On August 9, 2021 Cytocom Inc. (Nasdaq: CBLI), a leading biopharmaceutical company creating next-generation immune therapies that focus on immune restoration and homeostasis, reported an update regarding its portfolio of clinical programs (Press release, Cleveland BioLabs, AUG 9, 2021, View Source [SID1234586175]).

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 excited and believe that we are well positioned to further the development of our clinical-stage pipeline and showcase the power of our expanded post-merger drug development capabilities," stated Michael K. Handley, President and CEO of Cytocom. "We are strategically focused on immune-modulating treatments designed to address anemia and neutropenia, emergent viruses, cancer, and autoimmune diseases."

Mr. Handley continued, "The coming months will be busy. We are completing the necessary steps to begin enrolling patients by year-end 2021 in a Phase 3 clinical trial for our lead drug candidate, CYTO-201, for the treatment of Crohn’s disease. Building on our legacy Cytocom pipeline are the Cleveland BioLabs assets, specifically entolimod, an immune-stimulatory agent. We are eager to explore new indications for entolimod and remain excited about the potential for toll-like receptor 5 agonists in treating neutropenia and anemia in cancer patients. Our team is working to put together development plans in hematology and we are in talks with a renowned medical facility to begin a clinical study using entolimod in the coming months."

Mr. Handley concluded, "In addition, we plan to follow in the first half of 2022 with a clinical trial exploring CYTO-401 as an adjunct to standard of care therapy to extend the duration of disease remission in patients with pancreatic cancer. We are also completing the necessary steps to activate clinical trial sites and begin enrolling patients in clinical trials exploring CYTO-205 in patients with acute and post-acute COVID-19. This could be a particularly compelling opportunity given the pernicious nature of the SARS-CoV-2 virus, which, despite growing vaccination rates, continues to impact millions of people worldwide. What’s more, the medical community has specifically expressed interest on a "long haulers" study."

CYTO-201 and Crohn’s Disease

Cytocom’s lead investigational drug candidate, CYTO-201, is being studied for the treatment of pediatric patients with Crohn’s disease, an inflammatory bowel disease that causes chronic inflammation of the gastrointestinal (or digestive) tract, causing symptoms such as persistent diarrhea, abdominal pain and rectal bleeding. Studies show that because the signs and symptoms of the disease are unpredictable, patients living with the disease endure significant burdens, not only physical, but also emotional and economic.

On July 27, 2021, Cytocom completed a successful end of Phase 2 meeting with the U.S. Food and Drug Administration (FDA) regarding a clinical development plan for a Phase 3 clinical trial evaluating CYTO-201 in pediatric Crohn’s patients. Cytocom is partnering with ICON plc (NASDAQ: ICLR), a global contract research organization (CRO), to manage the trial, and expects to begin enrolling patients by year-end 2021.

Entolimod and Hematology

Cytocom’s new management team is now reviewing information regarding the past development work for entolimod, as well as previous clinical trial data. The company plans to address the clinical hold imposed by the U.S. Food and Drug Administrations (FDA) in 2019 in order to initiate clinical trials for new indications. In addition to continuing work on the Acute Radiation Syndrome or ARS, Cytocom plans to explore new indications for entolimod based on the potential of toll-like receptor 5 agonists in hematology indications, specifically the treatment of neutropenia and anemia in cancer patients. Cytocom is developing a hematology program and discussions are underway with a renowned academic institution to potentially initiate the first study later this year using entolimod in a hematology indication.

CYTO-401 and Pancreatic Cancer

Cytocom is developing CYTO-401 as an adjunct to standard of care therapy to extend the duration of disease remission in patients with pancreatic cancer. In August 2021, the company received FDA feedback regarding the clinical development program. Having feedback on the development program and establishing an advisory panel of oncology experts, Cytocom is now evaluating contract research organizations (CROs) and is planning to initiate the clinical trial in the first half of 2022.
CYTO-205 and Acute and Post-Acute COVID-19

With the FDA having cleared an Investigational New Drug (IND) application, Cytocom expects to begin a Phase 2 clinical trial in the third quarter of 2021 to evaluate the safety and efficacy of CYTO-205 to slow or halt the progression of the SARS-CoV-2, the virus that causes COVID-19.

Cytocom is finalizing protocols for a study evaluating CYTO-205 in patients with post-acute COVID-19 syndrome (PACS). Also known as "long haulers," these patients represent a high unmet medical need with roughly 30% of all COVID-19 infections developing into long-haul syndrome. Cytocom plans to conduct the trial under the existing IND and expects to begin enrolling patients by year-end 2021. CYTO-205 is designed to modulate immune system function by decreasing elevated inflammatory responses associated with viral infection.

