On November 12, 2020 Oncorus, Inc. (Nasdaq:ONCR), a viral immunotherapies company focused on driving innovation to transform outcomes for cancer patients, reported third quarter 2020 financial results and highlighted recent achievements and developments (Press release, Oncorus, NOV 12, 2020, View Source [SID1234570765]).

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’ve made significant strides thus far in 2020 advancing our mission to realize the full promise of viral immunotherapy for cancer patients, including the commencement of a Phase 1 clinical trial for our lead clinical candidate, ONCR-177, as well as entering into a clinical trial collaboration with Merck to study ONCR-177 in combination with KEYTRUDA as part of the trial," said Theodore (Ted) Ashburn, M.D., Ph.D., President and Chief Executive Officer of Oncorus.

Dr. Ashburn further commented, "Bolstered by the completion of our IPO last month and a strong cash position, we continue to progress our pipeline of intratumorally and intravenously administered viral immunotherapies across our oncolytic Herpes Simplex Virus (oHSV) and Synthetic Virus Platforms. Our goal is to treat a broad spectrum of cancers to bring the full potential of this therapeutic class to as many patients as possible."

Third Quarter 2020 and Recent Highlights

Completed initial public offering (IPO) in October. Oncorus’s common stock commenced trading on the Nasdaq Global Market under the ticker symbol "ONCR" on October 2, 2020. The IPO, at a public offering price of $15.00 per share, raised $98.4 million in aggregate gross proceeds, including shares sold to the underwriters pursuant to the partial exercise of their option to purchase additional shares.
Initiated Phase 1 clinical trial of ONCR-177. In June, Oncorus initiated a Phase 1 clinical trial of its lead product candidate, ONCR-177, an intratumorally administered oHSV viral immunotherapy being developed for multiple solid tumor indications. The Phase 1 open-label, multi-center, dose escalation and expansion clinical trial is designed to evaluate the safety and tolerability of ONCR-177 and to determine the recommended Phase 2 dose, as well as its preliminary anti-tumor activity, alone and in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab), in patients with advanced and/or refractory cutaneous, subcutaneous or metastatic nodal solid tumors. Oncorus anticipates reporting interim data from the Phase 1 clinical trial in the second half of 2021 through the second half of 2022.
Announced clinical trial collaboration with Merck. In July, Oncorus announced a clinical trial collaboration and supply agreement with Merck, known as MSD outside of the United States and Canada, through a subsidiary, to evaluate the combination of ONCR-177 with KEYTRUDA, as part of the ongoing Phase 1 clinical trial of ONCR-177 in adult patients with advanced and/or refractory cutaneous, subcutaneous or metastatic nodal solid tumors.
Continued to advance lead Synthetic Virus Platform programs toward clinical candidate nominations. Oncorus continues to advance its lead synthetic, intravenously (IV) administered viral immunotherapy programs based on the coxsackievirus A21 (CVA21) and the Seneca Valley Virus and expects to nominate clinical candidates for both programs in the first half of 2021. IV administration of viral immunotherapies is an attractive approach for improving the standard of care for many oncology patients because it allows for all tumors in a patient, including the micro-metastases that are sometimes difficult to detect and treat, to be treated directly. In addition, it allows for the potential treatment of certain tumors, such as those of the lung, that are less amenable to repeat intratumoral injection of anti-cancer therapies for both safety and feasibility reasons. Oncorus’s Synthetic Virus Platform includes its novel lipid nanoparticle delivery strategy designed to overcome the challenges caused by neutralizing antibodies that have limited the efficacy of previous industry efforts to treat tumors utilizing IV administration of CVA21.
Continued to advance second oHSV viral immunotherapy candidate, ONCR-GBM. Leveraging its oHSV Platform, Oncorus is pursuing ONCR-GBM to specifically target brain cancer, including glioblastoma multiforme (GBM). Oncorus is utilizing its knowledge of microRNA expression to engineer a microRNA attenuation strategy to protect healthy brain tissue and select a combination of payloads that address the specific drivers of immune suppression in brain cancer. Oncorus plans to nominate its ONCR-GBM clinical candidate in the second half of 2021.
Third Quarter Financial Results

