On May 7, 2020 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported first quarter 2020 financial results (Press release, Oncternal Therapeutics, MAY 7, 2020, View Source [SID1234557299]).

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"Despite the COVID-19 pandemic, we are pleased to see continuing progress for our promising oncology pipeline, as we recently reported deep clinical responses in 28.5% of patients with Ewing sarcoma treated with TK216 at the selected Phase 2 dose with or without vincristine, and a 50% complete response ("CR") rate in patients with Mantle Cell Lymphoma ("MCL") treated with cirmtuzumab plus ibrutinib," said James Breitmeyer, M.D., Ph.D., President and CEO, Oncternal. "Our team has worked closely with our clinical investigators to help ensure the safety of the patients and the clinical study staff, while preserving regulatory compliance and data quality. At this time, we do not expect delays to our previously articulated clinical data read-outs in 2020."

Recent Highlights

In April 2020, we announced an interim clinical data update for our ongoing, open-label, multicenter Phase 1 clinical trial of TK216, a targeted investigational small-molecule inhibitor of the E26 transformation-specific ("ETS") family of oncoproteins, in patients with relapsed or refractory Ewing sarcoma. As of the data cut-off date of March 26, 2020, seven patients treated at the recommended Phase 2 dose ("RP2D") of TK216 (200 mg/m2/day for 14 days without or with vincristine) were evaluable for clinical responses. Two of the seven patients (28.5%) had achieved partial responses ("PR"), one with a surgical CR, and one with 90% shrinkage of target lesions. Both responding patients remain on treatment, including one who has no evidence of disease after more than twelve months on study. Two patients had stable disease and three had progressive disease, for an overall clinical benefit of 57% (4/7).

In March 2020, we announced an interim clinical data update for cirmtuzumab, a monoclonal antibody targeting ROR1, or Receptor tyrosine kinase-like Orphan Receptor 1, in combination with ibrutinib in patients with relapsed/refractory MCL enrolled in our ongoing Phase 1/2 clinical trial. As of the data cut-off of March 6, 2020, we reported a 50% CR rate for the 12 evaluable patients and an 83% best objective response rate ("ORR", including CR or PR). Responses were determined by Cheson criteria, and one of the six patients with CR had a complete metabolic response by PET scan, with a bone marrow biopsy pending.

In February 2020, we presented ROR1 CAR-T preclinical data at the ASCO (Free ASCO Whitepaper)-SITC Clinical Immuno-Oncology Symposium. ROR1 CAR-T cell therapy demonstrated expansion, persistence and anti-tumor activity in an animal model of ROR1-expressing human leukemia. This research is being conducted by our collaborators at the University of California San Diego (UC San Diego) under a grant from the California Institute of Regenerative Medicine ("CIRM").

Oncternal’s first priority during the unprecedented public health emergency caused by the COVID-19 pandemic has been protecting the safety of the patients in our clinical trials, along with the safety of the staff at clinical sites and that of our own employees, while ensuring regulatory compliance and data integrity. Enrollment of new patients into our studies has slowed down, as most other drug developers are reporting, but we have continued to enroll additional patients into the Ewing sarcoma study recently despite COVID-19 related constraints. As of today, very few patients have discontinued their participation in our clinical trials because of COVID-19 concerns, and we remain on track to meet our previously disclosed expected clinical data milestones for 2020.

Expected Upcoming Milestones

TK216 program

Clinical data for 7-12 patients with Ewing sarcoma treated in the Phase 1 expansion cohort – in the second half of 2020

IND-enabling data in additional ETS-driven tumors – in the second half of 2020

Cirmtuzumab program

Clinical data update for patients with MCL, including for 15 or more patients treated with cirmtuzumab plus ibrutinib in the ongoing Phase 1/2 study – in mid-2020

Clinical data update for patients with CLL, including 12-month follow-up for up to 34 patients treated with cirmtuzumab plus ibrutinib in the ongoing Phase 1/2 study – in mid-2020

Clinical data update for patients with HER2-negative breast cancer in the ongoing Phase 1b study – in the second half of 2020

IND-enabling data in additional ROR1 expressing tumors – in the second half of 2020

ROR1 CAR-T program

First-in-human dosing in China – in the first half of 2021

First Quarter 2020 Financial Results

Our grant revenue was $0.6 million for the first quarter ended March 31, 2020. Our grant revenue is derived from a sub-award under a grant from CIRM to UC San Diego, which was awarded to advance our Phase 1/2 clinical trial evaluating cirmtuzumab in combination with ibrutinib for the treatment of patients with MCL or CLL.