On August 9, 2021 Epizyme (Nasdaq: EPZM), a fully integrated, commercial-stage biopharmaceutical company developing and delivering novel epigenetic therapies, reported its second quarter 2021 financial results and provided a business update (Press release, Epizyme, AUG 9, 2021, View Source [SID1234586079]).

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!

"TAZVERIK has significant potential as a backbone of therapy in both epithelioid sarcoma and follicular lymphoma based on real-world evidence and physician feedback. With that in mind, the challenging launch environment and evolving expectations of the future commercial landscape have led us to revise our commercial strategy and operating plans, prioritizing company resources on our most important value-driving activities," said Robert Bazemore, President and Chief Executive Officer of Epizyme. "Most significantly, we have taken steps to realign our current commercial organization, reducing traditional sales roles and creating new field roles to achieve better access to thought leaders and large community oncology practices. These, and other changes across our business, are designed to reduce our cash burn and allow us to maintain sufficient operating capital to achieve important milestones."

"As we implement these changes, we continue to execute across the business. During the second quarter we launched EZH2Now, a sponsored program to improve access to EZH2 mutation testing; received FDA clearance of our IND for EZM-0414, our novel SETD2 inhibitor, which we anticipate advancing into clinical development later this year; and advanced the ongoing clinical trials of TAZVERIK, all of which remain on track with previous guidance. In addition, this morning we announced a strategic partnership with HutchMed to bring TAZVERIK to patients in China and expand the development of tazemetostat in new combinations. These important accomplishments bring diversity to our pipeline while advancing TAZVERIK’s potential."

Recent Highlights

Commercial Execution: TAZVERIK generated net product revenue of $8.0 million in the second quarter of 2021 consisting of $4.8 million in commercial sales in Epithelioid Sarcoma (ES) and Follicular Lymphoma (FL) and $3.2 million related to the sale of commercial product to a third-party pharmaceutical company for use in its combination clinical trials. Although commercial sales were down from the first quarter 2021, total patient demand slightly increased by 3%, offset by higher demand for the Company’s patient assistance program during the quarter. The Company continued to expand adoption through the addition of new prescribing accounts, including among large community practices, in the second quarter of 2021.
Announced Collaboration with HutchMed in China: The commercial and development collaboration with HutchMed will extend TAZVERIK’s reach to China and allow for additional exploration of TAZVERIK in new combinations across multiple tumor types. Epizyme will receive an upfront payment of $25 million from HutchMed in the fourth quarter of 2021 as a result of the collaboration, with potential future development, regulatory and commercial milestone payments of up to an aggregate of $285 million over the life of the collaboration, in addition to royalties on TAZVERIK sales in Greater China.
EZH-302 Phase 1b/3 Confirmatory Study of TAZVERIK in Follicular Lymphoma: The combination of TAZVERIK with R2 (lenalidomide and rituximab) is being evaluated in a Phase 1b/3 confirmatory study in relapsed or refractory (R/R) FL patients. During the second quarter of 2021, Epizyme completed enrollment of all Phase 1b cohorts of the Phase 1b/3 trial. Of the 36 patients enrolled in this safety run-in, 17 patients are evaluable for efficacy to date based on the availability of tumor scans. All 17 patients have achieved an objective response to treatment, with six patients having a complete response and 11 patients having a partial response.
IND Clearance for Epizyme’s Novel SETD2 Inhibitor: Epizyme reported the clearance of its Investigational New Drug (IND) application from the FDA for its novel SETD2 inhibitor, EZM-0414. The Company expects to initiate a first-in-human clinical trial later this year. SETD2 inhibition has been shown to have clinical potential in multiple settings, including high risk t(4;14) multiple myeloma and in other B-cell malignancies such as Large-cell Lymphoma, as monotherapy and in combination with existing and emerging therapies including tazemetostat.
Launched EZH2 Now Testing Program: Epizyme launched the EZH2Now Testing Program on June 16, 2021, with Quest Diagnostics, the leading provider of diagnostic information services, to provide EZH2 mutation testing for patients with R/R FL. The Company expects this program will promote interest in, and access to, EZH2 single gene mutation testing. Epizyme created this program with Quest in response to market research conducted in the first quarter of 2021 that indicated approximately one-third of physicians surveyed did not have an easy way to test their patients for EZH2 mutation.
Additional Ongoing Clinical Trials of Tazemetostat in Follicular Lymphoma: EZH-1401, Epizyme’s Phase 2 trial evaluating TAZVERIK plus rituximab in R/R FL continues to move forward as planned and is actively enrolling. Patient enrollment also continues in the Lymphoma Study Association (LYSA) trial in front-line FL and Diffuse Large B-cell Lymphoma (DLBCL), as well as other investigator sponsored trials.
IND Clearance for Solid Tumor Basket Trial: Epizyme received clearance of its IND from the FDA for a solid tumor basket trial, EZH-1301, which will evaluate tazemetostat safety and efficacy across multiple solid tumors. With this approach, the Company plans to study multiple combinations with standard-of-care therapies and novel mechanisms of action to expand the potential of tazemetostat. Epizyme plans to initiate the study later this year.
EZH-1101 Phase 1b/2 Study of Tazemetostat in Prostate Cancer: Based on encouraging preliminary safety and activity data, Epizyme initiated enrollment in the Phase 2 efficacy portion of this study evaluating enzalutamide plus tazemetostat compared to enzalutamide alone earlier this year in metastatic castration resistant Prostate Cancer (mCRPC) patients. The Phase 2 portion of the study is now over one-third enrolled. Epizyme plans to present updated safety and efficacy data from the Phase 1b safety run-in as part of a poster presentation during the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2021 in September.
Presented Preclinical and Clinical Data at EHA (Free EHA Whitepaper) and ASCO (Free ASCO Whitepaper) in June: Epizyme shared the discovery of a selective inhibitor of the SETD2 histone methyltransferase with potent in vitro and in vivo activity in a European Hematology Association (EHA) (Free EHA Whitepaper) 2021 oral presentation. The Company separately shared results of the Phase 1b Soft-tissue Sarcoma (STS) portion of its ongoing global randomized, double-blind, placebo-controlled study of tazemetostat plus doxorubicin as front-line therapy for advanced ES in an American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 poster presentation.
Operating Plan Refinement

