On May 26, 2021 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical company focused on oncology, reported its financial results for the quarter ended March 31, 2021 and provides a corporate update (Press release, BioLineRx, MAY 26, 2021, View Source [SID1234580609]).

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Significant events and achievements during the first quarter 2021 and subsequent period:

Announced positive topline results from GENESIS Phase 3 trial of Motixafortide in stem-cell mobilization (SCM). The data demonstrate that the study successfully met all primary and secondary endpoints with an exceptionally high level of statistical significance (p<0.0001).
88.3% of patients receiving Motixafortide + G-CSF underwent transplantation after only ONE administration of Motixafortide and in only ONE apheresis session, compared to 10.8% for G-CSF alone; potentially supports Motixafortide on top of G-CSF as new standard-of-care mobilization agent in autologous bone-marrow transplantation.
The Company is proceeding with activities in support of an NDA submission in this indication anticipated in the first half of 2022, including a pre-NDA meeting with the FDA planned for the second half of this year.
Presented data at the 2021 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting analyzing results by liver metastasis status from the Company’s Phase 2a COMBAT/KEYNOTE-202 triple combination study testing Motixafortide in metastatic pancreatic cancer. The analysis further strengthened the results reported from the study in December 2020, since not only were substantially all patients initially diagnosed with stage 4 disease, but the vast majority (~80%) of the patients had liver metastases, emphasizing the extremely difficult patient population in this study.
Strengthened balance sheet with underwritten public offering resulting in gross proceeds of $34.5 million.
"Subsequent to the end of the first quarter, we were extremely excited to announce positive topline results from our GENESIS Phase 3 trial of Motixafortide in stem-cell mobilization for autologous bone marrow transplantation in multiple myeloma patients," stated Philip Serlin, Chief Executive Officer of BioLineRx. "The results demonstrated, with a high degree of statistical significance, a meaningful clinical benefit from adding Motixafortide to the current standard of care, G-CSF, for the mobilization of the targeted number of stem cells required for transplantation. While this was not a head-to-head study, our results compare very favorably to the registrational study of plerixafor.

"Importantly, almost 90% of patients in the treatment cohort underwent transplantation after only one administration of Motixafortide and in only one apheresis session, compared to 10.8% for G-CSF alone. We believe this positions Motixafortide to become the new standard of care in this indication, with a clear clinical benefit of ‘one dose, one apheresis, 90% mobilization success rate.’ We are working diligently to submit a New Drug Application to the FDA in the first half of next year. If approved, this would be transformative for BioLineRx, and a huge milestone in the Company’s history.

"Regarding our PDAC program, the compelling liver metastases data that we recently presented at AACR (Free AACR Whitepaper) further strengthen an already robust case for continued development in this very challenging indication. We continue to engage in discussions with potential partners regarding future development.

"To support these and other initiatives, including continued advancement of our second clinical candidate, the anti-cancer vaccine AGI-134, we raised $34.5 million in January that we believe will finance the Company through multiple potentially value-creating milestones," concluded Mr. Serlin.

Upcoming Significant Expected Milestones:

Initial results from Part 2 of the Phase 1/2a trial of AGI-134 in solid tumors in the second half of 2021;
Pre-NDA meeting with the FDA for SCM in the second half of 2021;
NDA submission for SCM in the first half of 2022.
Financial Results for the Quarter Ended March 31, 2021

Research and development expenses for the quarter ended March 31, 2021 were $4.3 million, a decrease of $1.1 million, or 21.1%, compared to $5.4 million for the quarter ended March 31, 2020. The decrease resulted primarily from lower expenses associated with the Motixafortide COMBAT clinical trial, as well as lower expenses associated with the AGI-134 study.

Sales and marketing expenses for the quarter ended March 31, 2021 were $0.2 million, similar to sales and marketing expenses for the quarter ended March 31, 2020.

General and administrative expenses for the quarter ended March 31, 2021 were $1.0 million, a decrease of $0.2 million, or 18.2% compared to $1.2 million for the quarter ended March 31, 2020. The decrease resulted primarily from a decrease in share-based compensation.

The Company’s operating loss for the quarter ended March 31, 2021 amounted to $5.5 million, compared to an operating loss of $6.8 million for the quarter ended March 31, 2020.

Non-operating expenses amounted to $4.6 million for the quarter ended March 31, 2021, compared to non-operating income of $0.5 million for the quarter ended March 31, 2020. Non-operating income (expenses) for both periods primarily relate to fair-value adjustments of warrant liabilities on the Company’s balance sheet.

Net financial expenses amounted to $0.2 million for the quarter ended March 31, 2021, compared to net financial expenses of $0.3 million for the quarter ended March 31, 2020. Net financial expenses for both periods primarily relate to interest paid on loans, offset by investment income earned on bank deposits.

The Company’s net loss for the quarter ended March 31, 2021 amounted to $10.2 million, compared with a net loss of $6.6 million for the quarter ended March 31, 2020.

The Company held $58.1 million in cash, cash equivalents and short-term bank deposits as of March 31, 2021.

