On January 13, 2021 Kintara Therapeutics, Inc. (Nasdaq: KTRA) ("Kintara" or the "Company"), a biopharmaceutical company focused on the development of new solid tumor cancer therapies, reported that patient recruitment has commenced in the Global Coalition for Adaptive Research (GCAR) registrational Phase 2/3 clinical trial for glioblastoma (GBM) (Press release, Kintara Therapeutics, JAN 13, 2021, View Source [SID1234573969]). The trial, titled GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment), is a revolutionary, patient-centered, adaptive platform trial for registration evaluating multiple therapies for patients with newly-diagnosed and recurrent GBM.

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Key GBM AGILE Highlights for VAL-083

Only therapeutic agent currently being evaluated in all three GBM patient subtypes: newly-diagnosed methylated MGMT; newly-diagnosed unmethylated MGMT; and recurrent
May accelerate VAL-083’s time to pivotal trial completion and potential regulatory submission by up to 18 months
Cost-effective opportunity to advance VAL-083 due to the GBM AGILE study’s expense sharing protocol
Anticipating rapid enrollment, targeting 150-200 patients with 34 active and recruiting U.S. sites with plans to increase to 40 sites during Q1, 2021, including Canada and other international locations to follow
"The entire Kintara team is grateful and excited to participate in GCAR’s groundbreaking GBM AGILE study as it offers an extraordinary opportunity to facilitate the advancement of VAL-083’s clinical development in a premier GBM study," commented Saiid Zarrabian, Kintara’s Chief Executive Officer. "This is truly an important milestone for Kintara as we believe the study will generate important insights into the breadth of VAL-083’s potential to address this deadliest form of brain cancer in all patient subtypes, while potentially bringing the program to the doorstep of commercialization."

GBM AGILE is an international, innovative platform trial designed to more rapidly identify and confirm effective therapies for patients with glioblastoma through response adaptive randomization and a seamless Phase 2/3 design. The trial, conceived by over 130 key opinion leaders, is conducted under a master protocol allowing multiple therapies or combinations of therapies from different pharmaceutical partners to be evaluated simultaneously. With its innovative design and efficient operational infrastructure, data from GBM AGILE can be used as the foundation for a new drug application and biologics license application submissions and registrations to the FDA and other health authorities.

Kintara’s VAL-083 is a "first-in-class," small molecule bifunctional alkylating agent that crosses the blood-brain barrier. VAL-083 is independent of the MGMT resistance mechanism and has been assessed in over 40 Phase 1 and Phase 2 clinical trials in multiple indications sponsored by the U.S. National Cancer Institute (NCI). Published pre-clinical and clinical data indicate that VAL-083 has activity against a range of tumor types, including lung, brain, cervical, ovarian tumors and hematologic (blood) cancers. VAL-083 has been granted Orphan Drug Designation for GBM by the FDA and EMA and has also been granted Orphan Drug Designations for medulloblastoma and ovarian cancer by the FDA. In addition, the FDA granted Fast Track Designation for VAL-083 in recurrent GBM. VAL-083 is approved as a cancer chemotherapeutic in China for the treatment of chronic myelogenous leukemia and lung cancer. VAL-083 has not been approved for any indications outside of China.

On January 13, 2021 Harpoon Therapeutics, Inc. (NASDAQ: HARP), a clinical-stage immunotherapy company developing a novel class of T cell engagers, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for HPN217 for the treatment of multiple myeloma (Press release, Harpoon Therapeutics, JAN 13, 2021, View Source [SID1234573968]). HPN217, a tri-specific T cell activating recombinant protein construct (TriTAC) targets B-cell maturation antigen (BCMA), a well-validated antigen expressed on malignant multiple myeloma cells. Harpoon has four drug product candidates in clinical development for the treatment of solid and hematologic malignancies based on its proprietary TriTAC platform.

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"Orphan Drug Designation for multiple myeloma represents a significant milestone in the development of HPN217 and recognizes its potential to address a significant unmet medical need for the patients suffering from this condition," said Jerry McMahon, Ph.D., President and Chief Executive of Harpoon Therapeutics. "I am pleased with the clinical progress we are making with this program and we are planning to present interim data from the ongoing Phase 1/2 dose escalation trial later this year."