Organizational Changes: In response to challenging market dynamics experienced over the course of the last twelve months since launching TAZVERIK, Epizyme will be making important changes to its operating plans that reduce the Company’s budgeted workforce and effect other cost reductions across the business. These changes include a refinement of Epizyme’s current field organization, aligned with a revised strategy for improved customer access and TAZVERIK adoption. The Company is creating new field roles meant to achieve better access to thought leaders and to large community accounts at the executive decision-maker level, while reducing the number of traditional sales roles. Epizyme is also shifting commercial resources to implement several digital approaches to reach both healthcare providers and patients directly. The new operating plan also achieves reductions in headcount and external spending across other areas of the business. The Company reduced its budgeted headcount by 20 percent. This includes 11 percent of current employees, resulting in estimated severance and termination costs of approximately $2.0 million. Epizyme expects to record these charges in the third quarter of 2021.
These changes are intended to allow the Company to better deliver on TAZVERIK adoption, and execute more effectively on the most important value-creating initiatives, continuing to advance the four pillars of its long-term growth strategy.

Revised Financial Guidance: Based on its refined commercial strategy and operating plan, including the cash it expects to generate from product sales and the $25 million upfront payments from its collaboration with HutchMed, Epizyme expects its current cash runway to extend into the fourth quarter of 2022. Additionally, the Company expects its non-GAAP adjusted operating expenses for 2021 to be between $220 and $230 million, down from previous guidance of $235 to $255 million.
Second Quarter 2021 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $244.0 million as of June 30, 2021, as compared to $298.9 million as of March 31, 2021.
Revenue: Total revenue for the second quarter of 2021 was $13.0 million, compared to $2.5 million for the second quarter of 2020. Total revenue for the second quarter of 2021 consisted of $8.0 million of net product revenue, comprised of $4.8 million in commercial net sales of TAZVERIK and $3.2 million of TAZVERIK related to the sale of commercial product by one of the Company’s customers to a third-party pharmaceutical company for use in its clinical trials. The remaining $5.0 million was collaboration revenue related to Epizyme’s supply agreement with Eisai.
Operating Expenses: Total GAAP operating expenses were $71.2 million for the second quarter of 2021 compared to $60.0 million for the second quarter of 2020. Total non-GAAP adjusted operating expenses were $63.2 million for the second quarter of 2021 compared to $50.9 million for the second quarter of 2020.
R&D expenses: GAAP R&D expenses were $34.9 million for the second quarter of 2021 compared to $26.4 million for the second quarter of 2020. Non-GAAP adjusted R&D expenses were $32.7 million for the second quarter of 2021 compared to $23.4 million for the second quarter of 2020.
SG&A expenses: GAAP SG&A expenses were $33.9 million for the second quarter of 2021 compared to $32.7 million for the second quarter of 2020. Non-GAAP adjusted SG&A expenses were $29.1 million for the second quarter of 2021 compared to $27.1 million for the second quarter of 2020.
Net Loss (GAAP): Net loss attributable to common stockholders was $64.4 million, or $0.63 per share, for the second quarter of 2021, compared to $58.5 million, or $0.58 per share, for the second quarter of 2020.
A reconciliation of non-GAAP adjusted financial measures directly comparable to GAAP financial measures is presented in the table attached to this press release.
Conference Call Information

