On May 13, 2021 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported that financial results for the three-month period ended March 31, 2021 and provided a corporate update (Press release, Spectrum Pharmaceuticals, MAY 13, 2021, View Source [SID1234579909]).

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"Our top priority is submission of the NDA to the FDA for poziotinib based on the positive data from Cohort 2 of the ZENITH20 clinical trial," said Joe Turgeon, President and CEO of Spectrum Pharmaceuticals. "The additional data on twice daily dosing presented at AACR (Free AACR Whitepaper) has the potential to significantly expand the value of poziotinib. We also look forward to the FDA’s pre-approval inspection of the ROLONTIS manufacturing facility which has been scheduled for later this month."

Pipeline Updates

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

Spectrum is actively working on its new drug application (NDA) for the use of poziotinib in patients with previously treated locally advanced or metastatic NSCLC with HER2 exon 20 insertion mutations. Submission of the NDA, based on the positive results of Cohort 2 from the ZENITH20 clinical trial, is planned for later this year.
New data was presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting 2021 from ZENITH20 Cohort 5 which demonstrated improved efficacy and tolerability for twice daily (BID) dosing. In the 38 patients, with EGFR or HER2 exon 20 insertion mutations, who received 16mg per day and randomized either to poziotinib 16mg once daily (QD) or 8mg BID in Cohort 5, improved responses were observed in the BID arm with 31.6% of patients reaching a partial response, and Grade 3 or higher related adverse events were reduced by approximately 60%. Additionally, the BID dosing allowed for an improved rate of dose reductions and interruptions relative to the QD dose.
Faster enrollment is expected in Cohort 5 which is now dosing exclusively at 8mg BID.
Enrollment of first-line patients with NSCLC HER2 exon 20 mutations is continuing for Cohort 4 of the ZENITH20 clinical trial. Poziotinib is being administered at a dose of 8mg BID in first line treatment.
A poster titled "CNS activity of poziotinib in NSCLC with exon 20 insertion mutations" based on data from Cohorts 1-3 has been accepted for presentation at ASCO (Free ASCO Whitepaper).
ROLONTIS (eflapegrastim), a novel long-acting G-CSF

The FDA’s pre-approval inspection of the ROLONTIS manufacturing facility has been scheduled for later this month and pre-commercial preparation activities are underway.
Three-Month Period Ended March 31, 2021 (All numbers are from Continuing Operations and are approximate)

GAAP Results

Spectrum recorded a net loss of $35.7 million, or $0.25 loss per basic and diluted share, in the three-month period ended March 31, 2021, compared to a net loss of $40.6 million, or $0.36 loss per basic and diluted share, in the comparable period in 2020. Total research and development expenses were $19.4 million in the quarter, as compared to $16.0 million in the same period in 2020. Selling, general and administrative expenses were $14.3 million in the quarter, compared to $14.8 million in the same period in 2020.

The company ended the quarter with cash, cash equivalents, and marketable securities of $162.9 million.

Non-GAAP Results

Spectrum recorded a non-GAAP net loss of $29.4 million, or $0.20 loss per basic and diluted share, in the three-month period ended March 31, 2021, compared to a non-GAAP net loss of $25.0 million, or $0.22 loss per basic and diluted share, in the comparable period in 2020. Non-GAAP research and development expenses were $18.0 million, as compared to $14.6 million in the same period of 2020. Non-GAAP selling, general and administrative expenses were $11.5 million, as compared to $10.8 million in the same period in 2020.

Conference Call

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 May 13, 2021 at 4:30 p.m. Eastern/1:30 p.m. Pacific.

On May 13, 2021 EpiVax Therapeutics, Inc. ("EVT") reported the publication "Multi‑step screening of neoantigens’ HLA and TCR‑interfaces improves prediction of survival" in Scientific Reports (Press release, EpiVax, MAY 13, 2021, View Source [SID1234579907]). This study demonstrates a superior method of survival analysis for cancer patients using EVT’s Ancer platform. EVT spun-off from EpiVax, Inc. in 2017 to apply EpiVax tools to clinical research.

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Built on validated in silico tools EpiMatrix and JanusMatrix, Ancer analyzes individual cancer mutanomes to define the presence of T cell epitopes likely to be immunogenic. The concept behind Ancer is that the presence of a mutation alone is not sufficient to generate a protective immune response, but that the differences present between the natural (human genome) sequence and the mutated sequence at the face of the epitope that is identifiable to T cells (the TCR-face) should be accounted for when creating precision cancer vaccines and predicting survival outcomes for cancer patients. This is because some mutations create self-like epitopes and can be tolerated by the immune system.