Our total operating expenses for the first quarter ended March 31, 2020 were $5.3 million. Research and development expenses for the quarter totaled $2.7 million, and general and administrative expenses for the quarter totaled $2.6 million. Net loss for the first quarter was $4.7 million, or a loss of $0.31 per share, basic and diluted.

As of March 31, 2020, we had $16.0 million in cash and cash equivalents. We believe these funds will be sufficient to fund our operations into the fourth quarter of 2020. As of March 31, 2020, we had approximately 15.4 million shares of common stock outstanding.

Management Webcast

As previously announced, Oncternal will host a webcast today, May 7, 2020, at 3:00 p.m. ET (12:00 p.m. PT). The live webcast will be available online and may be accessed from the "Investors" page of the company website at View Source 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.

On May 7, 2020 Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results for the first quarter ended March 31, 2020 and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, MAY 7, 2020, View Source [SID1234557298]).

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"We are humbled by the tenacity and commitment of the patients and clinicians with whom we have the privilege to work as we continue to adjust to the challenges of the COVID-19 pandemic," said Nagesh Mahanthappa, Ph.D., President and CEO of Scholar Rock. "It is with a primary focus on the safety and well-being of our patients, clinical investigators and employees, that we are committed to continuing the execution of the SRK-015 TOPAZ Phase 2 and the SRK-181 DRAGON Phase 1 trials. Though we may continue to experience uncertainty during these unprecedented times, Scholar Rock is planning for additional updates on both programs later this year."

Company Updates and Upcoming Milestones

SRK-015 Program for Spinal Muscular Atrophy (SMA):

Anticipate Interim Efficacy and Safety Data from the TOPAZ Phase 2 Trial in the Fourth Quarter of 2020. COVID-19-related restrictions have impacted patient access to clinical trial sites, which have led to missed or delayed doses or assessments (including efficacy) in some patients and has affected Scholar Rock’s ability to monitor trial data collected by sites. As a result, Scholar Rock now plans to report interim efficacy, safety, pharmacokinetic (PK), and pharmacodynamic (PD) results in the fourth quarter of 2020 for patients across the three cohorts who have progressed through at least six months of the treatment period. This is an approximately one quarter delay compared to the previous guidance of interim 6-month results in mid-2020.

As of May 1, 2020, the majority of patients have either completed or are on track to complete their 6-month visit for the interim efficacy and safety analysis. For patients who have had their 6-month visit impacted, Scholar Rock is working closely with clinical trial sites on scheduling to minimize any additional delays in dosing and assessments.
Enrollment of the TOPAZ proof-of-concept trial was completed in January 2020. 58 patients with Type 2 or Type 3 SMA were enrolled across the three cohorts (23 patients in Cohort 1, 15 patients in Cohort 2, and 20 patients in Cohort 3).
To date, one patient (Cohort 1) has discontinued from the trial for reasons unrelated to the study drug and which occurred prior to the COVID-19 pandemic.
As of May 1, 2020, 41 of 57 patients have completed the 6-month visit for the interim efficacy and safety analysis.
As of May 1, 2020, 51 of 57 patients have completed the 5-month visit.
Approximately 70% of patients are enrolled in the U.S. and the remaining 30% of patients are enrolled in Europe, including Italy, Spain, and Netherlands.