Epizyme will host a conference call today, August 9, at 8:30 a.m. ET. To participate in the conference call, please dial (877) 844-6886 (domestic) or (970) 315-0315 (international) and refer to conference ID 3658407. A webcast, as well as supplemental slides to support the webcast, will be available in the investor section of the Company’s website at www.epizyme.com, and will be archived for 60 days following the call.

About Non-GAAP Financial Measures

In addition to financial information prepared in accordance with the U.S. generally accepted accounting principles (GAAP), this press release includes the following non-GAAP financial measures: total non-GAAP adjusted operating expenses on a historical and projected basis, non-GAAP adjusted R&D expenses on a historical basis and non-GAAP adjusted SG&A expenses on a historical basis. Epizyme derives these non-GAAP financial measures by excluding certain expenses and other items from the respective GAAP financial measure, that is most directly comparable to each non-GAAP financial measure. Specifically, the non-GAAP financial measures exclude stock-based compensation expense and depreciation and amortization of intangibles. The company’s management believes that these non-GAAP financial measures are useful to both management and investors in analyzing its ongoing business and operating performance. Management does not intend the presentation of these non-GAAP financial measures to be considered in isolation or as a substitute for results prepared in accordance with GAAP, but as a complement to provide greater transparency. In addition, these non-GAAP financial measures may differ from similarly named measures used by other companies. A quantitative reconciliation of projected non-GAAP adjusted operating expenses to total operating expenses is not available without unreasonable effort primarily due to the company’s inability to predict with reasonable certainty the amount of future stock-based compensation expense.

About TAZVERIK (tazemetostat)

TAZVERIK is a methyltransferase inhibitor indicated for the treatment of:

Adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection.
Adult patients with relapsed or refractory follicular lymphoma whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test and who have received at least two prior systemic therapies.
Adult patients with relapsed or refractory follicular lymphoma who have no satisfactory alternative treatment options.
These indications are approved under accelerated approval based on overall response rate and duration of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.

The most common (≥20%) adverse reactions in patients with epithelioid sarcoma are pain, fatigue, nausea, decreased appetite, vomiting and constipation. The most common (≥20%) adverse reactions in patients with follicular lymphoma are fatigue, upper respiratory tract infection, musculoskeletal pain, nausea and abdominal pain.

View the U.S. Full Prescribing Information here: Epizyme.com

On August 9, 2021 FibroGen, Inc. (NASDAQ: FGEN) reported financial results for the second quarter 2021 and provided an update on the Company’s recent developments (Press release, FibroGen, AUG 9, 2021, View Source [SID1234586115]).

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 be energized by the performance of roxadustat in China, where we are seeing impressive adoption of roxadustat in a rapidly expanding anemia of CKD market. In addition, the positive CHMP opinion in Europe brings roxadustat one step closer to patients in this important region," said Enrique Conterno, Chief Executive Officer, FibroGen. "We look forward to the European Commission decision following the positive CHMP opinion. On the other hand, we are disappointed with the FDA Cardiovascular and Renal Drugs Advisory Committee negative vote, and we will continue to work with our partner AstraZeneca and the FDA on a path forward."

Recent Key Events and Other Developments

Regulatory:

In June, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion relating to the use of roxadustat for the treatment of adult patients with symptomatic anemia associated with chronic kidney disease (CKD). A European Commission decision is expected in August 2021.
In July, the U.S. Food and Drug Administration (FDA) Cardiovascular and Renal Drugs Advisory Committee (CRDAC) voted to recommend not approving roxadustat for the treatment of anemia due to CKD. An FDA action on the new drug application is expected in the near future.
_____________________________

1 Total roxadustat net sales in China includes sales made by the distribution entity as well as FibroGen China’s direct sales, each to its own distributors. The distribution entity jointly owned by AstraZeneca and FibroGen is not consolidated into FibroGen’s financial statements.