Cash and cash equivalents were $54.0 million as of September 30, 2020, excluding the proceeds from the Company’s initial public offering in October 2020, compared to $45.3 million as of December 31, 2019.
Research and development expenses for the quarter ended September 30, 2020 were $6.9 million compared to $6.2 million for the corresponding period in 2019. The increase in research and development expenses was mainly attributable to increases in clinical trial costs for the Company’s Phase 1 clinical trial as well as increased personnel-related expenses driven by increased headcount.
General and administrative expenses for the quarter ended September 30, 2020 were $2.0 million compared to $1.5 million for the corresponding period in 2019. The increase in general and administrative expenses was primarily attributable to increases in audit and legal activity and related expenses as well as personnel-related expenses driven by increased headcount.
Other income (expense), net, for the quarter ended September 30, 2020 was an expense of $10.6 million compared to income of $0.2 million for the quarter ended September 30, 2019. Other expense in 2020 included a non-cash charge of $10.6 million related to an increase in the fair market value of the Company’s Series B preferred stock tranche rights liability that was settled in September 2020, upon the closing of the second tranche of the Series B financing, which occurred prior to the IPO.
Net loss attributable to common stockholders for the quarter ended September 30, 2020 was $22.4 million, or $(21.73) per share, compared to a net loss attributable to common stockholders of $9.2 million, or $(9.67) per share for the same period in 2019. The share and loss per share amounts do not reflect the impact of the Company’s IPO, which closed in October 2020.
Financial Guidance

Based upon its current operating plans and cash and cash equivalents, including the net proceeds from the IPO, the Company expects to have sufficient capital to fund its operating expenses and capital expenditure requirements into 2023.

On November 12, 2020 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer,reported third quarter financial results for the period ended September 30, 2020 and provided an update on recent company developments (Press release, SpringWorks Therapeutics, NOV 12, 2020, View Source [SID1234570763]).

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 pleased with our achievements in the third quarter of 2020 across our clinical development, business development and corporate initiatives. We completed enrollment in the Phase 3 DeFi trial, advanced our strategy to develop nirogacestat as a cornerstone of BCMA combination therapy for patients with multiple myeloma by signing additional collaborations with industry and academic leaders, and continued to progress our trials in biomarker defined metastatic solid tumors," said Saqib Islam, Chief Executive Officer of SpringWorks. "Our ten clinical development programs are progressing as planned and we look forward to providing further updates as we advance our pipeline and continue to execute on our strategy to build a leading targeted oncology company."

Recent Business Highlights and Upcoming Milestones

Late-Stage Rare Oncology

Completed enrollment in the Phase 3 DeFi trial evaluating nirogacestat in adult patients with progressing desmoid tumors. SpringWorks expects to report topline data from the DeFi study in the second or third quarter of 2021.
Recruiting patients in a Phase 2 study sponsored by the Children’s Oncology Group evaluating nirogacestat in pediatric patients with desmoid tumors.
Highlighted a publication by investigators at the University of Minnesota and the Dana-Farber Cancer Institute in the Pediatric Cancer & Blood journal reporting data from four pediatric and young adult desmoid tumor patients who received nirogacestat under the SpringWorks Expanded Access Program. In these four patients, the investigators reported one complete response, two partial responses, and one stable disease, with no grade 3 or 4 adverse events.
Enrollment is ongoing in the Phase 2b ReNeu trial evaluating mirdametinib in pediatric and adult patients with NF1-associated plexiform neurofibromas. SpringWorks expects to provide an update on the ReNeu trial in the fourth quarter of 2020 or first quarter of 2021.
B-cell Maturation Antigen (BCMA) Combinations in Multiple Myeloma