Top-line data for the 12-month treatment period are now expected in the first half of 2021 as compared to the previous guidance of the fourth quarter of 2020 and first quarter of 2021. There may be further impacts on the timing of future doses and assessments for patients in the trial, as the effects of the COVID-19 pandemic continue to evolve.
Identification of Second Indication for SRK-015 Planned for 2020. Scholar Rock continues to evaluate multiple potential opportunities beyond SMA, for which the selective inhibition of the activation of myostatin with SRK-015 may offer therapeutic benefit.
SRK-181 Program for Immuno-Oncology:

Update on Dose Escalation from the DRAGON Phase 1 Proof-of-Concept Trial in Solid Tumors Expected in the Fourth Quarter of 2020. SRK-181 is a potent and highly selective inhibitor of latent transforming growth factor beta 1 (TGFβ1) activation and is being developed with an aim of expanding anti-tumor responses from immunotherapy by overcoming primary resistance to anti-PD-1 or anti-PD-L1 therapy. The DRAGON Phase 1 dose escalation and dose expansion trial evaluating SRK-181 in patients with locally advanced or metastatic solid tumors was initiated in the first quarter of 2020. Scholar Rock continues to activate clinical trial sites and recently commenced patient enrollment and dosing. Update on dose escalation of SRK-181 as a single agent as well as in combination with anti-PD-(L)1 therapy is expected in the fourth quarter of 2020, subject to the pace of enrollment as impacted by the COVID-19 pandemic. Clinical response and safety data are anticipated in 2021. Timing of data read-outs may be further impacted by the COVID-19 pandemic.

The two-part DRAGON trial consists of a dose escalation portion (Part A) for SRK-181 as both a single-agent and in combination with an approved anti-PD-(L)1 therapy, followed by a dose expansion portion (Part B) evaluating SRK-181 in combination with an approved anti-PD-(L)1 therapy in patients with solid tumors exhibiting primary resistance to that anti-PD-(L)1 therapy. Part B will encompass multiple cohorts that are expected to include urothelial carcinoma, cutaneous melanoma, non-small cell lung cancer, and other solid tumors. Patients will be administered SRK-181 IV every 3 weeks (Q3W), and additional dosing regimens may be explored in the future. Key objectives of the study include evaluating the efficacy, PK, and safety of SRK-181.
Published Preclinical Data Detailing a Potent and Selective Inhibitor of TGFβ1 Overcoming Primary Resistance to Checkpoint Inhibition. In March 2020, Scholar Rock’s preclinical data establishing the therapeutic rationale for evaluating a potent and highly selective inhibitor of TGFβ1 activation to overcome primary resistance to checkpoint inhibitor therapy were published in the peer-reviewed journal Science Translational Medicine(1). Selective inhibition of latent TGFβ1 activation with SRK-181 has demonstrated in preclinical studies the potential to overcome primary resistance, which, if replicated in clinical studies, could meaningfully expand the number of patients who could benefit from checkpoint inhibitor therapy. In preclinical studies, an improved safety profile was observed for SRK-181 as compared to conventional inhibitors of TGFβ signaling.
RGMc Program for Iron-Restricted Anemias:

Nomination of a Product Candidate from the RGMc Program Planned in 2020. Scholar Rock is evaluating a number of highly specific inhibitors of repulsive guidance molecule C (RGMc) and plans to nominate an antibody as its third product candidate in 2020. RGMc’s known function is localized to hepatocytes and the identification of RGMc selective-antibodies may offer the potential for liver-specific modulation of BMP6 signaling to address iron-restricted anemias.
Additional Corporate Updates:

Chief Scientific Officer, Alan Buckler, Ph.D., to Step Down Effective June 1, 2020.
Alan Buckler, Ph.D., has decided to leave Scholar Rock to pursue other opportunities and will remain in his position through June 1 to ensure a smooth transition. Alan has been instrumental in helping to build a highly talented research team that has established Scholar Rock’s proprietary discovery platform as well as advanced two product candidates through research, discovery and preclinical development into the clinic.

On June 1, 2020, Gregory Carven, Ph.D., current Senior Vice President of Biologics, will be named to the new position of Head of Research to continue delivering on the Company’s commitment to drug discovery. Gregory is a gifted scientist with more than 15 years of experience in the discovery and development of antibody therapeutics and is a co-inventor of pembrolizumab. Since joining Scholar Rock in 2014, Gregory has led the Company’s biologics discovery, design, and manufacturing initiatives and played a central role in the discovery and preclinical development of both the SRK-015 and SRK-181 product candidates.
Announced Issuance of U.S. Patent Broadly Relevant to Antibodies that Modulate TGFβ Activation. The United States Patent Office (USPTO) issued U.S. Patent No. 10,597,443 with an expiry of May 2034 broadly covering methods for making activation modulators of TGFβ that utilize Scholar Rock’s proprietary platform approach of targeting the precursor form of growth factors. This broadly relates to TGFβ activation inhibitors that are specific to the context of presenting molecules, such as GARP and LTBP, as well as activation inhibitors that are not dependent on a specific presenting molecule, such as SRK-181.
First Quarter 2020 Financial Results

For the quarter ended March 31, 2020, net loss was $17.1 million or $0.58 per share compared to a net loss of $10.8 million or $0.42 per share for the quarter ended March 31, 2019.