Clinical:

Pamrevlumab included in the Pancreatic Cancer Action Network’s (PanCAN) Precision PromiseSM adaptive trial platform evaluating pamrevlumab for patients with metastatic pancreatic cancer.
China:

Roxadustat net product revenue in China of $13.4 million, on a US GAAP basis, including revenue generated from our sales to the distribution entity and FibroGen China’s direct sales, compared to $15.4 million last quarter.
Total roxadustat net sales in China of $52.8 million by FibroGen and the distribution entity jointly owned by FibroGen and AstraZeneca, compared to $43.5 million last quarter.
Hospital listings at the end of the second quarter represented approximately 81% of the CKD anemia market opportunity in China versus 74% last quarter.
Clinical Presentations / Publications:

FibroGen and its partners presented three roxadustat oral presentations and ten mini-oral presentations at the recent 58th European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) Virtual Congress.
Two additional roxadustat Phase 3 manuscripts on the treatment of anemia of CKD were published in a peer-reviewed medical journal, bringing the total to 7:
Roxadustat for the treatment of anaemia in chronic kidney disease patients not on dialysis: a phase 3, randomised, open-label, active-controlled study (DOLOMITES) Nephrology Dialysis Transplantation.
Efficacy and Cardiovascular Safety of Roxadustat for Treatment of Anemia in Patients with Non–Dialysis-Dependent CKD (NDD Pooled) Clinical Journal of the American Society of Nephrology.
FibroGen presented one oral presentation and one poster presentation of two-year data from a Phase 2 trial of pamrevlumab in non-ambulatory DMD patients at the recent Parent Project Muscular Dystrophy (PPMD) Annual Conference.
Upcoming Data Milestones:

Data from the Phase 2 WHITNEY study of roxadustat in chemotherapy-induced anemia (CIA) expected 3Q 2021 versus prior 2H 2021.
Topline data from the Phase 3 MATTERHORN study of roxadustat in anemia of myelodysplastic syndromes (MDS) now expected 2H 2022 / 1H 2023 versus prior 1H 2022.
Interim analysis of event free survival for potential accelerated approval of Phase 3 LAPIS study of pamrevlumab in locally advanced pancreatic cancer (LAPC) will be completed in 2H 2022.
Topline data from the Phase 3 LELANTOS-1 study of pamrevlumab in Duchenne muscular dystrophy (DMD) now expected 1H 2023 versus prior 2H 2022.
Topline data from the Phase 3 ZEPHYRUS-1 study of pamrevlumab in idiopathic pulmonary fibrosis (IPF) expected mid-2023.
Corporate

Appointed John Hunter, Ph.D. as Chief Scientific Officer.
FibroGen and HiFiBiO announced partnership to advance next-generation therapies for patients with cancer and autoimmune disease. FibroGen exclusively licensed HiFiBiO’s Galectin-9 program, and obtained an exclusive option to their CXCR5 and CCR8 programs.
Eluminex Biosciences exclusively licensed worldwide rights to develop and commercialize FibroGen’s investigational biosynthetic cornea program and the underlying recombinant human collagen Type III technology.
The Company has completed its internal review of the events leading to our April 6, 2021 disclosures in which we clarified that certain cardiovascular safety analyses from the roxadustat Phase 3 program for the treatment of anemia of chronic kidney disease included post-hoc changes to the stratification factors.
Financial:

Total revenue for the second quarter of 2021 was $24.4 million, as compared to $42.9 million for the second quarter of 2020. The current quarter revenue includes $13.4 million net product revenue for roxadustat sales in China, and $19.6 million in development revenue. The current quarter also includes $(8.6) million drug product revenue as a result of the recent unfavorable CRDAC vote.
Operating costs and expenses for the second quarter of 2021 included a one-time charge of $25 million related to our partnership with HiFiBio, and an increase of approximately $20 million driven primarily by pamrevlumab development expenses compared to one year ago.
Net loss for the second quarter of 2021 was $134.0 million, or $1.45 net loss per basic and diluted share, compared to a net loss of $85.3 million, or $0.95 net loss per basic and diluted share one year ago.
Conference Call and Webcast Details
FibroGen will host a conference call and webcast today, Monday, August 9, 2021, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time) to discuss financial results and provide a business update. A live audio webcast of the call may be accessed in the investor section of the Company’s website, www.fibrogen.com. To participate in the conference call by telephone, please dial 1 (877) 658-9081 (U.S. and Canada) or 1 (602) 563-8732 (international), reference the FibroGen second quarter 2021 financial results conference call, and use confirmation number 4951789. A replay of the webcast will be available shortly after the call for a period of four weeks. To access the replay, please dial 1 (855) 859-2056 (domestic) or 1 (404) 537-3406 (international), and use passcode 4951789.