Enrollment is ongoing in a Phase 1b trial evaluating nirogacestat in combination with BLENREP (belantamab mafodotin-blmf), GSK’s anti-B-cell maturation antigen (BCMA) antibody-drug conjugate, in patients with relapsed or refractory multiple myeloma.
Entered into a clinical collaboration with Janssen Biotech, Inc. (Janssen) to evaluate nirogacestat in combination with Janssen’s BCMA CD3 bispecific antibody, teclistamab, in patients with relapsed or refractory multiple myeloma. A Phase 1 study is expected to commence by early 2021, pending discussions with regulators.
Entered into a clinical collaboration with Pfizer Inc. (Pfizer) to evaluate nirogacestat in combination with Pfizer’s BCMA CD3 bispecific antibody, PF‐06863135, in patients with relapsed or refractory multiple myeloma. A Phase 1b/2 study is expected to commence in the first half of 2021.
An IND application is expected to be filed by the end of 2020 to evaluate nirogacestat in combination with Allogene’s BCMA AlloCAR T therapy, ALLO-715, in patients with relapsed or refractory multiple myeloma.
Entered into a clinical collaboration with Precision BioSciences, Inc. (Precision) to evaluate nirogacestat in combination with Precision’s allogeneic CAR T candidate targeting BCMA, PBCAR269A, in patients with relapsed or refractory multiple myeloma. A Phase 1/2a clinical study is expected to commence in the first half of 2021, pending discussions with regulators.
Entered into a sponsored research agreement with Fred Hutchinson Cancer Research Center (Fred Hutch) to further explore the ability of nirogacestat to modulate BCMA and potentiate BCMA-targeting therapies in a variety of preclinical and patient-derived multiple myeloma models developed by researchers at Fred Hutch.
Biomarker-Defined Metastatic Solid Tumors

Enrollment is ongoing in a Phase 1b/2 trial evaluating mirdametinib with BeiGene’s RAF dimer inhibitor, lifirafenib, in patients with RAS/RAF mutant and other MAPK pathway aberrant solid tumors. SpringWorks and BeiGene expect to report initial clinical data in 2021.
Enrollment is ongoing in a Phase 1 trial of BGB-3245 in patients with RAF mutant solid tumors. BGB-3245 is a selective RAF dimer inhibitor being developed by MapKure, LLC, an entity that is jointly owned by SpringWorks and BeiGene, Ltd. Initial clinical data from the Phase 1 study are expected in 2021.
General Corporate

In October 2020, SpringWorks completed a follow-on public offering that raised $269.5 million in net proceeds, which included the underwriters’ full exercise of their option to purchase additional shares of common stock.
SpringWorks and Jazz Pharmaceuticals entered into an asset purchase and exclusive license agreement under which Jazz acquired SpringWorks’ fatty acid amide hydrolase (FAAH) inhibitor program, PF-04457845. Under the terms of the agreement, Jazz assumed all milestone and royalty obligations owned by SpringWorks to Pfizer under the 2017 license agreement pursuant to which SpringWorks obtained rights to this asset. In addition, Jazz made an upfront payment of $35 million to SpringWorks, with potential future milestone payments of up to $375 million payable to SpringWorks based upon the achievement of certain clinical development, regulatory and commercial milestones. SpringWorks is also entitled to receive tiered royalties on future net sales of PF-04457845.
Third Quarter and Year to Date 2020 Financial Results

Research and Development (R&D) Expenses: R&D expenses were $13.9 million and $36.6 million for the third quarter and year-to-date periods, respectively, compared to $10.7 million and $30.4 million for the comparable periods of 2019, respectively. The increases in R&D expenses in 2020 were primarily attributable to growth in employee costs associated with increases in the number of R&D personnel, an increase in non-cash share-based compensation expense and increases in external costs related to drug manufacturing and clinical trial costs.
General and Administrative (G&A) Expenses: G&A expenses were $7.7 million and $20.9 million for the third quarter and year-to-date periods, respectively, compared to $4.6 million and $11.5 million for the comparable periods of 2019, respectively. The increases in G&A expenses in 2020 were primarily attributable to growth in employee costs associated with increases in the number of G&A personnel supporting the growth of the organization, and an increase in non-cash share-based compensation expenses, as well as increases in expenses related to the expansion of business activities.
Net Loss Attributable to Common Stockholders: SpringWorks reported net losses of $21.7 million, or $0.51 loss per share, and $56.8 million, or $1.35 loss per share, for the third quarter and year-to-date periods ended September 30, 2020, respectively. This compares to net losses of $16.8 million, or $1.77 loss per share, and $34.4 million, or $9.24 loss per share, for the comparable periods of 2019, respectively.
Cash Position: Cash, cash equivalents and marketable securities were $276.8 million as of September 30, 2020. This does not include the net proceeds of $269.5 million from the Company’s follow-on public offering completed in October 2020 or the $35 million upfront payment Jazz Pharmaceuticals made to SpringWorks in October 2020.
COVID-19 Update
To date, the COVID-19 pandemic has had a relatively modest impact on SpringWorks’ business operations, in particular on SpringWorks’ clinical trial programs, and SpringWorks is undertaking considerable efforts to mitigate the various challenges presented by this crisis. For further details and descriptions of the risks associated with the COVID-19 pandemic, please see the Risk Factors in SpringWorks’ Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 12, 2020 and refer to the Forward-Looking Statements section in this press release.

