1stOncology™: Cancer Intelligence Service – Page 9757 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Bolt Biotherapeutics Reports Fourth Quarter and Full Year 2020 Financial Results and Provides Business Highlights

On March 31, 2021 Bolt Biotherapeutics, Inc. (NASDAQ: BOLT) a clinical-stage biotechnology company pioneering a new class of immuno-oncology agents that combine the targeting precision of antibodies with the power of both the innate and adaptive immune systems, reported financial results for the fourth quarter and full year ended December 31, 2020 and provided an update on recent business highlights (Press release, Bolt Biotherapeutics, MAR 31, 2021, View Source [SID1234577436]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

"Our upsized Initial Public Offering, which we completed in February 2021, leaves us in a strong financial position to execute on our vision of developing this new class of immuno-oncology products to help patients. We continue to enroll patients in the dose escalation part of our Phase 1/2 trial for our lead candidate, BDC-1001, for the treatment of patients with HER2-expressing solid tumors. We reported preliminary clinical results from an initial 20 patients at a data cutoff of January 29, 2021, which demonstrated 4 patients with stable disease and one patient with a confirmed partial response. We’re looking forward to completing the dose escalation and initiating both the monotherapy Phase 2 dose expansions part and the combination studies with an anti-PD-1 antibody part later in 2021," said Randall C. Schatzman, Ph.D., Chief Executive Officer of Bolt. "We continue to progress our broader pipeline of targeted immunotherapies derived from our Boltbody ISAC platform, a novel technology that can be applied across a diverse range of tumor targets and has the potential to enable cancer patients to generate immunological memory against their own tumors. We plan to advance our second Boltbody ISAC BDC-2034, which targets the cancer antigen CEA, into the clinic in 2022."

Recent Business Highlights and Anticipated Milestones

Completed upsized Initial Public Offering in February 2021 – In February 2021, Bolt completed its Initial Public Offering (IPO) of 13,225,000 shares of common stock, inclusive of the full exercise by the underwriters of their option to purchase 1,725,000 shares, at a public offering price of $20.00 per share. Gross proceeds from the IPO were $264.5 million and net proceeds from the offering, after deducting underwriting discounts, commissions and offering expenses, were approximately $241.7 million.

Reported preliminary clinical results from the first 20 patients from the ongoing Phase 1/2 trial of lead candidate BDC-1001 for the treatment of patients with HER2-expressing solid tumors –BDC-1001 is a human epidermal growth factor receptor 2, or HER2, Boltbody Immune-Stimulating Antibody Conjugate (ISAC) comprised of a HER2-targeting biosimilar of trastuzumab conjugated to one of Bolt’s proprietary TLR7/8 agonists, for the treatment of patients with HER2-expressing solid tumors, including HER2-low tumors. A Phase 1/2 study evaluating BDC-1001 in patients with HER2-expressing cancers, which includes patients with breast and gastric cancers that are refractory to Herceptin and Kadcyla, as well as cancers for which no HER2-targeting therapies have yet been approved, is ongoing. Bolt is currently enrolling patients in the dose escalation portion of the trial. As of January 29, 2021, Bolt had treated 20 patients and BDC-1001 appeared to be well tolerated with mild to moderate adverse events and no dose-limiting toxicities or drug-related serious adverse events were observed. Clinical activity was seen in the form of stable disease, reductions in tumor volume including a confirmed partial response and increases in pharmacodynamic markers that Bolt believes are consistent with its proposed mechanism of action. Later this year, Bolt plans to advance to part 3 of the trial, monotherapy Phase 2 dose expansion cohorts, and part 2, BDC-1001 dose escalation in combination with an anti-PD-1 antibody.

Strengthened Board of Directors with appointment of Kathleen LaPorte – In January 2021, Kathleen LaPorte joined the Board of Directors as Chair of Bolt’s Audit Committee. Ms. LaPorte has more than 30 years of experience in building and operating private and public biotech companies as former chief executive officer of Nodality Inc. and a founding partner of New Leaf Venture Partners. The addition increased Bolt’s board to eight members.