About Roxadustat
Roxadustat, an oral medicine, is the first in a new class of medicines, HIF-PH inhibitors, that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin; improved iron absorption and mobilization; and downregulation of hepcidin. Roxadustat is also in clinical development for anemia associated with myelodysplastic syndromes (MDS) and for chemotherapy-induced anemia (CIA).

Roxadustat is approved in China, Japan, Chile, and South Korea for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). In Europe, the Marketing Authorisation Application is under review by the European Medicines Agency (EMA). In the U.S., the New Drug Application is under review by the U.S. Food and Drug Administration. Several other licensing applications for roxadustat have been submitted by Astellas and AstraZeneca to regulatory authorities across the globe, and are currently in review.

Astellas and FibroGen are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in territories including Japan, Europe, Turkey, Russia and the Commonwealth of Independent States, the Middle East, and South Africa. FibroGen and AstraZeneca are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in the U.S., China, other markets in the Americas, in Australia/New Zealand, and Southeast Asia.

About Pamrevlumab
Pamrevlumab is a first-in-class antibody developed by FibroGen that inhibits the activity of connective tissue growth factor (CTGF), an important biological mediator in fibrotic and proliferative disorders. Pamrevlumab is in Phase 3 clinical development for the treatment of locally advanced unresectable pancreatic cancer (LAPC), Duchenne muscular dystrophy (DMD), and idiopathic pulmonary fibrosis (IPF). For information about pamrevlumab studies currently recruiting patients, please visit www.clinicaltrials.gov.

On August 9, 2021 Cyteir Therapeutics, Inc. ("Cyteir") (Nasdaq: CYT), a company focused on the discovery and development of next-generation synthetically lethal therapies for cancer, reported financial results for the second quarter ended June 30, 2021 and provided an update on recent business highlights (Press release, Cyteir Therapeutics, AUG 9, 2021, View Source [SID1234586131]).

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 successful completion of our IPO in June gives us the capital to advance our drug candidates through mid-stage clinical development and places us in a strong position to advance our lead drug CYT-0851 and develop our DNA damage response (DDR) platform. We continue to enroll patients through the dose escalation portion of the Phase 1/2 trial for CYT-0851 and we plan to begin the Phase 2 expansion trial by year-end," said Markus Renschler, MD, President and Chief Executive Officer of Cyteir. "As we continue advancing our lead program and our DDR platform, we look forward to nominating new candidates in 2022 and beyond. I’d like to thank the entire Cyteir team for their dedication and excellent execution as we develop the next generation of synthetic lethal therapies to treat cancer."

Second Quarter Business Review and Operational Updates

CYT-0851

At the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, Ryan Lynch, MD of the Fred Hutchinson Cancer Center, presented an interim analysis of the Phase 1 portion of a first-in-human Phase 1/2 trial of CYT-0851. The presentation was given at an oral session at the conference.
The Phase 1 dose escalation trial portion of the Phase 1/2 trial continues to enroll patients. The 600 mg once daily cohort was recently cleared for safety and the 800 mg once daily cohort is now open for enrollment. Initiation of the Phase 2 expansion trial and Phase 1 combinations with chemotherapy are both expected by year-end.
Pipeline

Investigational New Drug (IND)-enabling studies with CYT-1853 are ongoing and an IND application is expected to be submitted in 2022. Cyteir is also advancing preclinical studies on additional targets to nominate the next target from the DDR platform.
Second Quarter Financial Results

Cash and cash equivalents: Cash and cash equivalents for the quarter ended June 30, 2021, were $198.6 million. The cash and cash equivalents total for the second quarter does not include the $15.9 million in gross proceeds raised from the exercise by the underwriters of the option to purchase additional shares of common stock.

Research and development (R&D) expenses: R&D expenses were $8.9 million for the second quarter of 2021 versus $3.6 million for the same period in 2020. The year-over-year increase in R&D spending in the comparative periods was due primarily to increased R&D and clinical activity and increased headcount.