The FDA’s Orphan Drug Designation program provides orphan status to drugs defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan Drug Designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity upon FDA approval.

About the Phase 1/2 Trial for HPN217

In April 2020, Harpoon announced dosing of the first patient with HPN217 (BCMA TriTAC) in a Phase 1/2 clinical trial focused on relapsed/refractory multiple myeloma (RRMM). HPN217 is covered by a global development and option agreement with AbbVie Inc. (NYSE: ABBV). Dose escalation for HPN217 in the Phase 1/2 clinical trial is progressing rapidly. HPN217 has been well tolerated, and no DLTs had been observed as of the most recent December 1, 2020 data cutoff date. A presentation of interim data is anticipated in 2021, with initiation of a dose expansion cohort in the second half of 2021.

On January 13, 2021 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium") reported the research collaboration with Astellas Pharma Inc. (TSE:4503) ("Astellas") to develop novel targeted radiotherapies using its Antibody Warhead Enabling (AWE) technology platform. (Press release, Actinium Pharmaceuticals, JAN 13, 2021, View Source [SID1234573967]) Under this agreement, Actinium will utilize its AWE Platform technology to develop and characterize selected Astellas targeting agents labeled with the potent alpha-emitting radioisotope Actinium-225 (Ac-225). This collaboration is a component of Astellas’ internal initiative to develop theranostics as part of its Rx+ business (For more information, please visit View Source).

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"We are excited to execute on this research collaboration with Astellas, a global leader at the forefront of healthcare innovation," stated Dr. Dale Ludwig, Chief Scientific and Technology Officer of Actinium. "Targeted radiotherapy has a highly differentiated mechanism of action with the potential to address multiple disease indications and therefore has been an area of significant exploration and investment of late. Through our AWE technology platform, we are able to bring our significant know-how in working with Actinium-225, as well as our robust clinical experience with targeted radiotherapies and supply chain to bear in this collaboration. With our enhanced laboratory capabilities and the expertise of our R&D team, we are well positioned to execute on this collaboration with Astellas as well as our own R&D strategy, to complement our Iomab-B and Actimab-A clinical programs."

The intellectual property encompassing Actinium’s AWE technology platform covers its gold standard linker technology, methods of ARC manufacture in addition to methods of use for ARCs in multiple diseases, including indication, dose and scheduling, radionuclide warhead, and therapeutic combinations. Actinium’s AWE technology patent portfolio includes 30 patent families comprised of over 135 issued or pending global patent applications, of which 10 are issued and 24 pending in the United States.

Sandesh Seth, Chairman and Chief Executive Officer of Actinium, added, "Through our development efforts, Actinium has established itself as a leader in the field of Actinium-225 based targeted radiotherapies. We have amassed the most experience treating patients with Actinium-225 via our CD33 program’s several Actimab-A trials and have gained important insights in developing targeted radiotherapies through the execution of the Actimab-A and also SIERRA Phase 3 pivotal trial for our lead asset, Iomab-B. This expertise and experience with radiopharmaceuticals has been invaluable to us and we believe will also be to Astellas in this collaboration. As Iomab-B and Actimab-A advance in the clinic with multiple clinical milestones upcoming from each of our trials, we look forward to executing this collaboration and furthering the field of targeted radiotherapy with Astellas."

About Our Antibody Warhead Enabling Platform Technology

The Antibody Warhead Enabling (AWE) Program has at its centerpiece the AWE Platform Technology. The Company’s proprietary AWE Platform Technology is supported by intellectual property and know-how that enables the creation of Actinium-225 (Ac-225) Radio-Conjugates (ARCs) wherein a biomolecular targeting agent is stably labeled with the powerful Ac-225 payload to enhance targeted cell killing. The AWE Platform is protected by intellectual property covering the use of the "gold standard" chelator DOTA, and any conceivable derivative thereof. Additionally, Actinium holds intellectual property protection covering methods of chelation or labeling of the targeting agent with Ac-225, including newer next-generation methodologies for chelation of Ac-225.

