On May 15, 2019 Achieve Life Sciences, Inc. (Nasdaq: ACHV), a clinical-stage pharmaceutical company committed to the global development and commercialization of cytisinicline for smoking cessation, reported that first quarter 2019 financial results (Press release, OncoGenex Pharmaceuticals, MAY 15, 2019, View Source [SID1234536324]).

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ORCA-1 Trial Status

Achieve’s 254-subject Phase 2b ORCA-1 trial of cytisinicline in U.S. smokers completed enrollment in February. The Company announced recently that the last study visit for the last subject enrolled in the ORCA-1 trial has occurred. The trial is evaluating cytisinicline in both the 1.5 mg and 3.0 mg doses on a declining titration schedule as well as three times daily dosing, both over 25 days. The primary efficacy endpoint is reduction in the number of cigarettes smoked during treatment with secondary analyses to be conducted on smoking cessation rates, safety, and compliance. ORCA-1 topline efficacy and safety data are expected to be announced by the end of the second quarter.

Maximum Tolerated Dose (MTD) Trial Extension

Achieve recently initiated a trial to assess the MTD for a single administered oral dose of cytisinicline in smokers. The starting dosage of cytisinicline was 6 mg and is increased in separate groups of subjects for each escalated dose level until stopping criteria, based on the occurrence of dose-limiting adverse events, are reached. To date, 21 mg cytisinicline has been evaluated without evidence of dose limiting toxicity. The trial’s Data Safety Monitoring Committee has recommended a protocol amendment to evaluate additional higher doses of cytisinicline.

Rick Stewart, Chairman and Chief Executive Officer of Achieve Life Sciences commented, "With the results of the ORCA-1 trial expected by the end of the second quarter, we are rapidly approaching another critical milestone for the cytisinicline development program. Importantly, as confirmed by the tolerability of high dose levels in our MTD study, we believe cytisinicline may offer a differentiated and new treatment option for the millions of people who are battling nicotine addiction."

Financial Results

As of March 31, 2019, the company’s cash, cash equivalents, short-term investments and restricted cash were $9.7 million. Each of total operating expenses and net loss for the first quarter of 2019 were $5.9 million.

As of May 15, 2019 Achieve had 6,865,950 shares outstanding.

Conference Call Details

Achieve will host a conference call at 4:30 p.m. Eastern time today, Wednesday, May 15, 2019. To access the webcast, log on to the investor relations page of the Achieve website at View Source Alternatively, access to the live conference call is available by dialing (877) 472-9809 (U.S. & Canada) or (629) 228-0791 (International) and referencing conference ID 6973659. A webcast replay will be available approximately two hours after the call and will be archived on the website for 90 days.

On May 15, 2019 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported that its abstract for BPX-601 has been accepted for poster presentation at the 2019 American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Bellicum Pharmaceuticals, MAY 15, 2019, View Source [SID1234536323]). The meeting is being held May 31 – June 4, 2019 in Chicago.

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Details of the poster presentation are as follows:

Title: Ligand-Inducible, Prostate Stem Cell Antigen (PSCA)-Directed GoCAR-T Cells in Advanced Solid Tumors: Preliminary Results with Cyclophosphamide (Cy) ± Fludarabine (Flu) Lymphodepletion (LD)
Poster Board: #180-Abstract 2536
Presenter: Carlos R. Becerra, M.D.
Time/Location: Saturday, June 1, 8 to 11 a.m. CDT, Hall A, Poster Session – Developmental Immunotherapy and Tumor Immunobiology

About BPX-601

BPX-601, the company’s first GoCAR-T product candidate, incorporates iMC, Bellicum’s inducible co-activation domain. iMC (inducible MyD88/CD40) is designed to provide a powerful boost to T cell proliferation and persistence and enable the CAR-T to override key immune inhibitory mechanisms, including PD-1 and TGF-beta. BPX-601 is being evaluated as a treatment for solid tumors expressing prostate stem cell antigen (PSCA), including pancreatic, gastric, and prostate cancers.

On May 15, 2019 Surface Oncology (NASDAQ:SURF), a clinical-stage immuno-oncology company developing next-generation immunotherapies that target the tumor microenvironment, reported that Jeff Goater, chief executive officer, will be participating and presenting a corporate update at the UBS Global Healthcare Conference on Wednesday, May 22, 2019 at the Grand Hyatt New York in New York City (Press release, Surface Oncology, MAY 15, 2019, View Source [SID1234536322]).

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Additionally, Dr. Pamela Holland, PhD, vice president of cancer biology, will present at the Brisbane Immunotherapy 2019 Conference. Her presentation, "Targeting the Adenosine Axis to Treat Cancer" will take place on Friday, May 24, 2019 at the Brisbane Convention Centre, in Brisbane, Australia.

