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Pieris Announces Amendment of Existing Immuno-oncology Multi-target Collaboration with Seagen, a Clinical Trial and Supply Agreement to Evaluate Cinrebafusp Alfa (PRS-343) in Combination with TUKYSA® (tucatinib) in Gastric Cancer, and Strategic Equity Investment by Seagen

On March 25, 2021 Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer, and other indications, reported that Seagen has made a strategic equity investment in Pieris as part of an ongoing collaboration between the companies (Press release, Pieris Pharmaceuticals, MAR 25, 2021, https://ir.pieris.com/news/detail/655/pieris-announces-amendment-of-existing-immuno-oncology-multi-target-collaboration-with-seagen-a-clinical-trial-and-supply-agreement-to-evaluate-cinrebafusp-alfa-prs-343-in-combination-with-tukysa-tucatinib-in-gastric-cancer-and-strategic-equity-investment-by-seagen [SID1234577151]). In addition, the companies have entered into a clinical trial collaboration agreement to evaluate the safety and efficacy of combining Pieris’ cinrebafusp alfa (PRS-343), a 4-1BB/HER2 bispecific, with Seagen’s TUKYSA (tucatinib), a small-molecule tyrosine kinase HER2 inhibitor, for the treatment of gastric cancer patients expressing lower HER2 levels (IHC2+/ISH- & IHC1+) as part of the upcoming phase 2 study to be conducted by Pieris. The companies have also amended their existing immuno-oncology collaboration agreement around joint development and commercial rights for the second of up to three products in the alliance.

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The combination of cinrebafusp alfa and TUKYSA could potentially address a high medical need in HER2 low-expressing gastric cancer patients who do not respond to traditional HER2-targeted therapies. Preclinical studies show that TUKYSA synergizes with cinrebafusp alfa to enhance its 4-1BB-mediated immune cell stimulation. This effect was observed across a range of HER2 expressing cell lines (IHC3+, 2+, and 1+), including those where cinrebafusp alfa had limited single-agent activity.

Under the amended and restated agreement, Pieris’ option to co-develop and co-commercialize the second of three programs in the collaboration has been amended to provide it with a co-promotion option in the United States, with Seagen solely responsible for the development and overall commercialization of that program. Under the co-promotion option, Pieris will also be entitled to increased royalties from that program in the event that it chooses to exercise the option. In connection with the amendment, on March 24, 2021, in a private placement transaction, Seagen made an equity investment of $13 million in Pieris through the purchase of 3,706,174 newly issued shares of Pieris common stock at a price of $3.51 per share.

"Seagen continues to be a supportive partner, and we look forward to combining efforts in studying the effects of cinrebafusp alfa with TUKYSA in gastric cancer patients expressing lower HER2-levels, following the generation of compelling preclinical data," said Stephen S. Yoder, President and Chief Executive Officer of Pieris. "We plan to initiate this combination study as part of our phase 2 protocol for cinrebafusp alfa, for which we will be sharing additional details at an upcoming corporate update."

"Preclinical data exploring the combination of cinrebafusp alfa and TUKYSA are encouraging and support evaluating the combination in the planned phase 2 clinical trial," said Marjorie Green, M.D., Senior Vice President, Late-Stage Development of Seagen. "We are pleased to supply drug for Pieris to explore the potential combination of these agents to address an important unmet medical need."

About Cinrebafusp Alfa
Cinrebafusp alfa (PRS-343) is a 4-1BB/HER2 fusion protein comprising a 4-1BB-targeting Anticalin protein and a HER2-targeting antibody. The drug candidate is currently in development for the treatment of HER2-positive solid tumors. Based on encouraging phase 1 study results, which demonstrated clinical benefit as single agent and biomarker data indicative of a 4-1BB-driven mechanism of action, the Company is actively working towards initiating a phase 2 study of cinrebafusp alfa in combination with ramucirumab and paclitaxel for the treatment of HER2-positive gastric cancer and, under the phase 2 protocol, in combination with tucatinib.

VBL Therapeutics Reports Fourth Quarter and Full Year 2020 Financial Results and Provides Corporate Update

On March 25, 2021 VBL Therapeutics (Nasdaq: VBLT) (the Company) reported financial results for the fourth quarter and fiscal year ended December 31, 2020 and provided a corporate update (Press release, VBL Therapeutics, MAR 25, 2021, View Source [SID1234577150]).

"2020 was year of significant progress for VBL as we reached several milestones across multiple assets in different stages of development, including a successful interim analysis and two subsequent successful DSMC reviews for the OVAL Phase 3 registration enabling study of VB-111 for the treatment of platinum-resistant ovarian cancer. We look forward to continuing this momentum in 2021," said Dror Harats, M.D., Chief Executive Officer of VBL Therapeutics.