Net cash used in operating activities was $6.2 million for the quarter ended March 31, 2021, compared with net cash used in operating activities of $6.7 million for the quarter ended March 31, 2020. The $0.5 million decrease in net cash used in operating activities between the two periods was primarily the result of a decrease in research and development expenses.

Net cash used in investing activities was $36.3 million for the quarter ended March 31, 2021, compared to net cash provided by investing activities of $6.2 million for the quarter ended March 31, 2020. The changes in cash flows from investing activities relate primarily to investments in, and maturities of, short-term bank deposits.

Net cash provided by financing activities was $41.9 million for the quarter ended March 31, 2021, compared to net cash provided by financing activities of $0.4 million for the quarter ended March 31, 2020. The cash flows in 2021 primarily reflect the underwritten public offering of the Company’s ADSs in January 2021, warrant exercises and net proceeds from an ATM facility, offset by repayments of a loan from Kreos Capital. The cash flows in 2020 primarily reflect the net proceeds from an ATM facility, offset by repayments of a loan from Kreos Capital.

Conference Call and Webcast Information

BioLineRx will hold a conference call today, Wednesday, May 26, 2021 at 10:00 a.m. EDT. To access the conference call, please dial +1-866-744-5399 from the US or +972-3-918-0610 internationally. The call will also be available via webcast and can be accessed through the Investor Relations page of BioLineRx’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast.

A replay of the conference call will be available approximately two hours after completion of the live conference call on the Investor Relations page of BioLineRx’s website. A dial-in replay of the call will be available until May 28, 2021; please dial +1-888-295-2634 from the US or +972-3-925-5904 internationally.

On May 26, 2021 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported that the company will present at the Jefferies Virtual Healthcare Conference on Wednesday, June 2, 2021 at 2:30 p.m. ET and the JMP Securities Life Sciences Conference on Thursday, June 17, 2021 at 1:30 p.m. ET (Press release, BioCryst Pharmaceuticals, MAY 26, 2021, View Source [SID1234580608]). Both are being conducted as virtual conferences.

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Links to a live audio webcast and replay of these presentations may be accessed in the Investors section of BioCryst’s website at http://www.biocryst.com.

On May 26, 2021 Anixa Biosciences, Inc. (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer and infectious diseases, reported that its ovarian cancer vaccine technology is part of an award from the National Cancer Institute (NCI) (Press release, Anixa Biosciences, MAY 26, 2021, View Source [SID1234580607]). Anixa Biosciences holds an exclusive worldwide license from Cleveland Clinic for this vaccine technology.

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The award from the NCI, part of the National Institutes of Health, was made to Cleveland Clinic through the NCI-PREVENT program.

The PREVENT program is a peer-reviewed agent development program designed to support preclinical development of innovative interventions and biomarkers for cancer prevention and interception towards clinical trials. The scientific and financial resources of the PREVENT program will be allocated to four general milestones as follows:

Milestone 1: Immunogenicity and efficacy testing of the vaccine in mouse model
Milestone 2: Development of the expression vector for optimal vaccine production
Milestone 3: cGMP (current Good Manufacturing Practice) manufacturing of vaccine and pre-IND (Investigational New Drug) meeting with the US Food and Drug Administration
Milestone 4: IND enabling cGLP (current Good Laboratory Practice) studies and IND filing
All of this work will be performed at NCI facilities, by NCI scientific staff with NCI financial resources. Milestone 1 will begin shortly after execution of contracts and design of experiments. Milestones 2-4 will be performed successively as progress is achieved.

The technology subject to the application titled, Immunoprevention of Human Epithelial Ovarian Carcinoma (EOC), was invented by a research team led by Dr. Vincent Tuohy of Cleveland Clinic’s Lerner Research Institute. Dr. Tuohy stated, "This peer-reviewed collaboration will provide us the vast laboratory and financial resources of the NCI to aid in the development of our vaccine. Our vision with this technology is to eliminate ovarian cancer worldwide, especially since women who suffer from ovarian cancer tend to have poor outcomes."

Dr. Amit Kumar, President and CEO of Anixa Biosciences, stated, "This technology along with Dr. Tuohy’s breast cancer vaccine have the potential to eliminate two of the most common malignancies in women. We continue to be pleased to be collaborating on these programs."

On May 26, 2021 AIM ImmunoTech Inc. (NYSE American: AIM) reported that it has completed dosing of Cohort 3 in a Phase 1 clinical study to assess the safety, tolerability and biological activity of Ampligen as an intranasal therapy, reporting no serious adverse events (Press release, AIM ImmunoTech, MAY 26, 2021, View Source [SID1234580606]).

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This is consistent with results in the first two cohorts of the study and at escalating doses.The study protocol called for a total of 40 healthy subjects to receive repeated administration of either Ampligen or a placebo. Subjects in Cohort 1 received 75 μg of Ampligen, subjects in Cohort 2 received 200 μg of Ampligen and subjects in Cohort 3 received 500 μg. The study is already proceeding with Cohort 4, with subjects receiving 1250 μg.

