On March 30, 2021 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported financial results for the three-month period and full year ended December 31, 2020 (Press release, Spectrum Pharmaceuticals, MAR 30, 2021, View Source [SID1234577350]).

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"The Fast Track designation for poziotinib is a significant achievement towards an expedited review," said Joe Turgeon, President and CEO of Spectrum Pharmaceuticals. "In addition, we are delighted that the FDA has scheduled the pre-approval inspection at the ROLONTIS manufacturing facility for May 2021. The company has made tremendous progress advancing our development programs and conducting our clinical trials, despite the challenges of the global pandemic. I am proud of our employees who demonstrated resiliency and creativity during these unprecedented times."

Pipeline Updates

Poziotinib, an irreversible tyrosine kinase inhibitor targeting EGFR and HER2 mutations

Poziotinib received Fast Track designation from the FDA for the treatment of non-small cell lung cancer (NSCLC) in previously treated patients with HER2 exon 20 insertion mutations. Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious and life-threatening conditions and fill unmet medical needs.
Spectrum is preparing a new drug application (NDA) for poziotinib in the treatment of patients with previously treated locally advanced or metastatic NSCLC with HER2 exon 20 insertion mutations after a successful pre-NDA meeting with the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2020. Submission of the NDA based on the positive results of Cohort 2 from the ZENITH20 clinical trial is planned for later this year.
Preliminary safety and efficacy data for poziotinib from Cohort 5 of the ZENITH20 clinical trial demonstrated improved tolerability with BID dosing, reduced dose interruption compared to once daily (QD) dosing, and a reduction in treatment emergent Grade 3 or higher adverse events. The preliminary data also demonstrated improved anti-tumor activity with 8mg BID dosing. These results were presented at the European Society for Medical Oncology Targeted Anticancer Therapies (ESMO TAT) Virtual Congress 2021 in early March. Spectrum will be presenting further data on BID dosing at the upcoming AACR (Free AACR Whitepaper) Annual Meeting 2021.
ROLONTIS (eflapegrastim), a novel long-acting G-CSF

The FDA’s pre-approval inspection of the ROLONTIS manufacturing facility has been scheduled for May 2021. In October 2020, the FDA deferred its action on the Biologics License Application (BLA) for ROLONTIS due to an inability to inspect the drug substance manufacturing facility, citing travel restrictions related to the COVID-19 pandemic.
IGN002, interferon/CD20 monoclonal antibody fusion protein

Anti-CD20-IFNα, an antibody-interferon fusion molecule directed against CD20 has two sites open for enrollment in a Phase 1 study for treating relapsed or refractory non-Hodgkin’s lymphoma patients, including diffuse large B-cell lymphoma.
Three-Month Period Ended December 31, 2020 (All numbers are from Continuing Operations)

GAAP Results

Spectrum recorded net loss of $49.9 million, or $0.36 per basic and diluted share, in the three-month period ended December 31, 2020, compared to net loss of $40.2 million, or $0.36 per basic and diluted share, in the comparable period in 2019. Total research and development expenses were $47.2 million in the quarter, as compared to $23.3 million in the same period in 2019. Selling, general and administrative expenses were $15.7 million in the quarter, compared to $15.1 million in the same period in 2019.

Non-GAAP Results

Spectrum recorded non-GAAP net loss of $28.9 million, or $0.20 per basic and diluted share, in the three-month period ended December 31, 2020, compared to non-GAAP net loss of $33.4 million, or $0.30 per basic and diluted share, in the comparable period in 2019. Non-GAAP research and development expenses were $17.1 million, as compared to $22.4 million in the same period of 2019. Non-GAAP selling, general and administrative expenses were $12.3 million, as compared to $11.6 million in the same period in 2019.

Twelve-Month Period Ended December 31, 2020 (All numbers are from Continuing Operations)

GAAP Results

Spectrum recorded net loss of $171.3 million, or $1.38 per basic and diluted share, in the twelve-month period ended December 31, 2020, compared to net loss of $135.4 million, or $1.22 per basic and diluted share, in the comparable period in 2019. Total research and development expenses were $109.4 million for the year, as compared to $79.3 million in the same period in 2019. Selling, general and administrative expenses were $60.4 million for the year, compared to $61.4 million in the same period in 2019.

Non-GAAP Results

Spectrum recorded non-GAAP net loss of $120.9 million, or $0.97 per basic and diluted share, in the twelve-month period ended December 31, 2020, compared to non-GAAP net loss of $111.9 million, or $1.01 per basic and diluted share, in the comparable period in 2019. Non-GAAP research and development expenses were $75.6 million, as compared to $72.0 million in the same period of 2019. Non-GAAP selling, general and administrative expenses were $47.2 million, as compared to $45.5 million in the same period in 2019.

Cash Position and Guidance

Spectrum reported cash, cash equivalents, and marketable securities of approximately $180.0 million as of December 31, 2020, compared to $224 million at December 31, 2019.

