On May 12, 2021 Immunocore Holdings Plc (Nasdaq: IMCR), a late-stage biotechnology company pioneering the development of a novel class of T cell receptor (TCR) bispecific immunotherapies designed to treat a broad range of diseases, including cancer, infectious and autoimmune disease, reported its results for the quarter ended March 31, 2021 (Press release, Immunocore, MAY 12, 2021, View Source [SID1234579813]).

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Highlights for the quarter included the presentation of the Phase 3 randomized data from the Company’s lead candidate tebentafusp in the plenary clinical trial session at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, the launch of a global early access program for tebentafusp, and the successful completion of the Company’s initial public offering resulting in net proceeds of $287 million.

Bahija Jallal, Chief Executive Officer of Immunocore, said: "Tebentafusp has been demonstrated to prolong survival in patients with metastatic uveal melanoma, a cancer that has historically proven insensitive to chemotherapy and immunotherapies. These data, recently presented at AACR (Free AACR Whitepaper), represent the first positive Phase 3 clinical trial for a TCR therapeutic and the first time that a bispecific T cell engager has demonstrated a survival benefit in a solid tumor, representing a significant breakthrough in the field of oncology."

First Quarter 2021 Highlights (including post-period)

Tebentafusp

In April, one oral presentation and three posters on tebentafusp were accepted at the 2021 American Society of Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held virtually from June 4-8, 2021. Per ASCO (Free ASCO Whitepaper)’s Embargo & Release Information, full abstracts will be released to the public on ASCO (Free ASCO Whitepaper)’s Meeting Library at 5:00 p.m. ET on May 19, 2021.

In April, the Company launched a global early access program for tebentafusp in metastatic uveal melanoma (mUM).

In April, the Company’s Phase 3 data of tebentafusp in metastatic uveal melanoma was also the subject of an oral presentation in the Phase 3 clinical trials plenary session at the AACR (Free AACR Whitepaper) Virtual Annual Meeting 2021. Tebentafusp demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) as a first-line treatment in mUM. In the intent-to-treat population, tebentafusp demonstrated a median overall survival of 21.7 months compared to 16.0 months for investigator’s choice and with 73% of patients alive at 1 year for tebentafusp vs. 58% for investigator’s choice. The OS Hazard Ratio (HR) favored tebentafusp, HR=0.51 (95% CI: 0.37, 0.71); p< 0.0001, over investigator’s choice (82% pembrolizumab; 12% ipilimumab; 6% dacarbazine). In addition, tebentafusp resulted in a statistically significant longer PFS. Treatment-related adverse events were manageable and consistent with the proposed mechanism.

In February, tebentafusp was granted Breakthrough Therapy Designation by the U.S. Food & Drug Administration (FDA) for unresectable or metastatic uveal melanoma. Additionally, the European Commission, upon recommendation of the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) awarded tebentafusp Orphan Drug Designation for the treatment of uveal melanoma. Medicines that meet the EMA’s Orphan Drug Designation criteria qualify for several incentives, including 10 years of market exclusivity, protocol assistance, and potentially reduced fees for regulatory activities.

Immunocore will be working to complete its BLA submission to the FDA in the third quarter of 2021, followed by submission of a Marketing Authorization Application to the EMA.

Additional Clinical Programs

IMC-C103C – MAGE-A4

In the first quarter, the Company continued to advance, IMC-C103C, an ImmTAC molecule targeting an HLA-A*02:01 MAGE-A4 antigen, in a first-in-human, Phase 1/2 dose escalation trial in patients with solid tumor cancers including non-small-cell lung cancer (NSCLC), gastric, head and neck, ovarian and synovial sarcoma. The Company plans to report Phase 1 initial data from this trial in the fourth quarter of 2021.

IMC-F106C – PRAME

In the first quarter, the Company continued to advance IMC-F106C, an ImmTAC molecule targeting an HLA-A*02:01 PRAME antigen, in a first-in-human, Phase 1/2 dose escalation trial in patients with multiple solid tumor cancers. PRAME is overexpressed in many solid tumors including NSCLC, SCLC, endometrial, ovarian, and breast cancers. The Company plans to report Phase 1 initial data from this trial in mid-2022.

IMC-I109V – HBV

In the first quarter, the Company continued to advance IMC-I109V, an ImmTAV molecule targeting a conserved Hepatitis B virus (HBV), envelope antigen, in a global Phase 1 single ascending dose trial. The Company plans to initiate dosing mid-year 2021.

Operational Highlights

In February, the Company made key appointments to management and Board of Directors. The appointment of Ralph Torbay as Immunocore’s new Head of Commercial and the appointment of Dr. Roy S. Herbst as a member of the Company’s Board of Directors became effective January 28, 2021. Dr. Herbst served as a member of Immunocore’s Scientific Advisory Board (SAB) and is currently Ensign Professor of Medicine (Medical Oncology), Professor of Pharmacology, Chief of Medical Oncology and Associate Cancer Center Director for Translational Research at Yale Cancer Center and Smilow Cancer Hospital.