Fourth Quarter and Recent Corporate Highlights

VB-111

In December 2020, the Company announced that the first patient has been enrolled in Europe in the OVAL Phase 3 registration enabling study of VB-111 for the treatment of platinum-resistant ovarian cancer.
In February 2021, a successful pre-planned DSMC review of the OVAL study found no safety issues with the trial and recommended its continuation as planned.
In March 2021, the Company announced the initiation of randomized, controlled and blinded Phase 2 investigator-sponsored trial of VB-111 in patients with recurrent glioblastoma.
VB-201

In January 2021, the Company announced the dosing of the first patient in a randomized controlled Phase 2 study of the Company’s proprietary investigational oral immune-modulator molecule, VB-201, for the treatment of COVID-19.
Corporate

In October 2020, the Company appointed Marc Kozin Vice Chairman of its Board of Directors.
In January 2021, the Company entered into an ordinary share purchase agreement of up to $20 million with Aspire Capital Fund LLC.
Financial Results for the Fourth Quarter and Full Year

As of December 31, 2020, the Company had cash, cash equivalents, short-term bank deposits and restricted bank deposit totaling $30.8 million and working capital of $24.5 million. The Company expects that its cash and cash equivalents and short-term bank deposits will be sufficient to fund operating expenses and capital expenditure requirements into the fourth quarter of 2022.

Revenues were $922 thousand for fiscal year 2020, as compared to $562 thousand for fiscal year 2019.

R&D expense was $19.7 million for fiscal year 2020, as compared to $14.7 million for fiscal year 2019. The increase in R&D expense was mainly due to the development of the VB-601 towards Investigational New Drug enabling studies.

G&A expenses were $5.3 million for fiscal year 2020, compared to $5.7 million for fiscal year 2019.

VBL reported a net loss of $24.2 million, or ($0.55) per share, for fiscal year 2020, compared to a net loss of $19.4 million, or ($0.54) per share, for fiscal year 2019.

The live webcast will be available online and may be accessed from the "Events and Presentation" page of the Company website. A replay of the webcast will be available beginning approximately one hour after the conclusion of the call and will remain available for at least 30 days thereafter.

Theralase Launches Sixth Clinical Study Site in the US for Phase II Bladder Cancer Clinical Study

On March 25, 2021 Theralase Technologies Inc. ("Theralase" or the "Company") (TSXV: TLT) (OTCQB: TLTFF), a clinical stage pharmaceutical company focused on the research and development of light activated Photo Dynamic Compounds ("PDC") and their associated drug formulations used to safely and effectively destroy various cancers, bacteria and viruses reported that Urology San Antonio ("USA") has received site Institutional Review Board ("IRB") approval to commence a Pivotal Phase II Non-Muscle Invasive Bladder Cancer ("NMIBC") Clinical Study to enroll and treat patients with Bacillus Calmette Guerin("BCG")-Unresponsive Carcinoma In-Situ ("CIS") or who are intolerant to BCG Therapy ("Study II") (Press release, Theralase, MAR 25, 2021, View Source [SID1234577149]).

This marks the sixth US Clinical Study Site ("CSS") that has obtained site IRB approval through a central IRB. Theralase is at different stages with additional US clinical study sites that are expected to launch in 2Q21 and 3Q21.

Urology San Antonio is the largest urology practice in South Texas with multiple locations offering care for men and women experiencing complications of the urinary system. Their subspecialists develop incredible expertise in different aspects of urologic care and can offer patients treatment options and guidance not commonly seen in a community urology practice. Dr. Daniel Saltzstein MD will be the Study II Principal Investigator for this site.

To date 16 patients have been treated in Study II. The Company has now launched 5 CSS’s in Canada and 6 in the US for patient enrollment and treatment under Study II clinical study guideline.

Shawn Shirazi PhD, Chief Executive Officer, Theralase, stated, "It is exciting to see Theralase hit its target of having 6 US clinical sites on board in 1Q 2021. It is truly uplifting to be working on the details of getting patients enrolled and treated at these sites. Theralase is another step closer in achieving its next milestone of enrolling and treating 9 additional patients in early 2021 to meet the target of 25 patients for potential Breakthrough Designation."

Japan’s Ministry of Health, Labour and Welfare Approves Breyanzi, a New CAR T Cell Therapy

On March 25, 2021 Bristol Myers Squibb K.K. reported that Japan’s Ministry of Health, Labour and Welfare (MHLW) approved Breyanzi (lisocabtagene maraleucel: liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy for the treatment of patients with relapsed or refractory (R/R) large B-cell lymphoma1 and R/R follicular lymphoma (Press release, Bristol-Myers Squibb, MAR 25, 2021, View Source [SID1234577148]).2

The approval is based on efficacy and safety from the TRANSCEND NHL 001 trial in patients with R/R B-cell non-Hodgkin lymphoma (NHL) and the TRANSCEND WORLD trial in patients with R/R aggressive B-cell NHL.