This conference call will also be webcast. Listeners may access the webcast, which will be available on the investor relations page of Spectrum Pharmaceuticals’ website: View Source on March 30, 2021 at 4:30 p.m. Eastern/1:30 p.m. Pacific.

On March 30, 2021 Bicycle Therapeutics plc (NASDAQ: BCYC), a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, reported progress updates for its Bicycle-based partnered programs outside of oncology (Press release, Bicycle Therapeutics, MAR 30, 2021, View Source [SID1234577366]).

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"Over the last five years, Bicycle’s strategy has been to use our novel technology to discover and develop a pipeline of innovative assets in oncology while additionally using non-dilutive funding and collaborations to explore the therapeutic potential of Bicycles in disease areas outside of oncology. Today, for the first time, we are providing an overview on our progress in demonstrating the broad utility of this platform to create molecules with the potential to treat some of the most serious diseases and address future healthcare challenges," said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. "It’s incredibly exciting to see how the Company has worked innovatively and highly collaboratively with many diverse institutions to address these challenges and, at the same time, generated significant revenues to offset the costs of developing and progressing our internal oncology pipeline. I would like to thank all of our collaborators for their enthusiasm in this endeavor and look forward to continuing our work to advance these important molecules."

Bicycle has achieved the first milestone in its collaboration with Dementia Discovery Fund (DDF) and the University of Oxford’s ARUK Oxford Drug Discovery Institute (ODDI)

The Company identified and optimized nM affinity Bicycles to TREM2, a genetically validated target for the treatment of dementia, and nM affinity Bicycles to transferrin receptor 1 (TfR1), a molecular shuttle. In 2019, Bicycle established a collaboration with DDF, later expanded to include ODDI, to use Bicycle technology for the discovery and development of potential novel therapeutics for the treatment of dementia. The three parties are collaborating to identify and characterize Bicycles that bind to and activate TREM2, a genetically validated dementia target. As part of the collaboration, Bicycle is also using its technology to identify binders to TfR1, a blood brain barrier transporter, that could potentially enable delivery of Bicycles, or any other molecular payload, into the central nervous system (CNS), or other organ systems, expressing TfR1. Under the terms of the collaboration with DDF, Bicycle retains all rights to the TfR1 molecules outside of the license granted for use with targets defined within the DDF collaboration.
Bicycle advances platform in multiple anti-infective areas, including antimicrobials and antivirals

Innovate UK’s Biomedical Catalyst (BMC) awarded the Company funding to advance a Bicycle inhibitor for a key cell wall biosynthesis target in Enterobacterales, Penicillin Binding Protein 3 (PBP3). Bicycle is using its proprietary platform to try to address the significant healthcare challenge of antimicrobial resistance and has identified several potential Bicycle PBP3 inhibitors. Through grants awarded by the UK government and in collaboration with investigators at the University of Warwick, Bicycle intends to progress these PBP3 inhibitors, potentially representing the first novel class of antibiotics identified in decades, to candidate and initial toxicology testing.
Innovate UK, under a specific program targeting key technologies to rapidly respond to the challenge of COVID-19 (UKRI Ideas to Address COVID-19 – Innovate UK Article 25), provided funding to support Bicycle’s efforts to discover new healthcare solutions to the SARS-CoV-2 pandemic. Bicycle has identified numerous discrete families of Bicycles which bind to at least 10 different epitopes on the SARS-CoV-2 spike protein, binders to the Nucleocapsid protein and the viral attachment site on the host ACE2 human receptor. Through Bicycle’s partnership with researchers at the MRC Laboratory of Molecular Biology, Bicycles identified by these screens have demonstrated nanomolar activity in inhibiting SARS-CoV-2 infection of human lung cells. These monomeric Bicycles have been conjugated together to make a diverse range of bi-paratopic and multi-valent small molecules (less than 10kD), some of which are active in the picomolar range in viral entry assays and may be resistant to the current SARS-CoV-2 variants of concern. Bicycle continues to advance these novel compounds in pre-clinical evaluation. Bicycle has also formed partnerships with diagnostic experts to evaluate its novel SARS-CoV-2 binding Bicycles in Lateral Flow Tests and other diagnostic screening formats.
Bicycle has made significant progress through partnerships with biopharmaceutical therapeutic area leaders in indications outside of oncology

The Company has successfully discovered and advanced targets outside of oncology through the ongoing collaboration with AstraZeneca, a global biopharmaceutical company, to discover novel agents for the treatment of respiratory and cardiometabolic diseases. Two assets have been transitioned to AstraZeneca for further development and are currently being progressed in their discovery pipeline. Two additional assets achieved transition criteria and were transitioned to AstraZeneca but were returned to Bicycle by AstraZeneca.
Bicycle has also identified targets in its collaboration with Bioverativ (acquired by Sanofi in 2018) for the treatment of rare hematological diseases. The Bioverativ collaboration successfully identified nM multi-valent inhibitors to P-Selectin, which inhibited human neutrophil binding and rolling, with potential applications in sickle cell disease and other inflammatory diseases. The Bioverativ collaboration also identified the first small molecule Factor VIII mimetic for the potential treatment of Hemophilia A. This mimetic is comprised of a multivalent Bicycle binding to both Factor IX and Factor X, leading to the production of activated Factor X (FXa), which successfully activated thrombin in plasma from Hemophilia A patients ex vivo. In 2019, Sanofi elected not to pursue further development, and the collaboration agreement was terminated. These "lead stage" assets have now been returned to Bicycle.
Kevin Lee commented, "With four Bicycle molecules now in clinical trials for both oncology and non-oncology indications, the technology has shown initial evidence of clinical tolerability and suitability for pharmaceutical development, and I look forward to seeing how Bicycles may bring important new treatment opportunities to poorly served patients in additional therapeutic areas."