The Centre for Human Drug Research (CHDR), an independent institute located in Leiden in the Netherlands, is conducting the Phase 1 clinical study AMP-COV-100 (CHDR2049), titled "A Phase I, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Activity of Repeated Intranasal Administration of Ampligen (Poly I:Poly C12U) in Healthy Subjects." AIM is sponsoring and funding the clinical study.

On May 26, 2021 ADC Therapeutics SA (NYSE:ADCT), a commercial-stage biotechnology company leading the development of novel antibody drug conjugates (ADCs) to treat hematological malignancies and solid tumors, reported that results of the Phase 1 clinical trial of camidanlumab tesirine (Cami), an anti-CD25 ADC, in patients with relapsed or refractory Hodgkin and non-Hodgkin lymphomas have been published online in The Lancet Haematology (Press release, ADC Therapeutics, MAY 26, 2021, View Source [SID1234580605]).

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"There is a significant unmet medical need for novel therapies that improve outcomes in patients with relapsed or refractory Hodgkin lymphoma," said Mehdi Hamadani, MD, Professor of Internal Medicine at the Medical College of Wisconsin, Division of Hematology & Oncology and lead author of The Lancet Haematology paper. "This patient population is often heavily pretreated, as was the case in this published study in which patients experienced a median of five previous systemic therapies. The Phase 1 study demonstrates encouraging potential for Cami to provide a new treatment option for patients with relapsed or refractory Hodgkin lymphoma."

The multicenter, open-label, single-arm, dose escalation and dose expansion Phase 1 clinical trial enrolled 133 adult patients, 77 (58%) with classical Hodgkin lymphoma and 56 (42%) with non-Hodgkin lymphoma. Enrolled patients were required to have pathologically confirmed relapsed or refractory Hodgkin lymphoma or non-Hodgkin lymphoma and no therapies with established clinical benefit for their disease stage available to them.

"We’re pleased the results of our Phase 1 trial of Cami in patients with relapsed or refractory lymphoma have been published in The Lancet Haematology," said Jay Feingold, MD, PhD, Senior Vice President and Chief Medical Officer at ADC Therapeutics. "The positive results in patients with Hodgkin lymphoma guided the design of our pivotal Phase 2 trial, which has completed enrollment. We’re continuing to follow patients and look forward to presenting data from the Phase 2 Cami trial at an upcoming congress."

Key results include:

In the total patient population, the overall response rate (ORR) was 58%, with 38 (29%) of 130 patients reported to have a complete response.
In heavily pretreated patients with Hodgkin lymphoma, across all doses, the ORR was 71%, with 32 (42%) of 77 patients reported to have a complete response.
In Hodgkin lymphoma patients who received 45 μg/kg (the recommended Phase 2 starting dose), the ORR was 86%, with 18 (49%) of 37 patients reported to have a complete response.
In Hodgkin lymphoma patients who received 30 μg/kg, the ORR was 55%, with seven (35%) of 20 patients reported to have a complete response.
In patients with non-Hodgkin lymphoma, the ORR was 38%, with five (9%) of 53 patients reported to have a complete response.
The overall median duration of response was 6.6 months for all patients with Hodgkin lymphoma and 7.2 months for Hodgkin lymphoma patients who received 45 μg/kg.
Cami demonstrated an acceptable safety profile. The most commonly observed adverse events included elevated liver enzymes (without hepatic synthetic dysfunction), rash, fatigue, oedema or effusion, and nausea.
Based on the data from this Phase 1 clinical trial, a Phase 2 trial to further evaluate the safety and efficacy of Cami in patients with relapsed or refractory Hodgkin lymphoma is ongoing. Interim data from the pivotal Phase 2 trial presented at the 2020 American Society of Hematology (ASH) (Free ASH Whitepaper) meeting demonstrated encouraging antitumor activity as a single agent with an ORR of 83%, a complete response rate of 38% and no new safety signals. These data highlight the potential for Cami to address an unmet need in heavily pretreated patients (median of seven prior lines of systemic therapy).

For information about the ongoing pivotal Phase 2 clinical trial of Cami in patients with relapsed or refractory Hodgkin lymphoma, please visit View Source (identifier NCT04052997).

About Camidanlumab Tesirine (Cami)

Camidanlumab tesirine (Cami, formerly ADCT-301) is an antibody drug conjugate (ADC) comprised of a monoclonal antibody that binds to CD25 (HuMax-TAC, licensed from Genmab A/S), conjugated to the pyrrolobenzodiazepine (PBD) dimer payload, tesirine. Once bound to a CD25-expressing cell, Cami is internalized into the cell where enzymes release the PBD-based payload, killing the cell. This applies to CD25-expressing tumor cells and also to CD25-expressing Tregs. The intra-tumoral release of its PBD payload may also cause bystander killing of neighboring tumor cells, and PBDs have also been shown to induce immunogenic cell death. All of these properties of Cami may enhance immune-mediated anti-tumor activity.

Cami is being evaluated in a pivotal Phase 2 clinical trial in patients with relapsed or refractory Hodgkin lymphoma and a Phase 1b clinical trial as monotherapy and in combination with pembrolizumab in solid tumors.