. Assuming positive findings, UroGen anticipates submitting an NDA for UGN-102 in 2024. Topline results from the primary endpoint evaluating the complete response rate at 3-months after first instillation are expected mid-year 2023.
Topline data from the ATLAS trial, the predecessor to ENVISION, is expected mid-year 2023 and will evaluate complete response, duration of response and safety from ~280 patients that completed the trial.
Shared new long-term follow up data from the Phase 2b OPTIMA II study at the Annual Society of Urologic Oncology (SUO) Meeting demonstrating a median DOR of 24.4 months (10.1 to 30.7 months) with UGN-102 in 15 of 25 evaluable patients.
Announced preliminary results of a study to assess the feasibility of home instillation of UGN-102. In this study, UGN-102 was suitable to administer at home by a visiting nurse under the supervision of a treating physician and resulted in 75% (n=8) of patients achieving a complete response, defined as no detectable disease 3 months after starting treatment.
JELMYTO (mitomycin) for pyelocalyceal solution in low-grade Upper Tract Urothelial Cancer (LG-UTUC):

JELMYTO generated net product revenues of $18.1 million and $64.4 million for the fourth quarter and full year 2022, respectfully, compared to $16.2 million and $48.0 million for the same periods in 2021.
Activated sites on March 1, 2023 were 983, compared to 930 on November 1, 2022, while repeat accounts on March 1, 2023 were 214, compared to 177 on November 1, 2022.
Highlighted results from the first and largest post-commercial utilization review of JELMYTO published in Urologic Oncology: Seminars and Original Investigations. The study evaluated 132 patients from 15 high-volume academic and community centers and describes several uses of JELMYTO that differ from the pivotal OLYMPUS trial, including for the treatment of large tumors (>3cm in 15% of cases), high grade tumors (9% of cases), ureteral tumors (35% of cases), and as a valuable multimodal adjunct following complete endoscopic ablation. JELMYTO is not approved for the treatment of high-grade upper tract urothelial cancer.
Shared new long-term follow up data from the OLYMPUS registration trial at the Annual Society of Urologic Oncology (SUO) Meeting demonstrating a median durability of response (DOR) of 28.9 months (14.6 to 47.6 months) with JELMYTO in 16 of 23 evaluable patients.
Retrospective, multi-center study of 32 patients evaluating the safety of antegrade administration of JELMYTO was published in The British Journal of Urology International showing a favorable safety and tolerability profile for JELMYTO when administered via nephrostomy tube. At a median follow-up of 15 months following initiation of induction therapy, ureteral stenosis occurred in only three (9%) patients, with none having recurrent stenosis at a median 16-month follow-up.
Fourth Quarter and Full Year 2022 Financial Results:

JELMYTO Revenue: UroGen reported JELMYTO net product revenues for the fourth quarter 2022 of $18.1 million, compared to $16.2 million for the same period in 2021. Net product revenues were $64.4 million for the full year 2022, compared to $48.0 million for the full year 2021.

R&D Expense: Research and development expenses for the fourth quarter 2022 were $14.5 million, including non-cash share-based compensation expense of $0.6 million as compared to $13.1 million, including non-cash share-based compensation expense of $0.9 million, for the same period in 2021. Research and development expenses for the full year 2022 were $52.9 million, including non-cash share-based compensation expense of $2.6 million. This compares to $47.6 million, including non-cash share-based compensation expense of $4.0 million, for the full year 2021.

SG&A Expense: Selling, general and administrative expenses for the fourth quarter 2022 were $21.6 million, including non-cash share-based compensation expense of $1.8 million. This compares to $21.4 million, including non-cash share-based compensation expense of $4.5 million, for the same period in 2021. Selling, general and administrative expenses for the full year 2022 were $82.8 million, including non-cash share-based compensation expense of $8.0 million. This compares to $87.5 million, including non-cash share-based compensation expense of $19.1 million for the full year 2021.

Financing on Prepaid Forward Obligation: UroGen reported financing expense related to the prepaid forward obligation to RTW Investments of $5.1 million for the fourth quarter 2022. Financing expense related to the prepaid forward obligation to RTW Investments totaled $21.6 million for the full year 2022. The rate for 2023 will be 13% based on $64.4 million of global net product sales of JELMYTO in 2022.

Net Loss: UroGen reported a net loss of $28.2 million, or basic and diluted net loss per ordinary share of $1.22, for the fourth quarter 2022 as compared to $28.5 million, or basic and diluted net loss per ordinary share of $1.27, for the same period in 2021. UroGen reported a net loss of $109.2 million, or basic and diluted net loss per ordinary share of $4.79, for the full year 2022 versus $110.8 million, or basic and diluted net loss per ordinary share of $4.96, for the full year 2021. Financial results subsequently filed on Form 10-K for the year ended December 31, 2022 may include immaterial changes related to income tax expense, which is still under review due to ongoing assessment of taxes as it relates to the $100 million term loan from funds managed by Pharmakon Advisors.

Cash & Cash Equivalents: As of December 31, 2022, cash, cash equivalents and marketable securities totaled approximately $100 million.

2023 Revenue, Operating Expense and Financing Expense Guidance: UroGen anticipates full year 2023 net product revenues from JELMYTO to be in the range of $76 to $86 million. UroGen anticipates full year 2023 operating expenses in the range of $135 to $145 million, including non-cash share-based compensation expense of $6.0 to $11.0 million, subject to market conditions. UroGen anticipates full year 2023 financing expense related to the prepaid obligation to RTW Investments in the range of $21.0 to $26.0 million, of which approximately $9.8 to $11.1 million will be in cash. Interest only payments on the $100 million term loan facility with funds managed by Pharmakon Advisors will be made quarterly and continue to accrue at a rate of LIBOR (or a replacement benchmark following the cessation of LIBOR in the first half of this year) + 8.25%.