On January 17, 2023 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a leader in the development of targeted radiotherapies, reported that the Company’s management team will participate in a fireside chat on Thursday, January 19, 2023 at 10:30 AM EST at the 3rd Annual B. Riley Securities Oncology Conference (Press release, Actinium Pharmaceuticals, JAN 17, 2023, View Source [SID1234626275]). The webcast of the presentation will be accessible on the event’s website at View Source and on the investor relations page of Actinium’s website View Source A replay of the presentation will be available on the day of the fireside chat through the same links.

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!

B. Riley Securities 3rd Annual Oncology Conference

B. Riley Securities 3rd Annual Oncology Conference, January 18-19, will feature over 30 SMiD cap healthcare companies focused on the development and commercialization of new medicines and key enabling technologies, cutting across multiple next-generation therapeutic modalities. The conference will feature KOL panel discussions with academic and industry leaders at the forefront of translational and clinical research underway with novel immunotherapy, cell therapy, and targeted oncology approaches, as well as imaging and radiation oncology initiatives, aimed at driving meaningful improvements to current standard of care for cancer patients.

On January 17, 2023 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, and Flame Biosciences, Inc., a privately-held biotechnology company, reported that the companies have entered into a definitive merger agreement pursuant to which Leap has acquired Flame and its assets, including FL-301, its clinical stage anti-Claudin18.2 monoclonal antibody, FL-302, its preclinical anti-Claudin18.2/CD137 bispecific monoclonal antibody, FL-501, its preclinical anti-GDF15 monoclonal antibody, and net cash of approximately $50 million as of December 31, 2022 (Press release, Leap Therapeutics, JAN 17, 2023, View Source [SID1234626274]).

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!

The combined company will continue to trade on Nasdaq under the ticker symbol "LPTX," will be led by Leap’s existing management team, and will also remain focused on advancing DKN-01, Leap’s anti-DKK1 monoclonal antibody, in Phase 2 clinical trials in gastric cancer, endometrial cancer, and colorectal cancer patients. The total cash balance of the combined company as of December 31, 2022 was approximately $115 million, expected to be sufficient to fund Leap’s planned operating expenses and development plans for DKN-01, FL-301, and the preclinical programs to mid-2025.

"This is a transformative transaction for Leap. Acquiring FL-301 is a perfect fit with our vision of developing novel biomarker-targeted therapies for cancer patients, that is represented by our DKN-01 program. We believe that DKK1 and Claudin18.2 will become important patient selection biomarkers in gastric cancer, alongside HER-2 and PD-L1 expression, with the potential for delivering personalized medicines to patients who currently have poor survival outcomes," said Douglas E. Onsi, President & CEO of Leap. "The additional pipeline strength is further enhanced by additional financial strength. With the combined company resources, Leap will focus on executing on the randomized controlled trial for DKN-01 in combination with BeiGene’s tislelizumab and chemotherapy in first-line gastric cancer patients, the new study of DKN-01 in colorectal cancer patients, and the investigator-sponsored trial of DKN-01 in combination with Merck’s pembrolizumab in endometrial cancer patients."

"Flame conducted an extensive strategic process. It was clear that the Leap development team, with its expertise in developing DKN-01, was the ideal partner for FL-301, our preclinical assets, and the Flame shareholders," said Patricia Martin, the Co-Chief Executive Officer of Flame.

"We share Leap’s commitment to developing novel, biomarker-targeted therapies in cancers where there is a significant unmet need and look forward to joining them to bring DKN-01 and FL-301 to patients around the world," said Christian Richard, Head of Public Research of Samsara BioCapital.