General and administrative (G&A) expenses: G&A expenses were $2.4 million for the second quarter of 2021 compared to $0.9 million for the same period in 2020. The year-over-year increase in G&A expenses was primarily due to increased headcount.

Net loss: Net loss was $11.3 million, or $4.83 per share in the second quarter of 2021 compared to $4.5 million, or $3.18 per share in the second quarter of 2020.

On August 9, 2021 Sesen Bio (Nasdaq: SESN), a late-stage clinical company developing targeted fusion protein therapeutics for the treatment of patients with cancer, reported operating results for the second quarter ended June 30, 2021 . The Biologics License Application (BLA) for the Company’s lead program, Vicineum for the treatment of BCG-unresponsive non-muscle invasive bladder cancer (NMIBC), is currently under Priority Review with the US Food and Drug Administration (FDA) with a target Prescription Drug User Fee Act (PDUFA) date of August 18, 2021.

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 excited about the regulatory progress we are making across our global markets," said Dr. Thomas Cannell, president and chief executive officer of Sesen Bio. "We remain focused on executing a world-class launch in the US if Vicineum is approved, and we also continue to support our partners outside of the United States, as part of our commitment to deliver a therapy we believe can improve patient outcomes globally while reducing overall healthcare costs for patients."

US and Outside of the US (OUS) Regulatory Update

US:

On July 13, 2021, Sesen Bio participated in a productive Late-Cycle Meeting with the FDA regarding the BLA for Vicineum for the treatment of BCG-unresponsive NMIBC. In the meeting, the FDA confirmed that there is no Advisory Committee meeting planned at this time, and that no post-marketing requirements, including a confirmatory trial, have been identified at this time. Also in the meeting, the Company and the FDA discussed remaining questions related to manufacturing facility inspections, product quality information requests and additional information related to chemistry, manufacturing and controls (CMC), and a timeline to submit additional supporting information was agreed upon. The Company believes it remains on track for an FDA decision on its BLA for Vicineum by the target PDUFA date of August 18, 2021.
Europe:

In Europe, the Company believes it remains on track for potential approval of Vysyneum in 2022. The Company has received the Day 80 and Day 120 questions from the European Medicines Agency (EMA) and is responding to inquiries and providing supporting information as part of the official review process.
China:

On July 20, 2021, Sesen Bio and Qilu Pharmaceutical, the Company’s partner in Greater China, announced that the first patient had been enrolled in China in the clinical trial to assess the efficacy and safety of Vicineum in patients with BCG-unresponsive NMIBC. The trial, which plans to enroll approximately 53 patients with carcinoma in situ (CIS), is being run at the sole cost of Qilu Pharmaceutical. If the trial is successful, Qilu Pharmaceutical anticipates submission of the product market application for Vicineum in China in 2022, with potential approval expected in 2023.
Middle East and North Africa (MENA):

The Company continues to work closely with its partner in MENA, Hikma Pharmaceuticals, to submit marketing authorization applications for Vicineum in 2021 in seven key markets in the region: Kingdom of Saudi Arabia, Jordan, Morocco, Egypt, Lebanon, Kuwait and Algeria. These markets represent a significant opportunity, with some of the most advanced healthcare systems and largest economies in the MENA region. The Company believes it is on track for potential market approvals to begin in the region in 2022.
Business Development Update

On June 1, 2021, the Company entered into a global supply agreement with Qilu Pharmaceutical to become part of the manufacturing network for global commercial supply of Vicineum drug substance and drug product. This was an expansion of the initial commercial manufacturing and supply framework agreement entered into by Sesen Bio and Qilu Pharmaceutical in December 2020, and sets specific terms such as capacity, forecasts, pricing and product delivery. Along with existing world-class supply partners, Sesen Bio expects the global supply agreement with Qilu Pharmaceutical will enable the Company to meet anticipated significant global demand for Vicineum.
On August 5, 2021, Sesen Bio announced it had entered into a licensing agreement with Eczacibasi Pharmaceuticals Marketing (EIP) for the registration and commercialization of Vicineum in Turkey. Under the terms of the licensing agreement, Sesen Bio will receive an upfront payment of $1.5 million, is eligible to receive additional regulatory and commercial milestone payments and is also entitled to receive a 30% royalty on net sales in Turkey. EIP was granted an exclusive license to register and commercialize Vicineum for the treatment of BCG-unresponsive NMIBC in Turkey, where bladder cancer is the sixth most commonly diagnosed cancer and 11th most common cause of death. This agreement represents the third OUS partnership that Sesen Bio has entered to date.
Commercial Planning Update