Researchers compared three approaches to analyzing cancer mutanomes and evaluated their ability to predict patient outcomes. Ancer, the best performing method, improved the differentiation of patients that did poorly from those who did well, accurately predicting survival outcome nearly 6 years longer as compared to other approaches, which only predicted as far as 3 years. This study has important consequences in our ability to identify patients with a high risk of tumor progression and early mortality based on their tumor genome.

On study implications, Dr. Randy Sweis of the University of Chicago stated, "These results suggest that defining the number of true neoepitopes using Ancer may represent a novel prognostic or predictive biomarker for cancer patients." Dr. Annie De Groot, EVT co-founder, suggested "In addition to biomarker identification, using Ancer novel precision vaccines for individual cancer patients offers major advantages compared to other approaches, since Ancer prioritizes truly immunogenic CD8 and CD4 neoepitopes, while removing self-like or inhibitory neoepitopes."

On May 13, 2021 Certara, Inc. (Nasdaq: CERT), the global leader in biosimulation, reported the launch of new versions of its Immunogenicity (IG) and Immuno-oncology (IO) Quantitative Systems Pharmacology (QSP) Simulators to help address the challenges in the development of biologics and cancer Therapies (Press release, Certara, MAY 13, 2021, View Source [SID1234579906]). These new versions further expand applications of QSP and advance the field of using virtual patients to conduct computer-based trials throughout drug discovery and development.

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"We have seen an increasing interest in the potential of QSP to guide regulatory decisions and anticipate that this will only continue to grow, much like physiologically-based pharmacokinetic (PBPK) modeling, which is now a critical component in many regulatory submissions," said William F. Feehery, Ph.D., CEO of Certara.

QSP addresses the most complex challenges in drug development by combining computational modeling and experimental data to examine the relationships between a drug, biological system, and disease process. Immunogenicity is a key challenge for developing biologics, including novel modalities such as gene and cell therapies. Researchers use Certara’s IG Simulator to simulate immunogenicity in virtual patients. Version 4.0 of the IG Simulator now allows for the extrapolation of ex vivo patient assays in addition to in vitro assays to better inform the model and predict clinical outcomes. The IG Simulator has now been used in more than 20 case examples. Certara and its customers will be presenting several of these case examples at an upcoming FDA workshop in June.

In immuno-oncology, the sheer number of possible therapy combinations requires a quantitative framework to integrate the complex and dynamic factors that determine efficacy and historically have led to the selection of suboptimal combinations. Certara’s IO Simulator uses virtual patients to quickly test these different combinations to determine the optimal combination of therapies and dosing regimens. Version 3.0 of the IO Simulator vastly expands the number of targets and cell types including cytokines, immune cell types, and tumor neoantigens. It can also now test combinations of chemotherapy and radiotherapy. The IO Simulator has correctly predicted therapeutic outcomes of using drugs in various cancer types, including solid tumors and blood cancers.

"QSP is demonstrating its capacity to improve biopharmaceutical R&D, improving confidence in both the drug compound and target to increase the likelihood of success," said Piet van der Graaf, Ph.D., Senior Vice President, QSP at Certara. "The IG Simulator can use first-in-human data to design Phase II/III trials, predict impact of disease and co-medication, extrapolate to new populations, and predict if IG can be managed via dosing regimens. In tandem with the rise of IO therapeutics, we’ve expanded the number of drug targets and cell types in our IO Simulator enabling us to better predict optimal drug combinations and dosing regimens for more patient populations."

In recent years, the emergence of QSP has attracted increasing interest and is now becoming an essential part in model-informed drug discovery and development. The U.S. Food and Drug Administration (FDA) has seen an increase in evaluating QSP approaches in regulatory submissions.

On May 13, 2021 Southern Research reported that Two prominent Birmingham leaders have been named to leadership – the scientific discovery and research institution headquartered on Birmingham’s Southside (Press release, Southern Research Institute, MAY 13, 2021, View Source [SID1234579905]).

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Josh Carpenter has been named president and CEO, and Allen Bolton has been named executive vice president for strategy and finance. They begin the new roles on June 1.

They’ll be taking the reins at an organization with vast potential for shaping the future of Birmingham’s economy and its innovation ecosystem.

Carpenter, a Rhodes Scholar, most recently served as director of innovation and economic opportunity for the city of Birmingham. He previously worked at UAB.

Bolton most recently was senior vice president for finance and administration at UAB where he also served on the board of Southern Research.

"I couldn’t be more enthusiastic about what’s in front of us because of the people who are there," Carpenter said.

