On February 27, 2020 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported financial results for the three-month period and year ended December 31, 2019 (Press release, Spectrum Pharmaceuticals, FEB 27, 2020, View Source [SID1234554900]).

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"ROLONTIS is in active review by the FDA and we are preparing to launch shortly following approval," said Joe Turgeon, President and CEO, Spectrum Pharmaceuticals. "We believe this market represents a significant commercial opportunity and our prelaunch activities are well underway. We have a podium presentation on poziotinib in a few short weeks, we have taken steps to adjust our strategy and we have multiple data catalysts in 2020. I look forward to updating you on our progress throughout the year."

Pipeline Updates

ROLONTIS (eflapegrastim), a novel long-acting G-CSF

FDA is actively reviewing the BLA for ROLONTIS for the treatment of chemotherapy-induced neutropenia. The PDUFA target action date for the ROLONTIS BLA has been set for October 24, 2020.
In October 2019, Spectrum presented a poster at the ASCO (Free ASCO Whitepaper) Supportive Care in Oncology Symposium in San Francisco. The poster highlighted integrated efficacy data from the Phase 3 ROLONTIS clinical trials, ADVANCE and RECOVER, which studied more than 600 patients combined and met all primary and secondary endpoints.
An abstract reporting preclinical data on eflapegrastim or pegfilgrastim using "same day dosing" as chemotherapy has been accepted for presentation at the AACR (Free AACR Whitepaper) meeting April 24-29 in San Diego.
Poziotinib, an irreversible tyrosine kinase inhibitor targeting EGFR and HER2 mutations

The pre-specified primary endpoint in a Phase 2 clinical trial (ZENITH20) evaluating poziotinib in previously treated non-small cell lung cancer (NSCLC) patients with EGFR exon 20 insertion mutations was not achieved in Cohort 1.
Data from Cohort 1 will be presented in a podium presentation at the 11th Annual Congress on Pulmonary and Respiratory Medicine in Amsterdam on March 18th.
Cohort 2 of the ZENITH20 trial enrolling previously treated HER2 non-small cell lung cancer patients is fully accrued and is expected to have topline results by mid-year 2020. Cohort 3 of the ZENITH20 trial enrolling first-line EGFR NSCLC patients is expected to have topline results by year-end 2020. Either cohort has the potential to support a future NDA submission.
Three-Month Period Ended December 31, 2019 (All numbers are from Continuing Operations)

GAAP Results

Spectrum recorded net loss of $40.2 million, or $0.36 per basic and diluted share, in the three-month period ended December 31, 2019, compared to net loss of $53.1 million, or $0.50 per basic and diluted share, in the comparable period in 2018. Total research and development expenses were $23.3 million in the quarter, as compared to $29.9 million in the same period in 2018. Selling, general and administrative expenses were $15.1 million in the quarter, compared to $16.6 million in the same period in 2018.

Non-GAAP Results

Spectrum recorded non-GAAP net loss of $33.4 million, or $0.30 per basic and diluted share, in the three-month period ended December 31, 2019, compared to non-GAAP net loss of $43.4 million, or $0.41 per basic and diluted share, in the comparable period in 2018. Non-GAAP research and development expenses were $22.4 million, as compared to $29.2 million in the same period of 2018. Non-GAAP selling, general and administrative expenses were $11.6 million, as compared to $14.0 million in the same period in 2018.

Twelve-Month Period Ended December 31, 2019 (All numbers are from Continuing Operations)

GAAP Results

Spectrum recorded net loss of $135.4 million, or $1.22 per basic and diluted share, in the twelve-month period ended December 31, 2019, compared to net loss of $126.7 million, or $1.23 per basic and diluted share, in the comparable period in 2018. Total research and development expenses were $79.3 million for the year, as compared to $75.2 million in the same period in 2018. Selling, general and administrative expenses were $61.4 million for the year, compared to $62.7 million in the same period in 2018.

Non-GAAP Results

Spectrum recorded non-GAAP net loss of $111.9 million, or $1.01 per basic and diluted share, in the twelve-month period ended December 31, 2019, compared to non-GAAP net loss of $124.3 million, or $1.20 per basic and diluted share, in the comparable period in 2018. Non-GAAP research and development expenses were $72.0 million, as compared to $72.1 million in the same period of 2018. Non-GAAP selling, general and administrative expenses were $45.5 million, as compared to $53.2 million in the same period in 2018.