Revenue was $5.0 million for the quarter ended March 31, 2020 and was related to the Gilead fibrosis-focused collaboration (the "Gilead Collaboration Agreement") that was executed in December 2018.
Research and development expense was $16.9 million for the quarter ended March 31, 2020 compared to $10.7 million for the quarter ended March 31, 2019. The increase year-over-year primarily reflects manufacturing costs and costs associated with the initiation of the DRAGON Phase 1 clinical trial for SRK-181, costs associated with the TOPAZ Phase 2 clinical trial for SRK-015, and higher personnel-related costs, slightly offset by lower early development costs.
General and administrative expense was $5.8 million for the quarter ended March 31, 2020 compared to $4.1 million for the quarter ended March 31, 2019. The increase year-over-year was primarily attributable to increased headcount and professional services.
As of March 31, 2020, Scholar Rock had cash, cash equivalents, and marketable securities of $160.6 million, which is inclusive of a $25 million payment from Gilead for the achievement of the preclinical milestone under the Gilead Collaboration Agreement. This compares to cash, cash equivalents, and marketable securities of $157.4 million as of December 31, 2019.

(1)
Martin, C., Datta, A., Schürpf, T., et al. Selective inhibition of TGFβ1 activation overcomes primary resistance to checkpoint blockade therapy by altering tumor immune landscape, Science Translational Medicine, 2020 Mar 25; 12(536), eaay8456.

On May 7, 2020 Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, reported financial results for the first quarter ended March 31, 2020 (Press release, Zymeworks, MAY 7, 2020, View Source [SID1234557297]).

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"While the COVID-19 pandemic has presented unprecedented global challenges, the entire Zymeworks team remains committed to our mission of returning patients home to their loved ones, disease free," said Ali Tehrani, Ph.D., Zymeworks’ President & CEO. "The clinical trials for our lead candidates, ZW25 and ZW49, provide potential options for patients whose cancer has become refractory to the standard of care. I am extremely proud of how our team, our collaborators, and our clinical investigators have responded to ensure that patients enrolled in our studies continue to receive their treatments as we advance these therapies through the clinic."

Dr. Tehrani added, "With a strong balance sheet and runway into 2022, substantial drug supply, and clinical trials in diverse sites around the globe, Zymeworks is well-prepared to continue moving forward through these challenging times. We look forward to providing a corporate update midyear to share our progress."

COVID-19 Business Update

Zymeworks continues to closely monitor the COVID-19 pandemic and adapt its business operations while prioritizing the health and well-being of patients, clinical investigators, and personnel. In accordance with recommendations from health authorities, Zymeworks has transitioned to a remote working arrangement to protect employees and the broader community while maintaining business continuity. All clinical trial sites remain open and active with a heightened focus on patient safety and data integrity. While the effects of the pandemic are expected to slow the pace of patient recruitment due to the diversion of healthcare resources to COVID-19 response activities, they have not had a material impact on the company’s financial condition, liquidity, or longer-term strategic development and commercialization plans. Following the guidance of local health authorities, Zymeworks has begun implementing plans for employees to return working on site.

First Quarter 2020 Business Highlights and Recent Developments

•Strong Financial Position; Extended Runway
Zymeworks completed an upsized public financing with gross proceeds of US$320.8 million and ended the first quarter with $562.7 million in cash resources. Based on current operating plans, Zymeworks expects to have cash to fund research and development programs and operations into 2022 and potentially beyond.

•ZW25 Advances in Three New Global Clinical Trials
Zymeworks’ partner BeiGene dosed the first patient in a Phase 1b/2 trial evaluating ZW25 in combination with chemotherapy as a first-line treatment for patients with metastatic HER2-positive breast cancer and in combination with chemotherapy and BeiGene’s PD-1-targeted antibody tislelizumab as a first-line treatment for patients with metastatic HER2-positive gastroesophageal adenocarcinoma (GEA). In addition, Zymeworks initiated a Phase 2 trial and collaboration with Pfizer to evaluate ZW25 in combination with palbociclib and fulvestrant in HER2-positive, hormone receptor-positive breast cancer.