Large B-cell lymphoma comprises several disease types including diffuse large B-cell lymphoma (DLBCL). DLBCL is the most common form of non-Hodgkin lymphoma in Japan, accounting for 30-40% of all B-cell cases diagnosed,3 and is especially prevalent among people in their 60’s.4 There is currently no established standard-of-care treatment for patients with R/R large B-cell lymphoma,5 which underscores the need for new treatments for in this disease area. Follicular lymphoma accounts for 10-20% of all B-cell NHL cases in Japan. Patients initially respond to chemotherapy, but relapse is common, especially in advanced-stage patients.5 There is also no established standard-of-care treatment for patients with follicular lymphoma grade 3B.5

Jean-Christophe Barland, President and CEO of Bristol-Myers Squibb K.K. and Celgene K.K., said, "I am pleased that we have received regulatory approval in Japan for Breyanzi, our first CAR T cell therapy, which will allow us to provide a new treatment option for patients fighting relapsed or refractory large B-cell lymphoma and relapsed or refractory follicular lymphoma. In addition, we are filing an application for a further CAR T cell therapy to address more unmet medical needs. As a game-changer committed to ‘innovation with heart’, Bristol Myers Squibb will continue on its journey to help patients prevail over serious diseases."

Breyanzi is a chimeric antigen receptor T cell therapy designed to target CD19, which is expressed on the cell during normal B-cell development and maintained even after malignant transformation of B cells. Breyanzi aims to target CD19-expressing cells and is administered in a one-time infusion as a defined, purified composition to reduce variability of the CD8 and CD4 component dose. Breyanzi will be manufactured at Bristol Myers Squibb’s cellular immunotherapy manufacturing facility in Bothell, Washington and at a partner company facility in Japan.

Breyanzi was approved by the U.S. Food and Drug Administration on February 5, 2021 for the treatment of adult patients with relapsed or refractory large B-cell lymphoma (LBCL) after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma), high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B. Breyanzi is not indicated for the treatment of patients with primary central nervous system lymphoma. Breyanzi has been granted Priority Medicines (PRIME) designation for relapsed or refractory DLBCL in the European Union and a Marketing Authorization Application is currently under review by the European Medicines Agency.

TRANSCEND NHL 001 / TRANSCEND WORLD Trial Key Results

In the TRANSCEND NHL 001 trial, of 133 subjects in the primary efficacy evaluation population, the overall response rate [95% CI], which was the primary endpoint, was 74.4% [66.2, 81.6] (April 12, 2019 data cutoff), and the lower limit of the 95% CI therefore exceeded the protocol-defined threshold overall response rate of 40%. In addition, the overall response rate [95% CI] in the 256 subjects in the efficacy analysis population treated with anti-CD19 CAR T cells was 72.7% [66.8, 78.0] (August 12, 2019 data cutoff). In the TRANSCEND WORLD trial, of 34 subjects who received BREYANZI, the overall response rate [95% CI], which was the primary endpoint, was 58.8% [40.7, 75.4] (September 13, 2019 data cutoff), which was statistically significant compared to the threshold value of 40%. In addition, the overall response rate [95% CI] in the 10 Japanese subjects was 70.0% [34.8, 93.3]. Moreover, the overall response rates [95% CIs] in the 46 subjects in the study population overall and in the 10 Japanese subjects at the June 19, 2020 data cutoff were 63.0% [47.5, 76.8] and 70.0% [34.8, 93.3], respectively.

In the TRANSCEND NHL 001 trial, 269 patients treated with Breyanzi were evaluated for safety. Adverse reactions occurred in 201 patients including cytokine release syndrome (42.0%), fatigue (17.8%), neutropenia (16.4%), anemia (13.8%), headache (13.4%), thrombocytopenia (11.5%), confusion (11.5%), tremor (11.2%), and hypotension (10.4%) (aggregated until approval). Adverse reactions occurred in 42 patients out of 46 (including 10 Japanese) treated with Breyanzi in the TRANSCEND WORLD trial. Adverse reactions included neutropenia (52.2%), cytokine releasesyndrome (41.3%), anemia (39.1%), thrombocytopenia (39.1%), pyrexia (39.1%), leukopenia (23.9%), confusion (15.2%), fatigue (13.0%), and febrile neutropenia (13.0%) (aggregated until approval).