Cash Position and Guidance

Spectrum reported cash, cash equivalents, restricted cash, and marketable securities of approximately $224 million as of December 31, 2019, compared to $204 million at December 31, 2018. Based on current operating plans, Spectrum expects that SG&A expense will increase in the second half of this year as we scale up our commercial operations in preparation to launch ROLONTIS.

Conference Call

Thursday, February 27, 2020 @ 4:30 p.m. Eastern/1:30 p.m. Pacific

Domestic:

(877) 837-3910, Conference ID# 4475135

International:

(973) 796-5077, Conference ID# 4475135

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 February 27, 2020 at 4:30 p.m. Eastern/1:30 p.m. Pacific.

On February 27, 2020 Harbour BioMed (HBM) reported U.S. Food and Drug Administration (FDA) approval of its Investigational New Drug (IND) application for a Phase 2 clinical trial of HBM9167, its humanized IgG1 monoclonal antibody targeting programmed death-ligand 1 (PD-L1), for the treatment of nasopharyngeal cancer (NPC) (Press release, Harbour BioMed, FEB 27, 2020, View Source [SID1234554899]). Separately, the Agency’s Office of Orphan Products Development granted HBM9167 Orphan Drug Designation (ODD) for the use in treating NPC.

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"Nasopharyngeal cancer is a tumor type for which patients remain in need of effective treatment options," said Dr. Jingsong Wang, Founder, Chairman and CEO of Harbour BioMed. "The Orphan Drug Designation for HBM9167 will provide an accelerated development path for HBM9167. In addition, we were also able to work with the FDA to enable a direct entry into a Phase 2 clinical study in patients with metastatic or recurrent disease."

HBM9167 has been evaluated by HBM’s partner Kelun-Biotech in China in studies involving more than 300 subjects to date. These trials include Phase 1 and ongoing Phase 2 trials in multiple cancer types, including NPC and classical Hodgkin’s Lymphoma. Preliminary results from these ongoing trials have shown a tolerable safety profile in patients.

The FDA grants ODD status to drugs intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. ODD qualifies a drug sponsor for various development incentives under the Orphan Drug Act, such as eligibility for a seven-year period of market exclusivity following regulatory approval, clinical trial assistance and a 50 percent tax credit on the cost of clinical trials conducted in the U.S.

About HBM9167
HBM9167 is a humanized IgG1 monoclonal antibody (mAb) directed against human programmed death-ligand 1 (PD-L1). Through binding with PD-L1, HBM9167 blocks its interaction with programmed cell death protein 1 (PD-1), thereby releasing the PD-L1/PD-1 mediated inhibition on immune response, and subsequently restoring the cytotoxic immune function of T-cells to enhance anti-tumor immunity. Monoclonal antibodies that target PD-1 or PD-L1 can inhibit their binding activity, maintain the function of effector T-cells to prevent cancer cells from escaping the immune surveillance, and thus maintain the body’s immune ability against cancer cells. Harbour BioMed in-licensed HBM9167 from Kelun-Biotech with the right to develop and commercialize it worldwide, excluding Greater China.

On February 27, 2020 Sorrento Therapeutics, Inc. (Nasdaq: SRNE, "Sorrento") reported that an interim analysis of an ongoing Phase 1b study of resiniferatoxin (RTX) administered via epidural route in patients with intractable cancer pain has generated positive data (Press release, Sorrento Therapeutics, FEB 27, 2020, View Source [SID1234554898]). The study is completing enrollment of an additional 25 ug expansion dose cohort. Based on full study results a dose will be selected for RTX to proceed to Phase 3 pivotal trials in patients with advanced cancer pain.

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Safety in the higher dose cohort is expected to be supportive for testing RTX epidural administration in other advanced non-cancer disease related unmet clinical needs (with a choice of starting dose at 15 or 25 ug depending on the indication considered).

Results as planned in the original study (to 15 ug) are being presented at the American Academy of Pain Medicine Annual Meeting on February 27, 2020.