•ZW49 Continues to Advance in Phase 1 Dose-Escalation Study
An update in January highlighted that there had been no dose-limiting toxicities observed and the maximum tolerated dose had not been reached. The majority of treatment-related adverse events were grade 1 or 2, and were reversible and manageable on an outpatient basis. Preliminary results from these initial dose cohorts included anti-tumor activity.

•Strengthens Commercial Leadership and Board of Directors
James Priour recently joined as Senior Vice President, Commercial, providing invaluable direction as Zymeworks begins registration-enabling trials with ZW25 this year. Before joining Zymeworks he held various international commercial leadership roles with Procter & Gamble Pharmaceuticals, Bristol Myers Squibb, and most recently at Amgen where he was the Global Product General Manager for Kyprolis and the early myeloma pipeline. Zymeworks also appointed Dr. Kelvin Neu, Partner at Baker Bros. Advisors LP, to the Board of Directors adding clinical knowledge and partnering expertise.

Financial Results for the Quarter Ended March 31, 2020

Revenue for the three months ended March 31, 2020 was $8.3 million as compared to $11.9 million in the same period of 2019. Revenue for the first quarter of 2020 included recognition of a $5.0 million development milestone and $2.2 million in drug supply revenue under the license and collaboration agreements with BeiGene as well as $1.1 million in research support and other payments from Zymeworks’ partners. Revenue for the first quarter of 2019 related primarily to an $8.0 million development milestone payment under the license and collaboration agreement with Lilly, recognition of $3.5 million of deferred revenue from the licensing and collaboration agreement with BeiGene, as well as $0.4 million in research support payments.

For the three months ended March 31, 2020, research and development expenses were $36.5 million as compared to $17.5 million in the same period of 2019. The change was primarily due to an increase in clinical trial activity and associated drug manufacturing costs for ZW25, as well as an increase in other research and discovery activities as compared to the same period in 2019. Research and development expenses also included non-cash stock-based compensation expense of $2.0 million from equity classified equity awards and a $1.8 million recovery related to the non-cash mark-to-market revaluation of certain historical liability classified equity awards.
For the three months ended March 31, 2020, general and administrative expenses were $7.6 million as compared to $9.0 million in the same period in 2019. The change was primarily due to a decrease in employee compensation expense relating to non-cash stock-based compensation, which was partially offset by an increase in head count in 2020 over 2019, associated with year-over-year corporate growth. General and administrative expenses included non-cash stock-based compensation expense of $2.2 million from equity classified equity awards and a $5.4 million recovery related to the non-cash mark-to-market revaluation of certain historical liability classified equity awards.
Net loss for the three months ended March 31, 2020 was $31.1 million as compared to $13.6 million in the same period of 2019. This was primarily due to the decrease in revenue and increase in research and development expenses referred to above, partially offset by higher interest and other income as well as lower general and administrative expenses.
Zymeworks expects research and development expenditures to increase over time in line with the advancement and expansion of the Company’s clinical development of its product candidates, as well as its ongoing preclinical research activities. Additionally, Zymeworks anticipates continuing to receive revenue from its existing and future strategic partnerships, including technology access fees and milestone-based payments. However, Zymeworks’ ability to receive these payments is dependent upon either Zymeworks or its collaborators successfully completing specified research and development activities.
As of March 31, 2020, Zymeworks had $562.7 million in cash resources consisting of cash, cash equivalents, short-term investments and certain long-term investments.

On May 7, 2020 Sesen Bio (Nasdaq: SESN), a late-stage clinical company developing targeted fusion protein therapeutics for the treatment of patients with cancer, reported that the Company has received positive Scientific Advice from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) related to the regulatory pathway for Vicinium in Europe (Press release, Sesen Bio, MAY 7, 2020, View Source [SID1234557296]). The Company’s lead program, Vicinium, also known as VB4-845, is currently in the follow-up stage of a Phase 3 registration trial for the treatment of high-risk, BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). In December 2019, the Company initiated the BLA submission for Vicinium to the United States Food and Drug Administration (FDA) under Rolling Review.

