On December 16, 2020 Novartis reported that the U.S. Food and Drug Administration (FDA) granted iptacopan (LNP023) Breakthrough Therapy Designation (BTD) in paroxysmal nocturnal hemoglobinuria (PNH) and Rare Pediatric Disease (RPD) Designation in C3 glomerulopathy (C3G) (Press release, Novartis, DEC 16, 2020, https://www.novartis.com/news/media-releases/novartis-investigational-oral-therapy-iptacopan-lnp023-receives-fda-breakthrough-therapy-designation-pnh-and-rare-pediatric-disease-designation-c3g [SID1234634302]).

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 breakthrough designation is intended to expedite the development and review of medicines for serious conditions to address unmet medical need, where early clinical evidence indicates a drug may demonstrate substantial improvement over available therapy on clinically significant endpoints. The FDA granted BTD to iptacopan for the treatment of PNH based on positive interim results from two ongoing Phase II studies, where iptacopan showed substantial benefits both in patients who remained anemic and dependent on transfusions despite standard of care anti-complement treatment8, as well as monotherapy in anti-C5 naïve PNH patients.

PNH is a rare and life threatening blood disorder characterized by complement-driven hemolysis, thrombosis and impaired bone marrow function9,10, resulting in anemia, fatigue and other debilitating symptoms that can impact patients’ quality of life1–3. Despite current standard of care – anti-C5 therapy eculizumab or ravulizumab – a large proportion of PNH patients remain anemic and dependent on transfusions1,2,9,11,12.

FDA grants the rare pediatric designation for serious or life-threatening diseases primarily affecting individuals aged 18 years or younger and impacting fewer than 200,000 people. C3G, an ultra-rare and severe form of primary glomerulonephritis4,13, is characterized by complement dysregulation. It has a poor prognosis; about 50% of patients progress to end-stage renal disease (ESRD) within 10 years, and 50–70% experience disease recurrence post kidney transplant14. It has a worldwide annual incidence of 1-2 per million15.

About iptacopan
Iptacopan (LNP023) is a first-in-class, orally administered, potent and highly selective factor B inhibitor of the alternative complement pathway6,7. It is currently in clinical development for PNH, as well as C3G and a number of other renal conditions with complement system involvement where significant unmet needs exist, including IgA nephropathy (IgAN), atypical hemolytic uremic syndrome (aHUS), and membranous nephropathy (MN).

Positive Phase II data from one study in PNH were presented at the European Society for Blood and Marrow Transplantation (EBMT) congress in August8, and Phase II interim analysis results in C3G were presented at the virtual 2020 Annual Meeting of the American Society of Nephrology (ASN) in October. Novartis is planning to initiate Phase III studies in several indications.

Iptacopan has the potential to become the first complement pathway inhibitor to slow disease progression in a number of complement-driven diseases. Based on disease prevalence and the positive interim data from Phase II studies, iptacopan has also received orphan drug designations from the FDA and EMA in C3G and PNH16, as well as EMA PRIME designation for C3G17, and EMA orphan drug designation in IgAN18.

On December 16, 2020 The University System of Maryland (USM) Momentum Fund reported that it has invested $250K in miRecule, an emerging biotech company with a cutting-edge discovery platform to create RNA therapeutics alongside theFSHD Society as a co-investor (Press release, miRecule, DEC 16, 2020, View Source [SID1234574132]). This investment is complemented by recent funding raised over the past year, which includes lead investor Alexandria Venture Investments as well as Pathway Bioventures, Alumni Ventures Group, and angel investors. In total, miRecule’s seed round raised more than $3.5M in private seed funding. In addition to its private investments miRecule has also been awarded a non-dilutive $2M Phase II Small Business Innovation Research (SBIR) grant from the National Cancer Institute.

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!

miRecule’s DREAmiRTM discovery platform was developed through more than a decade of research in collaboration with the National Institutes of Health in Bethesda, Md. DREAmiR analyzes genomic and outcome data from thousands of patients to identify the underlying genetic abnormalities causing a disease in an individual patient. The company then creates an RNA therapeutic to directly target and fix the abnormality. miRecule is applying this platform to a variety of diseases, developing lifesaving RNA therapeutics for cancer, muscular dystrophy, and even COVID-19.