Published data in Nature Cancer highlighting proof of concept for Boltbody ISAC platform to eliminate tumors following systemic administration – In December 2020, Bolt announced the publication of a manuscript in Nature Cancer highlighting the development and use of Boltbody ISACs for the treatment of HER2-expressing tumors in preclinical models. The data indicate that Boltbody ISACs activate the innate and adaptive immune systems, ultimately resulting in complete tumor regressions in multiple tumor models and durable anti-tumor immunity. ISAC-mediated immunological memory extended beyond the target antigen as ISAC-treated mice were protected from re-challenge with the parental tumor lacking HER2 expression.

Completed an oversubscribed $93.5 million Series C financing with notable crossover investors – In July 2020 and January 2021, the Company raised funding to support BDC-1001 and continued development of the Boltbody ISAC platform and Bolt’s pipeline of immuno-oncology programs. Sofinnova Investments led the investment, which included notable crossover investors RA Capital Management, Surveyor Capital (a Citadel Company), Rock Springs Capital and Samsara BioCapital.
Upcoming Events

Bolt’s Chief Scientific Officer David Dornan, Ph.D. will present a talk on BDC-1001 as part of "New Drugs on the Horizon: Part 2" at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting on Saturday, April 10, 2021, from 4:00 PM – 5:45 PM ET.

Manish R. Sharma, M.D. of START Midwest, a principal investigator in Bolt’s ongoing BDC-1001 Phase 1/2 trial, will present a trial-in-progress e-poster entitled "CT218 – Phase 1/2 study of a novel HER2 targeting TLR7/8 immune-stimulating antibody conjugate (ISAC), BDC-1001" in the session "PO.CT08.01 – Phase I Clinical Trials in Progress" at AACR (Free AACR Whitepaper) on Saturday, April 10, 2021, from 8:30 AM – 11:59 PM ET.

Bolt’s CEO, Randall Schatzman, Ph.D., will present a corporate overview at the virtual 20th Annual Needham Virtual Healthcare Conference on Thursday, April 15, 2021 at 10:15 AM ET.
Fourth Quarter and Full Year 2020 Financial Results

Cash Position – Cash, cash equivalents, and marketable securities were $22.8 million as of December 31, 2020, as compared to $34.8 million as of December 31, 2019. Total cash, cash equivalents, and marketable securities at December 31, 2020 does not include total net proceeds of approximately $293.6 million from Bolt’s C-2 convertible preferred stock offering in January 2021 and its IPO in February 2021. Bolt expects its cash balance to fund operations into 2023, through achievement of key milestone for the BDC-1001 and BDC-2034 programs.

Research and Development (R&D) Expenses – R&D expenses were $14.9 million for the quarter and $40.4 million for the full year ended December 31, 2020, compared to $7.4 million and $26.0 million for the same quarter and year in 2019. The increase in R&D spending from 2019 to 2020 is due primarily to the 2020 start of Bolt’s Phase 1/2 clinical trial for BDC-1001, increased manufacturing of BDC-1001 to support the clinical trial and additional hiring.

General and Administrative (G&A) Expenses – G&A expenses were $2.1 million for the quarter and $9.1 million for the full year ended December 31, 2020, compared to $2.1 million and $5.2 million for the same quarter and year in 2019. The increase in G&A spending from 2019 to 2020 is due primarily to an increase in accounting and legal fees associated with IPO preparation and additional hiring to support operations as a public company.

Loss from Operations – Loss from operations was $16.9 million for the quarter and $49.2 million for the full year ended December 31, 2020 compared to $9.5 million and $31.0 million for the same quarter and year in 2019.

Notable Names Dr. Thomas A. Bock as CEO

On March 31, 2021 Notable Labs Inc., a leader in technology-powered life science with a proprietary platform for predicting patient outcomes and accelerating precision drug development, reported the appointment of Thomas A. Bock, MD, MBA, as Chief Executive Officer (Press release, Notable Labs, MAR 31, 2021, View Source [SID1234577435]). Dr. Bock joined Notable’s Board of Directors in August 2020 and now succeeds Laurie Heilmann as the company’s executive leader, with Notable Founder Matt De Silva remaining as Chairman of the Board.