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"We are very pleased to have received positive guidance from the CHMP on the regulatory approval pathway for Vicinium," said Dr. Thomas Cannell, president and chief executive officer of Sesen Bio. "We strongly believe that Vicinium is a highly differentiated product candidate that is well positioned to address the considerable unmet need in NMIBC. This encouraging progress reinforces our confidence in bringing Vicinium to market in Europe, which represents a tremendous opportunity for the company. We will continue working collaboratively with the EMA to move Vicinium through the approval process as expeditiously as possible."

Key Elements of CHMP Scientific Advice

The CHMP agreed that the Company’s nonclinical, clinical pharmacology and safety database are all sufficient to support a marketing authorization application (MAA). Furthermore, additional clinical trials were not requested by the CHMP in support of the MAA submission for Vicinium.
The CHMP agreed that due to the well-known impact of cystectomy on morbidity and quality of life of patients, a new local treatment that enables patients to avoid radical cystectomy would be meaningful, especially for patients who are contraindicated for cystectomy.
The CHMP provided guidance on additional data analyses they expect to be included in the MAA, and the Company is confident that these can be fully addressed with the completed Phase 3 dataset.
Based on the guidance received, the Company expects to submit the MAA for Vicinium to the EMA in early 2021, with potential approval anticipated in early 2022.
The Company expects to receive Scientific Advice from the CHMP on the Chemistry, Manufacturing and Controls (CMC) program for Vicinium at a later date.
The Company believes there is a significant commercial opportunity in Europe and projects European peak sales to be substantially greater than US peak sales, driven by the significantly higher incidence rates and strong pricing benchmarks in the region.

About Vicinium

Vicinium, a locally administered fusion protein, is Sesen Bio’s lead product candidate being developed for the treatment of high-risk non-muscle invasive bladder cancer (NMIBC). Vicinium is comprised of a recombinant fusion protein that targets epithelial cell adhesion molecule (EpCAM) antigens on the surface of tumor cells to deliver a potent protein payload, Pseudomonas Exotoxin A. Vicinium is constructed with a stable, genetically engineered peptide tether to ensure the payload remains attached until it is internalized by the cancer cell, which is believed to decrease the risk of toxicity to healthy tissues, thereby improving its safety. In prior clinical trials conducted by Sesen Bio, EpCAM has been shown to be overexpressed in NMIBC cells with minimal to no EpCAM expression observed on normal bladder cells. Sesen Bio is currently conducting the Phase 3 VISTA trial, designed to support the registration of Vicinium for the treatment of high-risk NMIBC in patients who have previously received a minimum of two courses of bacillus Calmette-Guérin (BCG) and whose disease is now BCG-unresponsive. Additionally, Sesen Bio believes that cancer cell-killing properties of Vicinium promote an anti-tumor immune response that may potentially combine well with immuno-oncology drugs, such as checkpoint inhibitors. The activity of Vicinium in BCG-unresponsive NMIBC is also being explored at the US National Cancer Institute in combination with AstraZeneca’s immune checkpoint inhibitor durvalumab.

On May 7, 2020 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported financial results for the first quarter 2020 and provided an operational update (Press release, Bellicum Pharmaceuticals, MAY 7, 2020, View Source [SID1234557295]).

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"Bellicum entered the year with a renewed strategic focus on our novel GoCAR programs and we are pleased with the progress of our three development assets in the face of the COVID-19 pandemic," said Rick Fair, President and Chief Executive Officer of Bellicum. "Despite the public health challenges, we continued enrollment in our BPX-601 clinical trial, advanced our non-clinical research, and completed the strategically important sale of our facility to MD Anderson. Most importantly, we remain committed to the health and safety of our employees, our clinical collaborators, and patients involved in our clinical trials, and are closely monitoring and responding to public health guidance as we continue our important work."
PROGRAM HIGHLIGHTS AND CURRENT UPDATES

BPX-601 GoCAR-T
•Bellicum is currently enrolling patients in cohort 5C of the BPX-601 clinical trial in relapsed pancreatic cancer to evaluate the safety of repeat rimiducid dosing to re-activate BPX-601 cells over time. This is the first-in-human experience using the GoCAR platform as intended. Initial results from Cohort 5C are expected to be presented at a medical meeting by the end of 2020. Bellicum presented new Phase 1 translational data for BPX-601 at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO GI) in San Francisco in January 2020. Primary observations included tumor infiltration, GoCAR-T mediated immunomodulation, survival and persistence of cells for up to nine months, and changes in the tumor microenvironment gene expression consistent with a productive CAR-T cell immune response.