Transaction Details

In the merger, Leap will issue approximately 19,794,373 shares of its common stock and approximately 136,833 shares of a newly designated Series X non-voting convertible preferred stock to Flame stockholders. Upon approval by the stockholders of Leap, each share of the Series X non-voting convertible preferred stock will be automatically converted into 1,000 shares of common stock. Flame’s institutional shareholders include Rock Springs Capital, funds and accounts advised by T. Rowe Price Associates, Inc., Janus Henderson Investors, Samsara BioCapital, Adage Capital Management LP, Cormorant Asset Management LP, Surveyor Capital (a Citadel company), Terra Magnum Capital Partners, Logos Capital, and Acuta Capital Partners. In addition, Leap will pay the Flame shareholders 80% of the after-tax net proceeds, if any, from certain post-merger transactions to out-license or sell FL-101 or FL-103, Flame’s anti-IL-1b antibodies.

Leap plans to hold a special meeting of stockholders to approve the conversion of the Series X non-voting convertible preferred stock into shares of common stock and related matters. The number of shares of Leap common stock issuable upon conversion of the Series X non-voting convertible preferred stock are subject to adjustment in the event of any corporate transactions or reverse stock split that may be effectuated by Leap. HealthCare Ventures, which holds 6,763,210 shares or 6.83% of Leap’s outstanding common stock, has signed a support agreement to vote in favor of the proposals to be presented at the shareholder meeting. Assuming the approval by the Leap stockholders, on an as-converted basis, the Flame shareholders will in the aggregate own 58% of the outstanding shares including pre-funded warrants of Leap, and 47.4% on a fully-diluted basis assuming the exercise of all outstanding warrants, options and restricted stock units exercisable for Leap common stock and shares eligible for grant under Leap’s equity incentive plans.

The securities sold in the merger have not been registered under the Securities Act of 1933, as amended ("Securities Act"), or any state or other applicable jurisdiction’s securities laws, and may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Securities Act and applicable state or other jurisdictions’ securities laws. Leap has agreed to file a registration statement with the U.S. Securities and Exchange Commission (the "SEC") to register the resale by the Flame stockholders of the shares of Leap common stock to be issued in connection with the merger.

Raymond James acted as exclusive financial advisor to Leap on the transaction.

Management and Organization

Effective as of the closing of the transactions, the Leap leadership team will continue to be responsible for all executive positions of the combined company. Leap has added two members nominated by Flame to its existing eight person Board of Directors: Patricia Martin and Christian Richard.

About DKN-01

DKN-01 is a humanized monoclonal antibody that binds to and blocks the activity of the Dickkopf-1 (DKK1) protein. DKK1 modulates the Wnt/Beta-catenin and PI3kinase/AKT signaling pathways and has an important role in promoting tumor proliferation, metastasis, angiogenesis, and in mediating an immune suppressive tumor microenvironment through enhancing the activity of myeloid-derived suppressor cells and downregulating NK cell ligands on tumor cells. The U.S. Food and Drug Administration has granted DKN-01 Orphan Drug Designation for the treatment of gastric and gastroesophageal junction cancer and Fast Track Designation in combination with tislelizumab for the treatment of patients with gastric and gastroesophageal junction adenocarcinoma whose tumors express high DKK1 protein, following disease progression on or after prior fluoropyrimidine- and platinum- containing chemotherapy and if appropriate, human epidermal receptor growth factor (HER2)/neu-targeted therapy.

About FL-301

FL-301 is a fully human monoclonal antibody that binds to and blocks Claudin18.2. Claudin18.2 regulates barrier properties and contributes to cell-to-cell adhesion. Expression of Claudin18.2 is very limited in normal tissue, as it is typically buried in the tight junction complex of gastric mucosal cells. In the development of cancer, however, cells lose their polarity and structure. As a result, Claudin18.2 may be exposed and accessible as a target for cancer therapy and is highly expressed on gastric cancer and pancreatic cancer cells. The U.S. Food and Drug Administration has granted FL-301 Orphan Drug Designation for the treatment of gastric and gastroesophageal junction cancer and for the treatment of pancreatic cancer. FL-301 is being developed through an exclusive license from NovaRock Biotherapeutics for territories excluding China and is currently in a Phase 1 clinical trial in cancer patients in China.