The Phase 1b open-label study was used for dose escalation to assess the safety and preliminary efficacy of a single epidural administration of resiniferatoxin for the treatment of intractable pain due to cancer. Initial expectations for safety and efficacy have been met for the Phase 1b study, though optimal dose selection will follow assessment of the additional dose group in which 25 ug is also being evaluated. Thus far, after Institutional Review Board approval was obtained, and with oversight by an independent data monitoring committee, data is available from 14 subjects with intractable cancer-related pain who received a single epidural injection of RTX from 0.4 to 15 ug.

Safety Outcomes

No dose limiting toxicities or notable adverse events unrelated to progression of underlying disease were encountered for any of the subjects. The most common treatment-related adverse event was transient post-procedural pain: 7 of 14 subjects (50%) reported moderate severity. Two additional treatment-related adverse events of moderate severity were back pain and increase in blood pressure in a patient. All events resolved in less than two days following drug administration.

Efficacy Outcomes

The ongoing trial will follow subjects for at least 84 days. The lower doses of 0.4, 1.0, 2.0, 4.0 ug did not demonstrate notable pain relief, but permitted dose escalation to the next designated dose level based on how well the drug was tolerated.

Three patients had marked pain relief starting shortly after initial administration that were still observed weeks after treatment: 1 of 3 subjects who received 8 ug (a 58-year-old woman with gastrointestinal stromal cancer with severe lower back pain reported a decrease in numerical pain rating scale (NPRS) scores from >6/10 to 2/10), and 2 of 3 subjects who received 15 ug (a 62-year-old man with rectal cancer noted significant improvement in pain, physical strength, mood, and appetite with NPRS scores reduced from 7-8/10 to 3/10; and a 57-year-old man with multiple myeloma and severe pain in his back, hips, and lower extremities subsequently reported mild pain in the target areas after RTX injection). Improvement in pain and mobility within 24 hours of dosing in the three responders at the higher dose levels of RTX suggests the clinical potential of the drug for intractable cancer pain.

Sorrento intends to present the detailed results of the completed study upon completion of the additional expansion cohort later this year. A pivotal Phase 3 trial is being planned using epidural RTX for severe pain states associated with advanced disease.

"We are encouraged by the benefits of using RTX to treat intractable pain in patients with metastatic cancer," said Associate Professor of Anesthesia, Srdjan S. Nedeljkovic, M.D. from the Department of Anesthesiology, Perioperative and Pain Medicine, Brigham and Women’s Hospital at Harvard Medical School. "Even in patients with high levels of pain, RTX given via an epidural injection has been found to reduce pain intensity without having any long-term adverse safety consequences. The addition of RTX to existing cancer pain therapies represents a positive step forward in improving the care of patients with intractable cancer pain and offers the hope of enhancing the overall quality of life experienced by this population."

For access to the scientific presentation (AAPM poster) please visit
View Source

About Resiniferatoxin (RTX)

A thousand times "hotter" than pure capsaicin (16 Billion Scoville units versus 16M), and with a high affinity for afferent pain nerves, resiniferatoxin binds to TRPV1 receptors and selectively ablates the nerve endings responsible for pain signals experienced by patients1. Delivered peripherally (into the joint space) the transient nerve ending ablation effect can have profound clinical benefits lasting for months to years (as shown in canine studies2).

RTX-001 is a multicenter, open-label dose escalation Phase 1b study to assess the safety and define the maximally tolerated dose of resiniferatoxin administered via the epidural route for the reduction of moderate to severe pain signal intensity associated with advanced cancer. The Phase 1b study is a dose-escalation protocol in which cohorts of patients receive increasing doses of resiniferatoxin until the maximum tolerated dose is achieved. The primary objective of the study is to evaluate the safety of resiniferatoxin and identify the recommended Phase 3 dose. The secondary objective is to assess the preliminary efficacy of resiniferatoxin measured by assessing changes in the intensity of pain using the NPRS score, a widely used proprietary validated pain scale.

RTX is not approved for clinical use by regulatory authorities. Safety and efficacy have not been established.