The AWE Program is structured to provide the opportunity for partners or collaborators to derive maximum value from a collaboration by leveraging Actinium’s extensive technical know-how, access to its ARC drug development infrastructure and to its underlying AWE Platform Technology. The AWE Program provides a partner or collaborator with access to Actinium’s knowledge bank and infrastructure allowing collaborators to benefit from accelerated development timelines for its ARCs.

To learn more about the AWE Technology Platform or the AWE Program please contact our Business Development team at [email protected].

On January 13, 2021 Affimed N.V. ("Affimed" or the "Company") (Nasdaq: AFMD), a clinical-stage immuno-oncology company committed to giving patients back their innate ability to fight cancer, reported the pricing of its previously announced public offering of 16,666,667 of its common shares at a public offering price of $6.00 per common share (Press release, Affimed, JAN 13, 2021, View Source,of%20its%20common%20shares%20at [SID1234573966]). In addition, Affimed has granted the underwriters a 30-day over-allotment option to purchase up to 2,500,000 common shares at the public offering price less underwriting discounts. After deducting the underwriting discounts, the net proceeds of the public offering are expected to be approximately $94 million. The offering is expected to close on or about January 15, 2021, subject to customary closing conditions.

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Jefferies LLC, SVB Leerink LLC and Credit Suisse Securities (USA) LLC are acting as joint book-running managers and Laidlaw & Company (UK) Ltd. is acting as co-manager. A shelf registration statement relating to these securities filed with the Securities and Exchange Commission (the "SEC") was declared effective by the SEC on December 30, 2020. The offering will be made only by means of a prospectus and prospectus supplement. A preliminary prospectus supplement and accompanying prospectus related to the offering have been filed with the SEC and are available at the SEC’s website located at www.sec.gov. A final prospectus supplement and accompanying prospectus will be filed with the SEC. Copies of the preliminary prospectus supplement and accompanying prospectus related to the offering may be obtained by contacting Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, or by telephone at (877) 821-7388 or by email at [email protected], SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525 ext. 6132, or by email at [email protected], or Credit Suisse Securities (USA) LLC, Attention: Prospectus Department, 6933 Louis Stephens Drive, Morrisville, NC 27560, by telephone at (800) 221-1037, or by email at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On January 13, 2021 Castle Biosciences, Inc. (Nasdaq: CSTL), a skin cancer diagnostics company providing personalized genomic information to improve cancer treatment decisions, reported certain unaudited preliminary performance results for the fourth quarter and full-year 2020 (Press release, Castle Biosciences, JAN 13, 2021, View Source [SID1234573965]).

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"At Castle, we are committed to providing innovative, clinically actionable dermatologic tests to improve patient outcomes," said Derek Maetzold, president and chief executive officer of Castle Biosciences. "In 2020, despite the challenges presented to us all by the global pandemic, our team remained focused on this commitment and performed exceptionally.

"We delivered year-over-year volume growth in 2020 in DecisionDx-Melanoma reports, our current lead revenue driver, despite a significant reduction in melanoma diagnoses that we believe was due to COVID-19. We also expanded our skin cancer suite of products, adding two new proprietary gene expression profile tests and increasing the estimated U.S. total addressable market (TAM) of our commercially available tests to $2 billion. We expanded our body of evidence in 2020, with 11 supportive peer-reviewed articles published for our four proprietary gene expression profile tests during the year. Evidence development remains a key component of our growth strategy, supporting adoption of our tests by clinicians and reimbursement by commercial payers.

"Even in the face of COVID-19, we more than doubled our employee headcount since the beginning of 2020 through December. Additionally, we completed two equity financings, further strengthening our balance sheet, as we look to accelerate future growth through expanded support for our currently marketed tests, including further commercial team expansion in the first half of 2021, and through our pipeline development."