Conference Call Information

Leap’s management team will host a conference call today at 9:00 a.m. Eastern Time to discuss the transaction. The live presentation of the conference call can be accessed by registering here. A replay of the event will be available for a limited time and may be access on the Investors page of Leap’s website at View Source

On January 17, 2023 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a biopharmaceutical company focused on advancing innovative immune-oncology technologies addressing hard to treat oncology indications, reported that the Canadian Intellectual Property Office (CIPO) has issued a notice of allowance for Patent Application No. 3,001,543 titled, "Method to Improve Safety and Efficacy of Anti-Cancer Therapy (Press release, Xenetic Biosciences, JAN 17, 2023, https://ir.xeneticbio.com/news/detail/132/xenetic-biosciences-inc-announces-notice-of-allowance-for-canadian-patent-covering-use-of-dnase-enzyme-for-preventing-or-ameliorating-toxicity-associated-with-chemotherapy [SID1234626273])." A patent from the recently allowed application is expected to be issued in the coming months.

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!

"We continue to be excited about the potential of the DNase platform technology and are committed to advancing its development as a top priority. As we continue to execute on our progress towards the clinic, strengthening the intellectual property portfolio around DNase is a focus for us. We are pleased to add this Canadian patent to our existing worldwide IP estate, which includes the U.S. as well as the other patent applications we have filed around the world," commented Jeffrey Eisenberg, Chief Executive Officer of Xenetic Biosciences.

The allowed patent covers claims including use of a therapeutically effective amount of a DNase enzyme for preventing or ameliorating a toxicity associated with a cytostatic and/or cytotoxic chemotherapy in a subject suffering from a cancer and received or deemed to receive said chemotherapy, wherein said amount of the DNase enzyme is effective to prevent or ameliorate at least one side effect of said chemotherapy.

The Company’s interventional DNase based oncology platform is aimed at improving outcomes of existing treatments, including immunotherapies. The Company’s exclusive license to CLS Therapeutics’ intellectual property for uses of DNases in cancer include systemic co-administration of DNases along with standard therapies, including chemotherapy, radiation and checkpoint inhibitors, or along with conventional chimeric antigen receptor (CAR) T therapies. Xenetic is focused on advancing its systemic DNase program into the clinic as an adjunctive therapy for pancreatic carcinoma and other locally advanced or metastatic solid tumors.

On January 17, 2023 Biomea Fusion, Inc. ("Biomea") (Nasdaq: BMEA), a biopharmaceutical company focused on the discovery and development of covalent small molecules to treat patients with genetically defined cancers and metabolic diseases, reported that the first patient has been dosed in COVALENT-102, the company’s Phase I/Ib trial of BMF-219, an oral, selective, covalent menin inhibitor in patients with KRAS-mutated unresectable, locally advanced, or metastatic NSCLC, CRC, and PDAC (Press release, Biomea Fusion, JAN 17, 2023, View Source [SID1234626272]).

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!

"We are eager to explore the potential of BMF-219 as a pan-KRAS inhibitor in patients with three of the most prominent KRAS-mutant solid tumor types, including those with tumors that have failed to respond to investigational and approved mutation-specific KRAS inhibitors," said Steve Morris, M.D., Biomea’s Chief Medical Officer. "As a covalent menin inhibitor, we believe BMF-219 has critical advantages over late stage, mutation-specific inhibitors of KRAS including independence on the KRAS activation state, reduced likelihood for acquisition of resistance mutations, and its potential to address multiple activating KRAS mutations."

KRAS is the most frequently mutated isoform amongst RAS oncogenes in human solid tumors, with high prevalence in NSCLC, CRC, and PDAC. KRAS G12C, KRAS G12D and KRAS G12V are among the most common KRAS mutations. However, there are numerous other known activating KRAS mutations. With only two approved therapies both targeting only KRAS G12C for locally advanced or metastatic NSCLC, KRAS-driven tumors continue to represent a significant unmet medical need.