The presentations will be accessible by visiting the Investors page of the website at View Source

On May 15, 2019 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. pharmaceutical company with a platform to develop and accelerate the launch of innovative therapeutics and pharmaceutical products in China, the U.S., and throughout the world, reported financial results for the first quarter 2019 and provided a review of recent accomplishments and anticipated upcoming milestones (Press release, CASI Pharmaceuticals, MAY 15, 2019, View Source [SID1234536320]).

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Wei-Wu He, Ph.D., CASI’s Chairman and Chief Executive Officer, commented, "We have made good progress in the first quarter of 2019. Most recently, we announced that we acquired exclusive worldwide rights to a novel anti-CD38 monoclonal antibody (CID-103). Preclinical data suggests an enhanced safety and efficacy profile against a broad array of hematological malignancies expressing CD38, making CID-103 an excellent strategic fit amid our broader oncology platform. We look forward to announcing progress in this program as well as across other key programs in our pipeline."

First Quarter and Recent Business Highlights

Acquired worldwide rights to CID-103, a novel anti-CD38 monoclonal antibody – On April 17, 2019, CASI acquired worldwide rights to CID-103, a novel anti-CD38 monoclonal antibody. CID-103 is a human monoclonal antibody targeting a specific epitope of the CD38 cell surface antigen. It is at the IND/IMPD submission stage of development with Phase 1 trials expected in late 2019 or early 2020. The addition of CID-103 compliments the Company’s oncology pipeline and allows the Company to provide more treatment options to patients in the future.

Entered into distribution agreement with China distribution partner for EVOMELA (melphalan hydrochloride for injection) – In March 2019, the Company entered into an exclusive distribution agreement with China Resources Guokang Pharmaceuticals Co., Ltd., (CRGK) to be the Company’s exclusive distributor in China for EVOMELA. CASI will maintain responsibility for direct marketing and sales.

Received National Medical Products Administration (NMPA) Clinical Trial Application (CTA) Approvals – In February and March 2019, CASI announced key NMPA clinical trial approvals (CTA) to conduct confirmatory registration trials for both ZEVALIN and MARQIBO, respectively. ZEVALIN is indicated for relapsed or refractory, low-grade or follicular B-cell non-Hodgkin’s lymphoma and MARQIBO is indicated for Philadelphia chromosome-negative (Ph-) acute lymphoblastic leukemia (ALL) in second or greater relapse or whose disease has progressed following two or more anti-leukemia therapies. The Company is working toward getting the trials started in China.

Chairman Wei-Wu He, Ph.D. expanded role to include Chief Executive Officer – On April 2, 2019, CASI appointed its Chairman of the Board of Directors as the Company’s Chief Executive Officer. Dr. He served on CASI’s Board of Directors since 2012. Dr. He has a very successful career building companies from early stage to revenue and profitability stages and most recently was the CEO of OriGene Technologies which he co-founded and built into a profitable life science company; OriGene ultimately merged with a publicly traded Chinese company in 2018.

Financial Results for the Quarter ended March 31, 2019

Cash Position: As of March 31, 2019, CASI had cash and cash equivalents of $99.7 million.

R&D Expenses: R&D expenses for the quarter ended March 31, 2019 were $2.6 million compared to $1.7 million in 2018, an increase of $0.9 million. The increase in R&D expenses primarily reflects costs associated with regulatory, consulting and manufacturing related services, primarily associated with our acquired ANDAs in 2018, as well as an increase in facility costs due to new lease space in China, as well as an increase in personnel costs due to growth in the number of employees.

G&A Expenses: G&A expenses for the quarter ended March 31, 2019 were $5.7 million compared to $1.3 million in 2018. The increase of $4.4 million in G&A over the prior year primarily reflects an increase of $1.6 million in non-cash stock compensation expense largely attributed to stock options issued to the Company’s Chairman and also the President of CASI China, an increase in salary, benefits and recruitment expense, as well as facilities costs primarily in China, related to sales and marketing efforts to prepare for the anticipated launch of the Company’s first commercial product in China, as well as other G&A functions. There were also increased costs associated with professional services fees, including audit and legal services during the 2019 period.

Net Loss: The Company reported a net loss of ($8.2 million), or ($0.09) per share, for the quarter ended March 31, 2019. This compares with a net loss of ($3.6 million), or ($0.05) per share for the first quarter of 2018. The larger net loss for both periods is primarily due to the increase in non-cash stock-based compensation expense during the 2019 period, costs associated with the manufacturing and regulatory support for our ANDA portfolio, and increased costs associated with G&A functions, including employment costs for sales and marketing efforts, as well as increased facilities cost in China and higher professional service fees.