In February, the Company completed its initial public offering (IPO) and concurrent private placement. The financing was $312.1 million in aggregate, of which approximately $287 million in net proceeds was from the IPO on Nasdaq of 11,426,280 American Depositary Shares (ADSs), including the exercise in full by the underwriters of their option to purchase an additional 1,490,384 ADSs, at an IPO price of $26.00 per ADS and $15 million from the completion of the concurrent sale of an additional 576,923 ADSs at the initial offering price of $26.00 per ADS, for gross proceeds of approximately $15.0 million, in a private placement to the Bill & Melinda Gates Foundation.

Financial Results

Basic and diluted loss per share was a £0.76 or $1.05 for the quarter ended March 31, 2021 compared to an adjusted to £0.74 for the quarter ended March 31, 2020. Total operating loss for the quarter was £31.9 million or $44.0 million compared to £22.1 million for the same period last year, largely due to an increase in employee costs associated with non-cash share-based payment charges.

For the three months ended March 31, 2021, revenue from collaboration agreements was unchanged at £8.3 million or $11.4 million compared to the same period last year. For the three months ended March 31, 2021, research and development expenses were £19.9 million or $27.4 million, as compared to £20.8 million for the three months ended March 31, 2020. For the quarter, administrative expenses were £20.2 million or $27.8 million compared to £9.6 million for the quarter ended March 31, 2020 including a £7.7 million increase in the non-cash share-based payment charges.

Cash and cash equivalents totaled £313.1 million or approximately $431 million as of March 31, 2021 compared to £68.4 million for the same period last year.

On May 12, 2021 IMV Inc. (the "Company" or "IMV") (TSX: IMV; NASDAQ: IMV), a clinical-stage biopharmaceutical company pioneering a novel class of immunotherapies, reported its financial and operational results for the first quarter ended March 31, 2021 (Press release, IMV, MAY 12, 2021, View Source [SID1234579812]).

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"We remain focused on our commitment to provide effective and well-tolerated immunotherapies for patients with difficult-to-treat cancers. Clinical programs with our lead immunotherapy are progressing as expected, and we are pleased that maveropepimut-S will soon be evaluated in subjects with HR+/HER2- breast cancer, another unmet medical need," announced Fred Ors, Chief Executive Officer at IMV. "We are reaching another milestone as our second immunotherapy, DPX-SurMAGE, is entering into a clinical trial in patients with bladder cancer later this year with the support of a C$1.8M grant. Also, we will now benefit from the expertise of two new board members who are recognized strategic thought leaders in immuno-oncology."

Clinical Programs

Maveropepimut-S: Phase 2B Study in Relapsed/Refractory DLBCL ("r/r DLBCL")

Following the results of the SPiReL study presented at The Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 35th Anniversary Annual Meeting in November, the Company announced in April that it has entered into an agreement with Merck (NYSE: MRK) to initiate a Phase 2B clinical trial to evaluate its lead compound, maveropepimut-S in combination with KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy in r/r DLBCL. The study is expected to begin in Q2.

Maveropepimut-S: Phase 2 DeCidE1 Study in Advanced, Recurrent Ovarian Cancer

IMV is currently analyzing translational data with the goal of identifying markers of activity to inform future development and to finalize the design of our Phase 2B trial.

Maveropepimut-S: Phase 2 Basket Trial in Multiple Advanced Metastatic Solid Tumors

Enrollment continues for subjects with metastatic bladder cancer, liver cancer (hepatocellular carcinoma, HCC), and for the microsatellite instability high (MSI-H) tumors. IMV intends to provide a clinical update on the trial in the second half of this year.

Maveropepimut-S with Standard of Care, with/without Radiotherapy, or Cyclophosphamide in Breast Cancer

This investigator-initiated clinical study will be conducted at the Providence Cancer Institute in Portland, Oregon. It is a three-arm Phase 1b trial designed to assess the combination of maveropepimut-S plus standard-of-care aromatase inhibitor with/without radiotherapy or CPA prior to surgery. Across the three arms of this study, IMV’s lead compound will be evaluated in 18 subjects with resectable, non-metastatic HR+/HER2 high breast cancer.

The Hormone Receptive (HR+) and HER2 negative (HER2-) is the most common form of breast cancer representing more than 70% of all cases. Investigators at Providence have identified ki67 as a prognostic marker of resistance to treatment that is associated with the upregulation of survivin expression. Targeting survivin with maveropepimut-S T cell therapy in this population represents a promising approach that will be tested in the study.