As a covalent menin inhibitor, BMF-219 has manifested a differentiated profile over the commercially approved KRAS-targeted inhibitors LUMAKRAS (sotorasib) and KRAZATI (adagrasib) in multiple pre-clinical studies. As previously reported by Biomea, KRAS-mutant NSCLC, CRC, and PDAC cell lines and ex vivo patient specimens were highly sensitive to BMF-219 in preclinical models. The higher levels of activity of BMF-219 were observed among various KRAS-mutant solid tumor cell lines, but not KRAS wild type, suggesting that BMF-219 broadly inhibited mutant KRAS in these tumor models. A targeted pan-KRAS inhibitor has the potential to treat a large number of NSCLC, CRC, and PDAC patients.

About COVALENT–102
COVALENT-102 is an open-label, multi-cohort, multicenter, Phase I/Ib dose escalation and expansion study evaluating the safety, tolerability, and optimal biologic dose of BMF-219 administered orally to adult patients with KRAS-mutated unresectable, locally advanced or metastatic NSCLC, CRC, and PDAC. Additional information about the Phase I/Ib clinical trial of BMF-219 can be found at ClinicalTrials.gov using the identifier NCT05631574.

About Non-Small Cell Lung Cancer (NSCLC)
NSCLC is the most common form of lung cancer, representing approximately 82% of all lung cancer cases or approximately 200,000 cases in the U.S. each year (Source: NCI SEER Data). The five-year survival rate of NSCLC is approximately 25%. While lung cancer is the third most common form of cancer in the U.S. based on incidence, it contributes to the highest number of annual cancer deaths in the U.S. KRAS is the most frequently mutated oncogene in NSCLC, occurring in approximately 30% of patients. There remains a great unmet need for targeted therapies to address all KRAS driver mutations and avoid known mechanisms of resistance.

About Colorectal Cancer (CRC)
CRC is the fourth most common form of cancer and the second leading cause of cancer death in the U.S., representing approximately 150,000 cases in the U.S. each year (Source: NCI SEER Data). These cancers start in the rectum or the colon and can be diagnosed/identified early, even potentially as noncancerous polyps. The five-year survival rate of CRC is approximately 65%. Among other mutations, KRAS mutations occur in approximately 40% of patients with CRC. These mutations can not only help predict the absence of response to anti-EGFR therapy, but also result in poorer overall survival. Therefore, there remains a significant unmet need for personalized therapies for patients with KRAS-mutant colorectal cancer.

About Pancreatic Cancer (PDAC)
Pancreatic cancer is a relatively rare form of cancer in the U.S., representing approximately 60,000 cases in the U.S. each year (Source: NCI SEER Data). Pancreatic cancer is an aggressive cancer with a very low five-year survival rate of approximately 11%, indicating that there is a large unmet need. 80% of patients are diagnosed at an advanced stage, contributing to the low survival rate. KRAS mutations are found in nearly all pancreatic cancer patients and are considered as a driver of the malignant process in most of those patients.

On January 17, 2023 Idera Pharmaceuticals, Inc. ("Idera") (Nasdaq: IDRA), a clinical-stage biopharmaceutical company committed to transforming the care of people living with rare pulmonary and rheumatic diseases, reported that the Company has changed its name and symbol to Aceragen, Inc. ("Aceragen," the "Company," "we," "us," or "our") and "ACGN". Additionally, the Company’s stockholders approved the conversion into common stock of the Series Z preferred shares resulting from the previously announced merger with Aceragen and authorized a reverse split of common stock (Press release, Idera Pharmaceuticals, JAN 17, 2023, View Source [SID1234626271]). The Company’s Board of Directors has approved the reverse stock split at a ratio of 1-for-17 shares. As a result of these changes, which will be effective upon the market open on Wednesday, January 18, 2023, the Company will be in compliance with all applicable Nasdaq listing standards. Nasdaq has issued an approval letter confirming Aceragen’s listing.

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!

"We are excited to have completed the transformation of Aceragen via our merger with Idera and the subsequent adjustments to our stock and Nasdaq listing. We believe this transition strengthens our portfolio of late-stage clinical assets in cystic fibrosis and Farber disease and aligns with our goal of delivering important therapies for people living with rare diseases," stated John Taylor, Aceragen’s Chief Executive Officer. "During the course of this year, we anticipate the achievement of significant clinical milestones that include two Phase 2 data read-outs for ACG-701, as well as lifting of the clinical hold and advancing toward the initiation of our Phase 2/3 trial in Farber disease for ACG-801."