"Thomas has built four successful life science organizations with tremendous impact for patients and investors, achieving leadership in novel and fast-growing markets including cancer precision medicine and ultra-rare disorders. His dual experience in life science and technology makes him the right leader to champion Notable in its technology-powered advancement of cancer medicine and precision oncology therapies," said De Silva. "Notable’s integration of cutting-edge predictive technology and life science therapeutic programs not only opens strategic commercial opportunities, but also creates a unique workplace blending top talent across tech and life science."

Dr. Bock founded and served as Chief Executive Officer of HeritX Inc., a pioneer in cancer prevention through pre-cancer vaccines, immunoprevention, and gene repair, with the largest cancer prevention pipeline in the industry. Previously, he served as Senior Vice President Medical Affairs on the executive management team of Alexion Pharmaceuticals, building a start-up into a global frontrunner in ultra-rare disorders, a growth leader on NASDAQ, and the world’s second most innovative company, according to Forbes. Before joining Alexion, Thomas built and led the worldwide Medical Departments of Novartis Oncology and Celgene as their Vice President and Global Head of Medical Affairs. Prior to Celgene, Thomas served as the medical head of Amgen Europe for hematology and oncology. He developed and commercialized six lifesaving paradigm-changing blockbuster medicines in cancer, ultra-rare disorders, and inflammation. He is recognized for achieving five of the most successful product launches in life science.

Thomas earned his MD degree at RWTH Aachen University and his MBA at Columbia Business School. He is the Chair of the Healthcare Advisory Board at Columbia Business School, former Chair of the Board of Directors of HeritX Inc., and former Board Chair and President of FORCE, the US patient organization for inherited breast and ovarian cancer.

"I am thrilled to work together with Notable’s outstanding team and seize the tremendous opportunities that our predictive technology platform offers for accelerating the development of personalized cancer treatments," said Bock. "Building on our robust platform, collaborations and insights, we can now focus on the most promising projects and advance them with the sense of urgency that patients deserve."

Affimed and NKMax America Announce FDA Clearance of IND Application to Study the Combination of AFM24, an EGFR Targeted Innate Cell Engager, with SNK-01 Natural Killer Cell Therapy in Solid Tumors

On March 31, 2021 Affimed N.V. (NASDAQ: AFMD), a clinical-stage immuno-oncology company, and NKMax America Inc., a clinical stage biotech company, reported that the U.S. Food and Drug Administration (FDA) cleared an investigational new drug application (IND) for an Affimed and NKMax America co-sponsored Phase 1/2a dose escalation and expansion study in which the two companies will investigate the combination of AFM24, an EGFR/CD16A innate cell engager (ICE), and SNK-01, an autologous NK-cell product, in patients suffering from tumors known to express EGFR (Press release, NKMax America, MAR 31, 2021, View Source [SID1234577434]). The combination represents a novel approach to exploring innate immunity-based therapeutics to treat patients with solid tumors who failed conventional therapy with the aim to improve outcomes for high-medical need patient populations.

"This combination is part of our overall development strategy for AFM24, the first and only innate cell engager in clinical development for solid tumors. In addition to NK cell-based combinations, we are also developing AFM24 as single agent and in combination with atezolizumab in several tumor indications," said Dr. Andreas Harstrick, Affimed’s Chief Medical Officer. "The mechanism of action of the two compounds could be highly synergistic as AFM24 has strong binding affinity to NK cells, directing them to kill tumor cells. Moreover, this combination approach represents an opportunity to supplement patients with dysregulated innate immune systems with targeted cellular therapy."

"The FDA clearance of our IND application for SNK-01 in combination with AFM24 is an important milestone for our Natural Killer cell therapy development program," said Stephen Chen, Chief Operating Officer and Chief Technical Officer of NKMax America. "We look forward to investigating this combination as part of our comprehensive strategy aimed at producing a cell therapy for patients with advanced/metastatic EGFR-expressing cancers."