On November 12, 2020 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) reported that preclinical data on BCX9930, an oral Factor D inhibitor, will be presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held as a virtual event, December 5-8, 2020 (Press release, BioCryst Pharmaceuticals, NOV 12, 2020, View Source [SID1234570761]).

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 abstract is available on the ASH (Free ASH Whitepaper) website at www.hematology.org.

Preclinical Characterization of BCX9930, a Potent Oral Complement Factor D Inhibitor, Targeting Alternative Pathway-Mediated Diseases Including Paroxysmal Nocturnal Hemoglobinuria (PNH); Poster #1680, Sunday, December 6, 2020, 10:00 a.m. – 6:30 p.m. ET
About BCX9930
Discovered by BioCryst, BCX9930 is a novel, oral, potent and selective small molecule inhibitor of Factor D currently in Phase 1 clinical development for the treatment of complement-mediated diseases. The U.S. Food and Drug Administration (FDA) has granted both Fast Track status and Orphan Drug designation for BCX9930 in PNH. In an ongoing dose ranging trial of BCX9930 in patients with PNH, BCX9930 was safe and well tolerated, with no drug-related serious adverse events. As a Factor D inhibitor, BCX9930 is designed as an oral monotherapy that can address both intravascular and extravascular hemolysis in PNH patients. Treatment-naïve PNH patients who have received more than six weeks of therapy at a monotherapy dose of 400 mg bid showed rapid and dose-dependent reductions in key biomarkers, including LDH, and increases in hemoglobin levels that were maintained without transfusions.

On November 12, 2020 Sierra Oncology, Inc. (SRRA), a late-stage biopharmaceutical company focused on the Phase 3 execution, registration and potential commercialization of momelotinib, a novel drug that may address serious unmet needs in myelofibrosis, reported that President and Chief Executive Officer Stephen Dilly, MBBS, PhD, will present an overview of the company at the Jefferies Virtual London Healthcare Conference taking place November 17-19, 2020 (Press release, Sierra Oncology, NOV 12, 2020, View Source [SID1234570760]).

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 presentation is scheduled for 7:20 pm GMT on Wednesday, November 18. A replay of the presentation will be available following the conference on the Investors section of Sierra’s corporate website in the Events & Webcast tab.

On November 12, 2020 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported its recent accomplishments and financial results for the quarter ended September 30, 2020 (Press release, Soligenix, NOV 12, 2020, View Source [SID1234570759]).

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 continue to look to the future with our Specialized BioTherapeutics business segment," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "With the recent successful completion of our pivotal Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) trial, SGX301 has demonstrated the potential to be a significant new treatment for early-stage cutaneous T-cell lymphoma (CTCL). In the double-blind, placebo controlled Cycle 1 portion of the study, a statistically significant treatment response (p=0.04) was achieved in the primary endpoint after just 6 weeks of therapy. This positive treatment response continued to significantly improve with extended SGX301 treatment in the open-label treatment cycles at 12 weeks (Cycle 2) and 18 weeks (Cycle 3), reinforcing the positive SGX301 primary endpoint treatment response demonstrated in Cycle 1. With the study now concluded, we will begin preparing our new drug application for submission to the FDA. We also continue to progress our pivotal Phase 3 DOM-INNATE (Dusquetide treatment in Oral Mucositis – by modulating INNATE Immunity) study for SGX942 (dusquetide), for the treatment of oral mucositis in patients with head and neck cancer receiving chemoradiation therapy. With enrollment of 268 subjects completed, top-line final results continue to be expected before the end of the year."