BPX-603 GoCAR-T
•Bellicum continues to conduct its investigational work in preparation of its response to the FDA’s request to further characterize this product candidate and support potential IND clearance. Management expects to provide an update on its progress for this program in the third quarter of 2020.

BCMA GoCAR-NK Program
•Preclinical development activities are continuing for Bellicum’s GoCAR-NK program targeting B-cell maturation antigen, or BCMA, for the treatment of multiple myeloma. Allogeneic GoCAR-NK cells may improve the durability of clinical responses while offering the anticipated advantages that an off-the-shelf product may provide, including faster and more certain time to treatment, greater scalability and convenience, and potentially lower cost. Management expects to present additional preclinical data for this program by the end of 2020.

Exhibit 99.1

Corporate Highlights
•In April 2020, Bellicum closed a transaction in which The University of Texas MD Anderson Cancer Center acquired Bellicum’s approximately 60,000-square-foot Houston facility, including manufacturing, office and laboratory space, for $15.0 million. In conjunction with the asset purchase agreement, Bellicum entered into a master services agreement with MD Anderson under which MD Anderson will manufacture Bellicum’s cellular therapy candidates, in addition to using the facility for its own internal programs. Concurrent with this transaction, Bellicum partially repaid approximately $7.0 million of its Oxford Finance debt obligations.

Potential for COVID-19 Impact
•Due to the COVID-19 pandemic, MD Anderson has temporarily closed its cell therapy manufacturing facilities. Bellicum does not currently expect the facility closure to significantly impact the company’s clinical trial progression and the timing of results, but such delays are possible depending on the duration of closure. The exact timing of potential delays and the overall impact of the COVID-19 pandemic on Bellicum’s business, its ability to obtain clinical material, and to conduct preclinical research and clinical trials in a timely manner is currently unknown. So far, the pandemic has not had a material impact on Bellicum’s ability to conduct its business activities, but that could change, and management is monitoring and responding to the crisis as it continues to evolve.

First Quarter 2020 Financial Results
R&D Expenses: Research and development (R&D) expenses were $10.4 million for the first quarter of 2020, compared to $16.8 million for the first quarter of 2019. The reduction in expenses in the first quarter of 2020 resulted primarily from reduced expenses related to rivo-cel and the reduction in force that was implemented during the second half of 2019, partially offset by an increase in expenses related to our GoCAR programs.

G&A Expenses: General and administrative (G&A) expenses were $4.2 million for the first quarter of 2020 compared to $7.5 million during the comparable period in 2019. The reduction in expenses in the first quarter 2020 relative to the comparable period in 2019 were primarily due to the reduction in rivo-cel related commercialization activities as well as the effects of the reduction in force that reduced employee-related charges.

Loss from Operations: Bellicum reported a loss from operations of $14.6 million for the first quarter of 2020 compared to a loss from operations of $23.9 million for the first quarter of 2019. Cash used in operating activities was $16.3 million for the first quarter of 2020, compared to cash used in operating activities of $24.6 million.
Net Income/Loss: Bellicum reported net income of $17.6 million for the first quarter of 2020 compared to a net loss of $24.5 million for the first quarter of 2019. The results included a non-cash gain of $32.8 million related to the change in fair value of warrant and private placement option liability in the first quarter of 2020.
Shares Outstanding: As of March 31, 2020, Bellicum had 5,049,892 shares of common stock outstanding and 534,000 shares of preferred stock outstanding. Each share of preferred stock can be converted into 10 shares of common stock.
Cash Position and Guidance: Bellicum reported cash and cash equivalents and restricted cash totaling $75.5 million as of March 31, 2020, compared to $93.8 million as of December 31, 2019. Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements into the second half of 2021. Management expects cash utilization of $55 to $65 million in 2020.