"We are excited to add another therapeutic company to our investment portfolio. miRecule’s technology has the potential to be incredibly impactful for the health care industry and certainly for individual patients," said Claire Broido Johnson, managing director of the Momentum Fund. "In addition to an innovative product, miRecule has a strong management team and we have a lot of confidence in their ability."

miRecule was founded in Biohealth Innovation’s incubator in Montgomery County (Md.) by Chief Executive Officer (CEO) Anthony D. Saleh, PhD, and Chief Operating Officer Ashwin Kulkarni, MS, an alum of University of Maryland, College Park. The majority of miRecule’s seed round funding will be used to advance their lead program, MC-30, which addresses head and neck cancer, towards clinical development. Head and neck cancer is the sixth most common form of cancer worldwide, and for many patients, survival is measured in months not years.

"We are hopeful that MC-30 will be a real game changer," said CEO Dr. Saleh. "By correcting the underlying mutations that make a patient resistant to treatment, we think we can triple response rates and give years of quality life back to many patients."

On December 16, 2020, Cannabics Pharmaceuticals Inc. (the "Company") reported that it entered into a Securities Purchase Agreement (the "SPA") with an institutional investor (the "investor" or a "holder") to sell a new series of senior secured convertible notes (the "Convertible Notes") of the Company in a four tranche private placement (the "Private Placement") to the investor, with an aggregate principal amount of $2,750,000 having an aggregate original issue discount of 10%, and ranking senior to all outstanding and future indebtedness of the Company (Filing, 8-K, Cannabics Pharmaceuticals, DEC 16, 2020, View Source [SID1234573192]). Pursuant to the SPA, one Convertible Note (the "Initial Note") in an aggregate original principal amount of $825,000 will be issued to the investor in the first tranche in reliance upon the exemption from securities registration afforded by Section 4(a)(2) of the Securities Act of 1933, as amended (the "1933 Act"), and Rule 506(b) of Regulation D ("Regulation D") as promulgated by the United States Securities and Exchange Commission (the "SEC") under the 1933 Act, together with the issuance of warrants (the "Warrants" and, together with the Convertible Note, the "Securities") to acquire our common stock , as described below. The Initial Note has a face amount of $825,000 for which the Company shall receive cash proceeds of $750,000. Subject to the satisfaction of customary closing and equity conditions, at any time prior to December 31, 2021 (or such later date as the parties shall mutually agree), the Company has the right to require additional closings of tranches of additional notes (the "Additional Notes", and together with the Initial Notes, the "Notes") as follows: (x) $825,000 in aggregate principal of Additional Notes upon the effectiveness of a registration statement with respect to the shares of our common stock issuable upon conversion of the Notes (or, if earlier the date such underlying shares of our common stock are initially available to be resold pursuant to Rule 144 of the Securities Act of 1933, as amended (or if later, such date as the Company is then current in its public filings)) (the "Applicable Date"), (y) $550,000 in aggregate principal of Additional Notes upon the 90th calendar day anniversary of the Applicable Date and (z) $550,000 in aggregate principal of Additional Notes upon the 180th calendar day anniversary of the Applicable Date s. The Convertible Notes are being sold with an original issue discount of 10% and do not bear interest except upon the occurrence of an event of default.

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 following descriptions of the Convertible Notes, Warrants, Security and Pledge Agreement, Guaranty, Registration Rights Agreement and SPA are not complete and are qualified in their entirety by reference to the full text of such agreements, the forms of which are attached as Exhibit 10.1 through 10.6 to this Form 8-K, respectively.

On December 16, 2020 TRIUMF, Canada’s particle accelerator centre, reported it has entered into a collaboration agreement with Fusion Pharmaceuticals Inc. (Nasdaq: FUSN), a clinical-stage oncology company focused on developing next-generation radiopharmaceuticals as precision medicines (Press release, Fusion Pharmaceuticals, DEC 16, 2020, View Source [SID1234573117]). Under the agreement, Fusion will provide a financial investment enabling TRIUMF to upgrade its actinium-225 production infrastructure, and in return will receive preferred access to actinium-225, a rare medical isotope that shows great promise in new, cutting-edge cancer therapies.