In addition to the expertise of the people and the legacy of Southern Research, Carpenter said he was attracted to the role because of the organization’s potential to transform the region’s economy.

Historically, he said Birmingham has been home to many islands of innovation. One of his goals is to create a better through-line to connect those pockets of R&D excellence in the community and raise their collective impact.

"Southern Research, perhaps better than any other organization, is specifically geared toward playing the role of that translational bridge – translating ideas to innovation and economic opportunity," Carpenter said. "If we can create the storefront to access those immense capabilities, we can present ourselves as the next frontier market in biotech."

Carpenter said Birmingham is well-positioned for growth in life sciences, precision medicine and related fields, and he said Southern Research, with expertise in those fields and others, can play a critical role in that innovation economy.

With the way the medical sector is trending, Carpenter sees a growing need for the type of capability expertise that can be found on the campus of Southern Research, which has 400 employees.

He’s also excited about the role the organization can play in building the local innovation ecosystem – an effort with vast implications for Birmingham’s economy.

There have been numerous examples of successful biotech companies that have originated from research conducted in Birmingham but ultimately moved elsewhere. One of his goals at Southern Research is to help build an environment that keeps those companies – and the jobs they create – in metro Birmingham.

"Southern Research is at it’s finest when the halls are brimming with first-rate intellect that come up with a novel discovery that becomes a commercial enterprise and creates jobs right here in Birmingham," he said.

Carpenter said there will be a focus on having a deeper and more profound relationship with UAB, which is located adjacent to Southern Research’s campus.

"We have not tapped the potential of working to integrate our approach," he said.

Carpenter also said a priority will be adapting Southern Research’s business model to capitalize on the opportunities at hand. What that will entail is likely to become more clear with a strategic plan that could be released later this year to coincide with Southern Research’s 80th anniversary.

He said he’s happy to have a veteran leader like Bolton joining him at Southern Research.

"No one has better experience navigating not just the UAB ecosystem, but a labyrinth of medical systems," Carpenter said. "He’s going to be a really effective arbiter of our strategic and financial interests."

On May 13, 2021 Helix BioPharma Corp. (TSX:HBP) ("Helix" or the "Company"), an immuno-oncology company developing innovative drug candidates for the prevention and treatment of cancer, reported that it has closed the first tranche of the previously announced private placement pursuant to the convertible security funding agreement (the "Agreement") with Lind Global Macro Fund, LP, a New York based institutional investment fund managed by The Lind Partners, LLC (together, "Lind") for gross proceeds of CAD$3.5 million (the "First Tranche") (Press release, Helix BioPharma, MAY 13, 2021, View Source [SID1234579904]).

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Pursuant to the First Tranche, the Company has issued a convertible security (the "Convertible Security") with a twoyear term and a face value of $4,112,500 and an aggregate of 1,957,056 common share purchase warrants exercisable into common shares in the capital of the Company ("Common Shares") for a period of 48 months at an exercise price of CAD$1.0283 per Common Share. The Convertible Security accrues a simple interest rate obligation of 8.75% per annum on the amount funded, which interest is prepaid and attributed to the face value of the Convertible Security. The Company has paid Lind a 3% commitment fee on the amount funded under the First Tranche and Alpha Bronze LLC is entitled to a finder’s fee equal to 5% of the amount funded.

As previously disclosed, Lind is entitled to convert the Convertible Security into Common Shares over the two year term, subject to certain limitations, at a conversion price equal to 85% of the five-day trailing volume-weighted average price of the Common Shares prior to the date a notice of conversion is provided to the Company by Lind. The Agreement includes certain restrictions on the maximum face value of each of the Convertible Securities that may be converted in any particular month. In addition, Helix has the option to buy-back 66.7% of the Convertible Security in cash at any time with no penalty, subject to the option of Lind to convert up to 1/3 of the face value of the Convertible Security into Common Shares at the time of such buy-back. If the Convertible Security is repaid by the Company within 180 days of issuance, the face value amount owed will be reduced pursuant to the terms of the Agreement. Lind is also entitled to accelerate its conversion right to the full amount of the face value or demand repayment of the face value in cash upon a default and other designated events as set out in the Agreement. To the extent that the full face value of the Convertible Security has not been converted at the maturity date of the Convertible Security, the outstanding balance of such face value shall be to be repaid to Lind by the Company in cash.

The securities issued in connection with the First Tranche are subject to a hold period pursuant to Canadian securities laws, expiring on September 14, 2021.

The Agreement also contemplates the issuance of a second Convertible Security upon the mutual agreement of the Company and Lind for gross proceeds to the Company of up to CAD$6.5 million.