Dr. Schaber continued, "Under our Public Health Solutions business segment, supported by non-dilutive government funding, we continue to advance our work with the University of Hawaiʻi at Mānoa (UHM) and Hawaii Biotech Inc. on filovirus vaccines (protecting against viruses such as Ebola and Marburg) and the development of vaccines to potentially combat coronaviruses, including SARS-CoV-2, the cause of COVID-19. We recently announced publication of positive pre-clinical data from immunogenicity studies with CiVax (heat stable COVID-19 vaccine candidate), demonstrating immunity of both broad-spectrum antibody and cell-mediated, rapid onset immunity is possible using the novel CoVaccine HT adjuvant in-licensed from BTG Specialty Pharmaceuticals (a division of Boston Scientific Corporation). Our heat stable ricin vaccine, RiVax, continues to be supported with a National Institute of Allergy and Infectious Disease contract award. With over $11M in cash, not including our non-dilutive government funding, along with the at-the-market sales issuance agreement with B. Riley FBR, Inc. to judiciously supplement our cash runway as needed, we anticipate having sufficient capital to achieve multiple inflection points across our rare disease pipeline, including final top-line results in our SGX942 Phase 3 clinical trial in oral mucositis."

Soligenix Recent Accomplishments

On October 22, 2020, the Company announced the continued optional treatment with SGX301 (synthetic hypericin) across all lesions during the compassionate use, safety portion of the trial (Cycle 3), for a total of 6 months in the study, continued to significantly improve responses and remained safe and well-tolerated in its FLASH study. This data reinforces the positive SGX301 primary endpoint treatment response demonstrated in Cycle 1. SGX30l treatment in Cycle 3 further improved response rates, with 49% of patients electing to receive SGX301 for a total of 18 weeks demonstrating a 50% or greater reduction in their combined CAILS (Composite Assessment of Index Lesion Score) lesion score compared to 40% of patients demonstrating such a reduction after completing 12 weeks of SGX301 treatment in Cycle 2 (p=0.046). In addition, continued analysis of results has revealed that 12 weeks of SGX301 treatment (Cycle 2) is equally effective on both patch (response 37%, p=0.0009) and plaque (response 42%, p<0.0001) lesions of CTCL when compared to Cycle 1 placebo lesion responses. SGX301 continued to be very well tolerated, benefiting from the lack of hypericin circulation in the blood stream after targeted topical application to the lesions, as well as the use of visible light. To view this press release, please click here.

On September 15, 2020, the Company announced the publication of nonclinical results characterizing filovirus protein antigens (including for Ebola and Marburg viruses) and their thermostabilization. The article, authored by collaborators at the University of Colorado, University of Hawaiʻi at Mānoa (UHM) and Soligenix, is titled, "Preservation of Quaternary Structure in Thermostable, Lyophilized Filovirus Glycoprotein Vaccines: A Search for Stability-Indicating Assays" and has been accepted for publication in the Journal of Pharmaceutical Sciences. A copy of manuscript has been made available here. To view this press release, please click here.

On September 10, 2020, the Company conducted an Investor Webcast presentation on the use of its thermostabilized glycoprotein vaccine platform for the development of a COVID-19 vaccine, called CiVax. To listen to this Webcast Event, please click here and to view the press release, please click here.
Financial Results – Quarter Ended September 30, 2020

Soligenix’s revenues for the quarter ended September 30, 2020 were $0.6 million as compared to $1.3 million for the quarter ended September 30, 2019. Revenues included payments on a contract in support of RiVax, our ricin toxin vaccine candidate, grants received to support the development of SGX943 for treatment of emerging and/or antibiotic-resistant infectious diseases, ThermoVax, our thermostabilization technology, and the assessment of SGX942 safety in juvenile animals.

Soligenix’s basic net loss was $1.8 million, or ($0.06) per share, for the quarter ended September 30, 2020, as compared to $2.7 million, or ($0.14) per share, for the quarter ended September 30, 2019. This decrease in net loss was primarily the result of decreased research and development spending due to the completion of the CTCL trial.

Research and development expenses were $1.3 million as compared to $2.3 million for the quarters ended September 30, 2020 and 2019, respectively. The decrease in research and development spending for the quarter ended September 30, 2020 was primarily attributable to the reduction in expense due to the completion of the CTCL trial.

General and administrative expenses were $0.8 million for both the three months ended September 30, 2020 and 2019.

As of September 30, 2020, the Company’s cash position was approximately $11.3 million.