Sesen Bio completed its commercial build phase in preparation for the anticipated launch of Vicineum, if approved, in the US, and has advanced to the implementation phase that will commence promptly, if approved, and will focus on executing the Company’s commercial strategy for Vicineum. The Company has completed the hiring of ~25 talented internal employees to support the Company cross-functionally, as well as the hiring of 35 sales representatives as part of the contract sales organization who will target approximately 2,000 high-prescribers of BCG to drive awareness, trial and adoption of Vicineum for the treatment of patients with BCG-unresponsive NMIBC.
In addition to building its sales force, as part of the Sesen Bio national speaker programs, the Company has identified and commenced training of 14 key opinion leader (KOL) speakers to educate their peers on Vicineum for the treatment of BCG-unresponsive NMIBC, if approved. Upon product availability, the Company will utilize a two-pronged market access and reimbursement strategy to ensure maximum coverage for Vicineum.

Promotional efforts will begin immediately upon the anticipated approval of Vicineum in the US, and the Company expects Vicineum product to be available to physicians and patients in the fourth quarter of 2021.

Results of market research conducted by the Company show that when given the choice between the product profile of Vicineum, based on Phase III clinical trial data1, and the product profile of Keytruda, physicians will choose Vicineum over 80% of the time. This data highlights urologists’ preferences that the Company believes spans clinical, emotional and economic reasons to prescribe Vicineum. If approved by the FDA, the Company believes Vicineum could be a best-in-class treatment option for patients, and a critical step in Sesen Bio realizing its mission to save and improve the lives of patients with cancer.
[1]The Phase III clinical data are based on the data submitted in the BLA on December 18, 2020 and are currently under review by the FDA. Final efficacy and safety data are pending.

Second Quarter 2021 Financial Results

Cash Position: Cash, cash equivalents and restricted cash were $151.1 million as of June 30, 2021, compared to $55.4 million as of December 31, 2020.
R&D Expenses: Research and development expenses for the second quarter of 2021 were $7.2 million compared to $4.6 million for the same period in 2020. The increase of $2.7 million was due to increased costs associated with technology transfer and manufacturing ($1.0 million), professional services in support of regulatory activity ($0.7 million), employee-related compensation ($0.7 million), and other increases ($0.3 million).
G&A Expenses: General and administrative expenses for the second quarter of 2021 were $6.8 million compared to $3.3 million for the same period in 2020. The increase of $3.5 million was due primarily to increases in sales and marketing expense for Vicineum pre-commercial launch planning ($1.6 million), employee-related compensation driven by increased headcount as part of the commercial build ($1.3 million), and other increases for commercial launch preparation ($0.6 million).
Net Loss: Net loss was $25.4 million, or $0.15 per share, for the second quarter of 2021, compared to net loss of $26.3 million, or $0.24 per share, for the second quarter of 2020. The change was attributable to license revenue recognized ($2.2 million), offset by higher operating expenses ($1.3 million).
About Vicineum

Vicineum, a locally administered fusion protein, is Sesen Bio’s lead product candidate being developed for the treatment of BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). Vicineum is comprised of a recombinant fusion protein that targets epithelial cell adhesion molecule (EpCAM) antigens on the surface of tumor cells to deliver a potent protein payload, Pseudomonas Exotoxin A. Vicineum is constructed with a stable, genetically engineered peptide tether to ensure the payload remains attached to the antibody binding fragment until it is internalized by the cancer cell. This fusion protein design is believed to decrease the risk of toxicity to healthy tissues, thereby improving its safety. In prior clinical trials conducted by Sesen Bio, EpCAM has been shown to be overexpressed in NMIBC cells with minimal to no EpCAM expression observed on normal bladder cells. Sesen Bio is currently in the follow-up stage of a Phase 3 registration trial in the US for the treatment of BCG-unresponsive NMIBC. In February 2021, the FDA accepted the Company’s BLA file for Vicineum for the treatment of BCG-unresponsive NMIBC and granted the application Priority Review with a target PDUFA date of August 18, 2021. Additionally, Sesen Bio believes that cancer cell-killing properties of Vicineum promote an anti-tumor immune response that may potentially combine well with immuno-oncology drugs, such as checkpoint inhibitors. For this reason, the activity of Vicineum in BCG-unresponsive NMIBC is also being explored at the US National Cancer Institute in combination with AstraZeneca’s immune checkpoint inhibitor durvalumab.