On March 30, 2021 Novartis reported that it has obtained exclusive worldwide rights to develop and commercialize therapeutic applications for a library of Fibroblast Activation Protein (FAP) targeting agents including FAPI-46 and FAPI-74, through an assignment agreement with iTheranostics, Inc., an affiliate of SOFIE Biosciences, Inc (Press release, Novartis, MAR 30, 2021, View Source [SID1234577323]). The FAP assets were originally developed at the University of Heidelberg. The agreement also includes co-exclusive rights for Novartis to develop imaging applications for these assets.

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Fibroblast activation protein (FAP) is a cell-surface protein expressed at low levels in most normal adult tissues, but over-expressed in common cancers, particularly on cancer-associated fibroblasts that form the tumor stroma, which is essential for growth1,2,3,4. High FAP expression on cancer-associated fibroblasts is generally associated with worse prognosis in solid tumors due to promotion of tumorigenesis and progression4,5,6,7.

"We continue to invest in radioligand therapy as one of the four unique platforms of Novartis Oncology. We believe working across multiple approaches is the key to reimagining cancer care," said Susanne Schaffert, PhD, President, Novartis Oncology. "FAP is an exciting target and these agents are a great fit with our radioligand therapy pipeline, which we are actively investigating across multiple tumor types. We believe this technology has the potential to transform many patients’ lives."

Targeted radioligand therapy is a type of precision medicine combining two key elements: a targeting compound, or ligand, and a radioactive isotope, causing DNA damage that inhibits tumor growth and replication. These targeted drugs bind to markers or proteins over-expressed by certain tumors, or tumor-associated tissue, such as stroma. Due to the high-affinity of these agents for specific tumor cells or associated tumor tissue, surrounding healthy tissue is less affected.

On March 30, 2021 Amgen Inc. (NASDAQ:AMGN) and Rodeo Therapeutics Corporation (Rodeo) reported an agreement under which Amgen will acquire Rodeo, a privately held biopharmaceutical company based in Seattle that develops small-molecule therapies designed to promote regeneration and repair of multiple tissues (Press release, Amgen, MAR 30, 2021, View Source [SID1234577351]). Rodeo’s 15-PGDH program is a strong strategic fit with Amgen’s inflammation portfolio and efforts to develop first-in-class therapeutics for patients.

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Under terms of the agreement, Amgen will acquire all outstanding shares of Rodeo in exchange for a $55 million upfront payment as well as future contingent milestone payments potentially worth up to an additional $666 million in cash. The transaction has been approved by the shareholders and the Board of Directors of Rodeo.

Rodeo is focused on developing first-in-class, orally available modulators of prostaglandin biology that play an important role in tissue regeneration and repair. Rodeo’s lead 15-prostaglandin dehydrogenase (15-PGDH) modulators have generated compelling data in extensive preclinical studies and have clinical potential in multiple indications.

"The enzyme 15-PGDH plays a key role in many disease-relevant processes such as stem cell self-renewal and epithelial barrier repair. Given the encouraging preclinical data to date, we are excited about the opportunity to develop a novel therapy with potential in a range of important inflammatory disease indications," said Raymond Deshaies, Ph.D., senior vice president of Global Research at Amgen.

Thong Q. Le, president and chief executive officer of Rodeo commented, "We are thrilled that Amgen recognizes the potential value and differentiated profile of our 15-PGDH inhibitor program. With decades of experience in developing, manufacturing and commercializing innovative therapies for patients suffering from a broad range of immunologic diseases and conditions, Amgen is ideally positioned to rapidly advance our program into the clinic."

Cooley LLP acted as legal advisor to Rodeo and Gunderson Dettmer LLP acted as legal advisor to Amgen on this transaction.

On March 30, 2021 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to create product candidates that address unmet medical needs, reported that the company will participate at the J.P. Morgan 10th Annual Napa Valley Biotech Forum (Press release, Ascendis Pharma, MAR 30, 2021, View Source [SID1234577367]). Company executives will provide a business overview and an update on the Company’s pipeline programs.

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Details

Event J.P. Morgan 10th Annual Napa Valley Biotech Forum
Location Virtual
Date Tuesday, March 30, 2021
Time 4:00 p.m. Eastern Time
A live audio webcast of the presentation will be available on the Investors and News section of the Company’s website at www.ascendispharma.com. A webcast replay will also be available on the Company’s website shortly after conclusion of the event for 30 days.