Further information regarding the Company, including its Quarterly Report on Form 10-Q for the quarter ended March 31, 2019, can be found at www.casipharmaceuticals.com.

On May 15, 2019 VBL Therapeutics (Nasdaq: VBLT) reported financial results for the first quarter ended March 31, 2019, and provided a corporate update (Press release, VBL Therapeutics, MAY 15, 2019, View Source [SID1234536306]).

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"As we continue to advance development of VB-111, we are getting a better understanding of its mechanism of action and how best to incorporate it into oncology treatment regimens," said Dror Harats, M.D., Chief Executive Officer of VBL Therapeutics. "We expect two additional VB-111 clinical trials to begin this year, a Phase 2 trial in colon cancer in combination with a checkpoint inhibitor (in collaboration with the National Cancer Institute) and an investigator-sponsored study in recurrent glioblastoma which will be conducted by a group of top US neuro-oncology centers. Therefore, together with the ongoing Phase 3 OVAL pivotal study in ovarian cancer, there will be a total of three VB-111 clinical trials up and running in the second half of 2019.

We are also pleased to announce that there will be two presentations on VB-111 at the upcoming 2019 ASCO (Free ASCO Whitepaper) Annual Meeting in early June, by Dr. Richard Penson from Massachusetts General Hospital on the final Phase 2 results in ovarian cancer, and by Dr. Benjamin Ellingson from UCLA on VB-111 Phase 2 and Phase 3 MRI data in recurrent GBM.

In addition to advancing VB-111, we are also moving forward with two parallel development programs targeting MOSPD2, which has significant potential as a therapeutic target for both inflammatory diseases and cancer."

"We had $47.7 million in cash and cash equivalents at March 31, 2019, which we believe is sufficient to continue development of VB-111 and other product candidates and to fund our operating expenses and capital expenditure requirements through 2021," said Amos Ron, Chief Financial Officer of VBL Therapeutics.

First Quarter and Recent Corporate Highlights:

Executed a strategic exclusive option license agreement with one of the world-leading European animal health companies, for the development of VBL’s proprietary anti-inflammatory molecule, VB-201, for veterinary use. VBL received an undisclosed up-front payment and upon exercising the option to license, VBL expects to receive additional milestones and royalties, which may exceed €50 million. VBL retains worldwide rights for the use of VB-201 for the treatment of humans.

Awarded a non-dilutive grant of over 10 million New Israeli Shekels (approximately $2.9 million) by the Israel Innovation Authority (IIA), to support continued development of VB-111 for 2019.

Presented new data indicating that VB-111 has potential to stimulate the immune system to induce a strong and durable response against ovarian tumors at the Society for Gynecologic Oncology 50th Annual Meeting on Women’s Cancer. The Company’s ongoing potential registration OVAL pivotal trial in platinum resistant ovarian cancer continues to enroll patients as planned.

MOSPD2 program for inflammation: Presented new data at the Keystone Symposia on the role of MOSPD2 in the migration of certain inflammatory cells, the myeloid cells. VBL is developing the VB-600 platform of antibodies targeting MOSPD2 for treatment of various inflammatory indications.

MOSPD2 oncology program: VBL is currently developing bi-specific antibodies that bring together tumor cells, via MOSPD2, and T cells, via CD3, to recruit the immune system to fight tumor cells.

First Quarter ended March 31, 2019 Financial Results:

Cash Position: At March 31, 2019, the Company had cash, cash equivalents and short-term bank deposits totaling $47.7 million and working capital of $43.1 million. The Company expects that its cash, cash equivalents and short-term bank deposits will enable it to fund operating expenses and capital expenditure requirements through 2021.

Revenues: Revenues related to VBL’s collaborations were $0.2 million in the first quarter of 2019.

R&D Expenses: Research and development expenses, net, after government grants, for the quarter ended March 31, 2019, were approximately $3.3 million, compared to approximately $5.8 million in the same period in 2018.

G&A Expenses: General and administrative expenses for the quarter ended March 31, 2019 were $1.3 million, compared to $1.4 million for the same period in 2018.

Comprehensive Loss: VBL reported a net loss for first quarter ended March 31, 2019, of $4.2 million, or ($0.12) per share, compared to a net loss of $7.2 million, or ($0.24) per share, in the quarter ended March 31, 2018.

For further details on VBL’s financials, please refer to Form 6-K filed with the SEC.

Conference Call:

Wednesday, May 15th @ 8:30am Eastern Time

From the US: 877-407-9208

International: 201-493-6784

Conference ID: 13690495

Webcast: View Source