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!

An alpha-emitting isotope with a short half-life, actinium-225 can be combined with a molecular agent that specifically targets cancer cells, seeking out and destroying the cancer while leaving the surrounding tissue unharmed. The new collaboration will enable TRIUMF to significantly increase its production and delivery of actinium-225.

"Today’s announcement marks an important step in positioning Canada to play a leading role in the development and deployment of next-generation radiotherapies, and in ensuring that researchers and patients around the world have a stable supply of life-saving medical isotopes," said Dr. Jonathan Bagger, Director of TRIUMF. "Enabled by decades of public investment in TRIUMF’s infrastructure and research programs, this collaboration recognizes the laboratory’s capacity to drive innovation, moving this promising treatment closer to market."

"Given Fusion’s opportunity to expand our pipeline of actinium-based Targeted Alpha Therapies (TATs), and the importance of isotope production in the supply chain of radiopharmaceuticals, we will continue to proactively address and prioritize actinium supply in our strategic plans," said Dr. John Valliant, Chief Executive Officer of Fusion. "We are excited to collaborate with TRIUMF, a leader in isotope production, as part of these plans."

"With its Targeted Alpha Therapy platform technology, Fusion has an opportunity to impact the cancer treatment landscape," said Kathryn Hayashi, Chief Executive Officer of TRIUMF Innovations. "Through this collaboration agreement, we are partnering with a premier developer of innovative radiotherapies to deepen TRIUMF’s leadership position in isotope production to help save the lives of patients in Canada and around the world".

On December 16, 2020 Cerecor Inc. (NASDAQ: CERC), a biopharmaceutical company focused on becoming a leader in development and commercialization of treatments for rare and orphan diseases, reported that it has dosed its first patient in a Phase 1b clinical trial of CERC-007 (Press release, Cerecor, DEC 16, 2020, View Source [SID1234573113]). CERC-007 is a high affinity, fully human anti-IL-18 monoclonal antibody (mAb) being developed for patients with relapsed or refractory multiple myeloma (MM). The study will determine the recommended Phase 2 dose, safety and preliminary efficacy of CERC-007. The Company anticipates initial data to be reported in the first quarter of 2021.

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 very excited to have dosed our first patient. We are on track for initial data during the first quarter of 2021," said H. Jeffrey Wilkins, MD, Chief Medical Officer of Cerecor. "Multiple myeloma is the second most common form of blood cancer and is characterized by anemia, bone pain and fatigue with the majority of patients relapsing or becoming refractory on current treatments. Elevated levels of IL-18 are correlated with poor survival in patients with multiple myeloma.1 The approximately 50% mortality rate at five years reinforces the need for novel and targeted therapies, such as CERC-007, as another treatment option."

The Phase 1b clinical trial is a U.S. multicenter, open-label, dose-escalation, sequential group study of CERC-007 as a monotherapy in approximately 30 patients with relapsed or refractory MM. The primary objectives of the study will be to determine the safety and tolerability of CERC-007, the recommended Phase 2 dose, and preliminary efficacy as measured by response rate in accordance with International Myeloma Working Group (IMWG) criteria.

About Multiple Myeloma
Multiple myeloma is the second most common blood cancer, with approximately 140,000 patients in the United States.2 Multiple myeloma is characterized by an excess proliferation of plasma cells. Despite increased availability of new agents, the disease is characterized by a pattern of recurrent relapses and remains incurable for the majority of patients, with a 5-year survival rate of approximately 50%.2

About CERC-007
CERC-007 is a high affinity, fully human monoclonal antibody targeting the proinflammatory cytokine IL-18. It is in development for multiple auto-immune diseases, including Still’s disease (adult onset Still’s disease (AOSD) and systemic juvenile idiopathic arthritis (sJIA)), and multiple myeloma (MM).