Bristol Myers Squibb: Creating a Better Future for People with Cancer

Bristol Myers Squibb is inspired by a single vision—transforming patients’ lives through science. The goal of the company’s cancer research is to deliver medicines that offer each patient a better, healthier life and to make cure a possibility. Building on a legacy across a broad range of cancers that have changed survival expectations for many, Bristol Myers Squibb researchers are exploring new frontiers in personalized medicine, and through innovative digital platforms, are turning data into insights that sharpen their focus. Deep scientific expertise, cutting-edge capabilities and discovery platforms enable the company to look at cancer from every angle. Cancer can have a relentless grasp on many parts of a patient’s life, and Bristol Myers Squibb is committed to taking actions to address all aspects of care, from diagnosis to survivorship. Because as a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future.

Phio Pharmaceuticals Reports 2020 Year End Financial Results and Provides Business Update

On March 25, 2021 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (INTASYL) therapeutic platform, reported its financial results for the year ended December 31, 2020 and provided a business update (Press release, Phio Pharmaceuticals, MAR 25, 2021, View Source [SID1234577147]).

"Over the past year, we established steady momentum across our development programs and expanded our pipeline, including our new collaboration with AgonOx, Inc. to develop T cell-based cancer immunotherapies using PH-762. This progress has resulted in a steady stream of preclinical data, which we plan to present for several programs throughout 2021. Also, we are on track to initiate two clinical studies for our lead asset, PH-762, later this year," said Dr. Gerrit Dispersyn, President and CEO of Phio. "In support of the planned initiation of two clinical programs for different applications of PH-762, and of the advancement of our broader development pipeline, we recently completed two equity financings for additional gross proceeds of $21.7 million. Our stronger financial footing is expected to fund our current programs for the next two years, including clinical studies on the safety and efficacy of PH-762 in adoptive cell therapy and with direct drug therapy."

Quarter in Review and Recent Corporate Updates

Presented data at SITC (Free SITC Whitepaper) 2020 showing that the antitumoral efficacy of our PH-762, PH-790 and PH-804 INTASYL compounds can be further improved by combining them in a single drug treatment.
Presented data resulting from our collaborations with AgonOx, Inc. and the Helmholtz Zentrum München, including poster presentations at SITC (Free SITC Whitepaper) 2020.
Announced a collaboration for the clinical development of novel T cell-based cancer immunotherapies using PH-762 and AgonOx, Inc.’s "double positive" (DP) tumor-infiltrating lymphocyte (TIL) technology. Data developed in collaboration with AgonOx, Inc. showed PH-762 can improve the tumor cell killing activity of DP TILs two-fold.
Bolstered the balance sheet with two financings in Q1 2021 for gross proceeds of $21.7 million in additional capital.
Completed a $7.7 million registered direct offering of common stock priced at-the-market.
Completed a $14.0 million private placement of common stock and warrants priced at-the-market.
Upcoming Pipeline Milestones for 2021

Scheduled to present new study data regarding direct drug therapy with PH-762 at the AACR (Free AACR Whitepaper) Annual Meeting 2021.
Additional data publications on the Company’s pipeline programs, expected during Q2-Q3 2021.
Start of a first-in-human clinical study, on the use of PH-762 in adoptive cell therapy with TILs, namely, to enhance the therapeutic responses in cancer patients. The study, to be executed in collaboration with Agonox, Inc., is expected to be initiated in Q3 2021.
Start of a first-in-human clinical study on the use of PH-762 as directly administered drug in patients with advanced melanoma. The study is expected to be initiated in Q4 2021.
Financial Results

Cash Position

At December 31, 2020, the Company had cash of $14.2 million as compared with $6.9 million at December 31, 2019. Subsequent to the end of the fourth quarter of 2020, the Company raised an additional $21.7 million in gross proceeds through a registered direct offering and a private placement offering. The Company expects its current cash will be sufficient to fund currently planned operations to the second quarter of 2023.

Research and Development Expenses

Research and development expenses were approximately $4.4 million for the year ended December 31, 2020, compared to approximately $4.3 million for the year ended December 31, 2019. The increase is primarily due to an increase in the use of sponsored research organizations to support the development of the Company’s pipeline programs as compared to the prior year period.

General and Administrative Expenses

General and administrative expenses were approximately $4.4 million for the year ended December 31, 2020, compared to approximately $4.7 million for the year ended December 31, 2019. The decrease is primarily due to decreases in legal-related expenses and recruiting fees to support employee hiring activities as compared to the prior year period.

Net Loss

Net loss was $8.8 million, or $1.92 per share, for the year ended December 31, 2020, compared with $8.9 million, or $19.33 per share, for the year ended December 31, 2019. The decrease in net loss was primarily attributable to the decrease in general and administrative expenses, as described above. The change in net loss per share was primarily due to an increase in the number of shares outstanding as a result of our capital raise activities as compared to the prior year period.