"With the positive result of the stockholder vote behind us, I share in John’s excitement and optimism for Aceragen and look forward to the progress that this team will make for patients in need," added Vincent J. Milano, Chair of Aceragen’s Board of Directors.

Clinical Development Overview
Aceragen has a portfolio of late-stage clinical assets in cystic fibrosis and Farber disease with clinical milestones anticipated in 2023.

ACG-701 for Acute Pulmonary Exacerbations in Cystic Fibrosis
ACG-701 is a proprietary oral, loading dose formulation of sodium fusidate being developed as a treatment for acute pulmonary exacerbations ("PEx") associated with cystic fibrosis ("CF"), a major factor driving lung function decline in people living with CF. Sodium fusidate has an established clinical efficacy and safety profile from more than 50 years of use in other countries, including as part of CF PEx treatment guidelines in the United Kingdom and Australia. Despite this, the compound has never been approved by the FDA and represents a new and potentially powerful approach in the United States to address the infection, inflammation, and enhanced mucin expression that are hallmark features of CF PEx.

A Phase 2 trial of ACG-701 in CF PEx (the REPRIEVE study), a randomized double-blinded, placebo-controlled study, was initiated in December 2022 at clinical sites in the United States in collaboration with the CF Foundation’s Therapeutic Development Network (TDN). The CF Foundation has also provided funding of $3.5 million in support of the study. If approved, ACG-701 would represent the first product in the United States indicated for the treatment of newly diagnosed CF PEx patients. Data from the REPRIEVE study is expected in 2H 2023. The FDA has granted Orphan Drug Designation, Fast Track and Qualified Infectious Disease Product (QIDP) status to ACG-701 for CF PEx.

ACG-701 for Melioidosis
Aceragen has also executed an ~$50 million development partnership with the Department of Defense’s Defense Threat Reduction Agency ("DTRA") to investigate ACG-701 as a potential medical countermeasure for melioidosis, a life-threatening infection caused by the B. pseudomallei pathogen. This program is centered around a Phase 2 trial, the TERRA study (NCT05105035), which is a randomized double-blind, placebo-controlled trial conducted in hospitalized melioidosis patients. TERRA was initiated in May 2022 and continues to actively enroll patients with an independent DMC review planned during Q1 2023 and a data read-out expected in 2H 2023.

ACG-801 for Farber Disease
ACG-801, recombinant human acid ceramidase, is an investigational biologic in development to be the first-ever enzyme replacement therapy for the treatment of Farber disease, a progressive, severe, and life-threatening lysosomal storage disorder that is caused by the monogenic deficiency of acid ceramidase. The biochemical hallmark of Farber disease is the loss of acid ceramidase enzyme activity leading to abnormal accumulation of ceramide, profound macrophage-driven inflammation and multi-organ disease affecting bone and joints, cartilage, the immune system, central nervous system, and the lungs. Complications of the disease are life threatening, with many patients dying in the first years of life. There are no ceramide-targeted medications currently available that can alter the disease natural history.

The Company expects to initiate the ADVANCE clinical study for ACG-801 in Farber disease, a randomized, double-blind, placebo-controlled, first-in-human study, in the first quarter of 2024 with data expected in the first quarter of 2025. Due to the ultra-rare nature of Farber disease, if successful, this study has the potential to support registration of the product. The FDA has granted Orphan Drug, Fast Track, and Rare Pediatric Disease designations for ACG-801. Rare pediatric disease designation permits priority review voucher eligibility, upon FDA marketing authorization.

Clinical Milestone Summary

ACG-701 – REPRIEVE study in CF PEx, data expected 2H 2023
ACG-701 – TERRA study in melioidosis, data expected 2H 2023
ACG-801 – ADVANCE study in Farber disease, initiation expected in Q1 2024
Projected cash available is expected to provide the Company with capital runway into Q3’2023.