On February 27, 2020 Boehringer Ingelheim and Trutino Biosciences reported they have entered into a research collaboration and worldwide licensing agreement based on Trutino’s innovative On-Demand-Cytokine (ODC) platform (Press release, Boehringer Ingelheim, FEB 27, 2020, View Source [SID1234554896]). Under the terms of strategic alliance, Boehringer Ingelheim gains access to Trutino’s ODC platform technology for the generation and development of up to three new ODC candidates. This new collaboration combines Boehringer Ingelheim’s long-term strategy to provide first-in-class, breakthrough therapies for cancer patients with Trutino’s unique knowledge and expertise in increasing the safety and efficacy of cytokine therapies.

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"Developing a strong and innovative cytokine therapeutic program as an additional component of our cancer immunology portfolio, demonstrates how we are ‘Taking Cancer On,’ and provides a high potential combination partner for our existing cancer vaccine, oncolytic virus, T cell engager and myeloid-targeting therapeutics portfolio," said Jonathon Sedgwick, Ph.D., Senior Vice President and Global Head, Cancer Immunology & Immune Modulation Research at Boehringer Ingelheim. "We are very pleased to partner with Trutino and harness the potential of their innovative scientific platform to develop treatment breakthroughs that will transform the lives of cancer patients."

Trutino’s ODC platform masks the activity of cytokines until they reach the tumor site and become fully activated, sparing systemic exposure and potentially leading to a higher margin of safety and greater efficacy than conventional cytokine treatments. Trutino will generate the new ODC molecules and carry out preclinical validation, handing over development to Boehringer Ingelheim for late pre-clinical testing through the rest of development.

"We are excited to embark on this partnership with Boehringer Ingelheim, a leader in cancer immunology, to advance cytokine therapeutic options that address the unmet medical needs of patients worldwide," said Phillip Kim, Ph.D., MBA, Founder and CEO of Trutino Biosciences. "Boehringer Ingelheim has a deep commitment to innovative scientific approaches and is a leader in bringing novel cancer therapies to market. This global partnership validates the broad potential of our proprietary ODC platform to create safer and more effective cytokine therapies that can be delivered systemically and activated locally. Together, we can rapidly develop a new generation of cytokine therapies to address critical unmet need in oncology."

Trutino’s clinical potential was initially recognized by Boehringer Ingelheim through its grass roots programs, including the ‘BI Innovation Prize,’ where the ODC platform technology was an early-stage standout in the 2019 program held in San Diego. Launched in 2015, the grass roots programs comprise of ‘BI Office Hours,’ ‘BI Academy’ and the ‘BI Innovation Prize.’ Through Office Hours, Boehringer Ingelheim has provided over 200 early-stage companies in the life-sciences community with mentoring and direct access to relevant expertise and industry perspective from senior leaders within the company. In partnership with BioLabs in Boston, New York and San Diego, Boehringer Ingelheim has awarded numerous entrepreneurs with free lab space through the ‘Golden Ticket’ program. As a company dedicated to improving health and quality of life, these programs give Boehringer Ingelheim the opportunity to lend expertise to the innovation community and offer guidance around the science to help enable ideas to deliver the next breakthroughs.

The partnership with Trutino will strengthen Boehringer Ingelheim’s next generation immune oncology portfolio, which combines cancer vaccines, oncolytic viruses, T Cell engagers and myeloid targeting platforms with the aim of making "cold" tumors that are invisible to the immune system "hot" to rally the immune system against the tumor. Under the terms of the agreement, Boehringer Ingelheim will provide an upfront payment, near-term pre-clinical milestone payments and clinical, regulatory and commercial milestone payments, including royalties on future product sales.

On February 26, 2020 Pfenex Inc. (NYSE American: PFNX) reported that it will report its financial results for the fourth quarter and year ended December 31, 2019, after the market close on Wednesday, March 11, 2020 (Press release, Pfenex, FEB 26, 2020, View Source [SID1234554895]). Pfenex will host a conference call and webcast to discuss its financial results and provide a company update that day at 1:30 PM Pacific Time (4:30 PM Eastern Time).

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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

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Conference Call & Webcast
Wednesday, March 11th @ 1:30 PM Pacific Time (4:30 PM Eastern Time)
Domestic: 866-376-8058
International: 412-542-4131
Webcast: View Source

Replays available through March 18th:
Domestic: 877-344-7529
International: 412-317-0088
Replay Access Code: 10139830