Fourth Quarter Ended December 31, 2020, Highlights

Delivered 5,157 total gene expression profile test reports for all four marketed tests combined in the fourth quarter of 2020:
DecisionDx-Melanoma test reports delivered in the quarter were 4,246.
DecisionDx-SCC test reports delivered in the quarter were 428.
DecisionDx DiffDx-Melanoma test orders received and reports delivered from Nov. 2 through Dec. 31, 2020, were 92 and 73, respectively.
DecisionDx-UM test reports delivered in the quarter were 410.
Medicare Administrative Contractor (MAC), Palmetto GBA MolDx, issued a final expanded local coverage determination (LCD) and an accompanying billing and coding article for the Company’s DecisionDx-Melanoma test. Noridian, the MAC that oversees Castle’s laboratory in Arizona, issued an identical LCD and billing and coding article, which became effective Dec. 6, 2020. Since the effective date of Dec. 6, 2020, more than 90% of Medicare beneficiaries whose clinicians order DecisionDx-Melanoma as part of their melanoma management plan were covered under the expanded LCD. Additionally, final 2021 Medicare rates for both DecisionDx-Melanoma and DecisionDx-UM were published on Dec. 28, 2020, on the Centers for Medicare & Medicaid Services (CMS) website.
The Company launched its DecisionDx DiffDx-Melanoma test on Nov. 2, 2020. DecisionDx DiffDx-Melanoma is designed to aid dermatopathologists and dermatologists in characterizing difficult-to-diagnose melanocytic lesions.
On Dec. 18, 2020, Castle completed its underwritten public offering of 4.6 million shares of its common stock at a public offering price of $58.00 per share. The gross proceeds to Castle from the offering before deducting the underwriting discounts and commissions and other offering expenses payable by Castle, were $266.8 million.
Year- Ended December 31, 2020, Highlights

Total gene expression profile test reports delivered in 2020 were 18,185:
DecisionDx-Melanoma test reports delivered in 2020 were 16,232. While third-party data for December is still finalizing, data through November suggests that diagnoses of melanoma were down 19% in 2020 over the prior year.
DecisionDx-SCC test orders received and reports delivered in 2020 (Aug. 31, 2020 – Dec. 31, 2020) were 562 and 485, respectively.
DecisionDx DiffDx-Melanoma test orders received and reports delivered in 2020 (Nov. 2, 2020 – Dec. 31, 2020) were 92 and 73, respectively.
DecisionDx-UM test reports delivered in 2020 were 1,395.
The Company launched its DecisionDx-SCC test on Aug. 31, 2020. DecisionDx-SCC is a 40-gene expression profile test that uses an individual patient’s tumor biology to predict individual risk of squamous cell carcinoma metastasis for patients with one or more risk factors. The test result, in which patients are stratified into a Class 1, 2A or 2B risk category, is designed to predict individual metastatic risk to inform risk-appropriate management.
11 peer-reviewed articles to support physician adoption and commercial payer reimbursement of Castle’s currently marketed tests were published in 2020.
Palmetto GBA MolDx, Noridian and associated Medicare Administrative Contractors (MAC) reviewed the DecisionDx-Melanoma data package as part of a reconsideration request in mid-2019 to review an expansion of coverage criteria for DecisionDx-Melanoma. LCD drafts were finalized in the second half of 2019 and effective in the fourth quarter of 2020. The Company believes that this data package demonstrating medical necessity also positively impacted reviews by commercial payers in that 288 insurance companies reviewed and approved claims for DecisionDx-Melanoma in 2020. Additionally, several regional health plans and a Blue Shield licensee have independently reviewed DecisionDx-Melanoma and have either issued or retired medical policies to support the medical necessity of DecisionDx-Melanoma.
Year-end 2020 cash and cash equivalents were approximately $410 million.
Castle Biosciences has not completed the preparation of its financial statements for the fourth quarter or full-year 2020. The preliminary, unaudited performance results presented in this press release for the quarter and year-ended December 31, 2020, are based on management’s initial review of the information presented and are subject to adjustment based on the completion of the Company’s end-of-period reporting processes and related activities, including the audit by our independent registered public accounting firm of the Company’s financial statements, as such, any financial information contained herein may differ materially from the information reflected in our financial statements as of and for the year-ended December 31, 2020. Additional information and disclosures would be required for a more complete understanding of the Company’s financial position and results of operations as of and for the quarter and year-ended December 31, 2020. Accordingly, undue reliance should not be placed on this preliminary information.