Further Information About the AFM24/SNK-01 Phase 1/2a study

The Phase 1/2a study is based on preclinical in vitro testing, combining Affimed’s ICE AFM24 with NKMax America autologous NK-cell product SNK-01, which showed enhanced activity of NK cell induced target cell killing. The Phase 1/2a study will be an open-label, non-randomized, multi-center, US only, dose escalation trial to evaluate the combination in adult patients with EGFR-expressing tumors. The primary objective of the phase 1 study part will be to establish the safety and the recommended phase 2 dose of AFM24/SNK-01 combination, as well as to evaluate pharmacokinetics, pharmacodynamics, and preliminary activity in patients with advanced cancers expressing EGFR. The phase 2a portion of the study will evaluate the preliminary efficacy of AFM24 in patients with select solid tumor subtypes.

BioCanRx-funded Researchers Publish Results from CAR T CSEI Project

On March 31, 2021 BioCanRx researchers Dean Fergusson, Justin Presseau, Natasha Kekre, Harold Atkins, Kednapa Thavorn, Rob Holt, Manoj Lalu, and patient representative Terry Hawrysh reported that they have recently published results in two medical journals from their BioCanRx-funded project, "Getting better Outcomes with Chimeric Antigen Receptor T-cell therapy (GO–CART): A BioCanRx Research Excelerator to Safely and Effectively Translate CAR T-Cell Therapy for Hematological Malignancies" (Press release, BioCanRx, MAR 31, 2021, View Source;utm_medium=rss&utm_campaign=biocanrx-funded-researchers-publish-results-car-t-csei-project [SID1234577433]).

The study, which began in 2017, aimed to bring together experts from different backgrounds as well as patient representatives to formulate a feasible, safe, effective, and economical trial protocol that addressed the pitfalls some early-phase trials have faced in the past. Its ultimate aim was to help BioCanRx scientists effectively use CAR T cells in Canada.

Read about the results of their research below:

Navigating choice in the face of uncertainty: using a theory informed qualitative approach to identifying potential patient barriers and enablers to participating in an early phase chimeric antigen receptor T (CAR-T) cell therapy trial

Partnering with patients to get better outcomes with chimeric antigen receptor T-cell therapy: towards engagement of patients in early phase trials

AIM ImmunoTech Reports 2020 Year-End Financial Results

On March 31, 2021 AIM ImmunoTech Inc. (NYSE American: AIM) reported financial results for the fiscal year ended December 31, 2020 and provides a business update (Press release, AIM ImmunoTech, MAR 31, 2021, View Source [SID1234577432]).

2020 Financial Highlights

As of December 31, 2020, AIM had cash, cash equivalents and marketable securities of $54.4 million, compared with $8.8 million as of December 31, 2019.

Research and development expenses for 2020 were $5.7 million, compared with $4.7 million for 2019. General and administrative expenses for 2020 were $8.7 million, compared with $7.0 million for 2019.

The net loss from operations for 2020 was $14.4 million, or $0.45 per share, compared with $9.4 million, or $2.58 per share, for 2019.

Please refer to the full 10-K for complete details.

Update on COVID-19 Pandemic Initiatives

AIM has been actively engaged in determining whether its drug Ampligen could be an effective treatment for COVID-19. Due to Ampligen’s established record of antiviral activity against closely related coronaviruses, AIM believes there is a reasonable probability that its antiviral effects against SARS-CoV-1 will extend to SARS-CoV-2. In animal studies, Ampligen demonstrated complete protection (100% survival) against SARS-CoV-1. This created a compelling case for pre-clinical and clinical testing of Ampligen against SARS-CoV-2 to evaluate Ampligen as a potential prophylactic and early-onset treatment for COVID-19, and, as discussed below, such human clinical trials are now underway.

AIM reached several significant COVID-19 milestones in 2020 and early 2021:

AIM announced that it had identified an effective in vitro model at The Institute for Antiviral Research at Utah State University for testing Ampligen, with the results showing that Ampligen was able to decrease SARS-CoV-2 infectious viral yields by 90% at clinically achievable intranasal Ampligen dosage levels.
AIM reported that Roswell Park Comprehensive Cancer Center’s Phase 1/2a study evaluating the two-drug combination of AIM’s Ampligen and interferon alpha-2b as a potential early-onset treatment for patients with cancer and mild-to-moderate COVID-19 is fully underway, with the first patient enrolled and treated on the study. Based upon that, in March 2020, the company also announced an Institutional Review Board amendment to add a single-agent Ampligen arm to the study.
AIM announced that the post-COVID-19 "Long Hauler" portion of the active AMP-511 Expanded Access Program (EAP) protocol received approval from the Institutional Review Board (IRB) for a public notification of potential patient enrollment. Eligible patients enrolled in the trial receive treatment with Ampligen.
Dosed its first COVID-19 "Long Hauler" patient with the drug Ampligen (rintatolimod). Additional patients are in the process of being enrolled.
Entered into a sponsorship agreement with the Centre for Human Drug Research (CHDR), an independent institute located in Leiden in the Netherlands, for the AMP-COV-100 (CHDR2049) clinical study in the Netherlands on the safety of AIM’s drug Ampligen as an intranasal therapy.
Received approval from the Ethics Committee in the Netherlands to commence its Phase 1 clinical study.
Dosed the first healthy subjects in its Phase 1 clinical study.
Ampligen has shown heightened levels of activity in Phase 2 and 3 trials with Chronic Fatigue Syndrome patients. Ampligen, while experimental in the United States for CFS, is approved in Argentina and is the only late-stage experimental drug for CFS in the U.S. pipeline. Ampligen is also the only drug approved for severe CFS in the world. As witnessed in the prior SARS epidemic of 2002-03, 27% of hospitalized survivors met the U.S. CDC criteria for chronic fatigue syndrome. More than 30 million people in the United States have been infected with SARS-CoV-2, representing a considerable number of people facing potential COVID-induced ME/CFS-like illness in their future. All these facts support our optimism and hopes for the development of a therapy for COVID-induced chronic fatigue.

Update on Cancer Clinical Trials/Programs

Ampligen demonstrated the potential for standalone efficacy in the clinical setting in a number of solid tumors. Six Ampligen clinical trials are currently underway at university cancer centers testing whether tumor microenvironments can be reprogrammed to increase the effectiveness of cancer immunotherapy, including checkpoint inhibitors.

AIM reported receipt of statistically significant positive pancreatic cancer survival results from a multi-year Early Access Program treating 27 subjects with advanced pancreatic adenocarcinoma conducted at Erasmus University Medical Center in the Netherlands. Prof. Casper van Eijck, MD Ph.D., and his team at Erasmus MC found a statistically significantly positive survival benefit when using AIM’s drug Ampligen in patients with locally advanced/metastatic pancreatic cancer after systemic chemotherapy. Median survival was 7.0 months higher in the Ampligen arm as compared to the historical controls. A manuscript for publication is being prepared by the Erasmus MC team.

Towards this end, AIM intends to seek FDA authorization for a follow-up pancreatic cancer Phase 2/3 clinical trial and cancer centers have already expressed interest in serving as clinical sites.

Additionally, Ampligen was awarded an FDA Orphan Drug Designation for the treatment of pancreatic cancer, and a European Medicines Agency orphan medicinal product designation for pancreatic cancer. AIM intends to seek FDA fast-track status for pancreatic cancer. The company will promptly update stockholders and the market as more information on these studies becomes available.

"We are proud to have achieved a number of important milestones throughout 2020 and believe we have a number of key upcoming catalysts. We are conducting important and potentially groundbreaking pre-clinical and clinical research in critical unmet medical needs within large addressable markets. We are in a solid financial position that enables us to continue to execute on our corporate strategy without relying on third-party grants or assistance. Our strong balance sheet also allows us to accelerate our clinical trials without the need to wait for grants. We look forward to updating stockholders and the market as developments unfold," commented Thomas K. Equels, Chief Executive Officer of AIM ImmunoTech.