This investigator-initiated clinical study is expected to begin during Q3 2021.

DPX-SurMAGE, a First-in-Human Dual T Cell Therapy for Bladder Cancer

This IMV-sponsored trial will be led by Yves Fradet, M.D., professor of surgery and researcher in cancer immunotherapy at le Centre de recherche du CHU de Québec-Université Laval and his team, in collaboration with IMV’s team.

Both survivin and MAGE-9 have been associated with a poorer prognosis in bladder cancer and represent promising therapeutic targets to improve outcomes. Dr. Fradet’s team identified immunogenic peptides of the MAGE protein family member A9 (MAGE-A9), a protein which is frequently expressed in various human cancers including bladder, lung, and kidney. These peptides will then be combined with selected immunogenic peptides from the survivin protein. Researchers believe that the formulation of MAGE-A9 and survivin peptides with the DPX platform can generate a sustained, dual targeted T cell response that has the potential to destroy tumors with limited off-target events. Dr. Fradet and his team will evaluate DPX-SurMAGE in an initial clinical application in non-muscle invasive and muscle invasive bladder cancer.

IMV and the CHUQ have successfully completed preclinical evaluations which support the clinical development of DPX-SurMAGE in two distinct Phase 1 studies:

DPX-SurMAGE with or without CPA prior to transurethral resection of recurrent low-grade or high-grade non-muscle invasive bladder cancer
DPX-SurMAGE, CPA and anti-PD-1 for the treatment of muscle invasive bladder cancer prior and after cystectomy.
Based on the current timeline, IMV anticipates that the Phase 1 clinical study will be initiated in subjects with non-muscle invasive cancer in the second half of this year.

DPX-COVID-19: A DPX-Based Vaccine Candidate Against SARS-CoV-2

Due to the evolution of the regulatory landscape and at the request of the Canadian regulatory authorities the Company is currently conducting complementary preclinical studies, including evaluating the impact of new variants, and will provide an update once these preclinical studies are completed.

Changes in Board of Directors and Management

Appointment of Mr. Kyle Kuvalanka to the Board of Directors

IMV recently announced the appointment of Mr. Kyle Kuvalanka to the Board of Directors on April 1, 2021. Bringing over 20 years of experience as a senior leader in the biopharmaceutical industry, Mr. Kuvalanka has a successful track record in forming and negotiating strategic collaborations, leading financings, facilitating strategy development, as well as building and directing business and finance functions. Currently, Mr. Kuvalanka serves as Chief Financial Officer and Chief Operating Officer at Goldfinch Bio, a kidney precision medicines company.

Appointment of Dr. Michael Kalos to the Board of Directors

On May 11, 2021, IMV also announced the appointment of Michael Kalos, Ph.D. to its Board of Directors effective May 11, 2021. Dr. Kalos is an internationally recognized expert in T cell therapy and immunotherapy. He brings over 25 years of experience and expertise in cell therapy and immuno-oncology. In his career, Mr. Kalos served as Vice President of Immuno-oncology and Oncology Cell Therapies at Janssen and as Chief Scientific Officer of immuno-oncology at Eli Lilly.

Wayne Pisano, who has served on IMV’s Board of Directors since October 2011, retired at the end of the quarter. James Hall will be retiring from the Board of Directors after the annual general meeting in June 2021. James served as a board member for more than 11 years. Andy Sheldon, Board Chairman of IMV, commented: "We are very grateful to Wayne and James for more than 10 years of dedicated service to IMV. On behalf of my fellow board members and IMV’s management team, I would like to thank them for their long service and important contribution to the Company."

Departure of Dr. Joanne Schindler

Dr. Joanne Schindler gave her resignation as Chief Medical Officer (CMO) for personal reasons, effective June 11, 2021. Over the next weeks, Dr. Schindler will transition responsibilities while remaining fully empowered in her role as IMV’s Chief Medical Officer. The Company is actively working with a recruitment firm to hire a new CMO who will drive maveropepimut-S towards registration. Fred Ors, CEO, commented: "We thank Joanne for her contribution during her tenure and wish her the best in the future."

Overview of First quarter 2021 Financial Results

All dollar amounts noted herein are denominated in United States dollars (unless otherwise noted herein).

As of March 31, 2021, the Company had cash and cash equivalents of $30.5 million and working capital of $31.6 million, compared with $36.3 million and $35.6 million, respectively as of December 31, 2020. Based on its current operating plan and not considering the $47.7 million remaining under the $50 million At-The-Market facility executed in October 2020, IMV expects its current cash position will be sufficient to fund operations until Q1 2022.

Research and development expenses were $4.7 million for the three months ended March 31, 2021 compared with $5.1 million for the three months ended March 31, 2020. This decrease of $400,000 was mainly due to a decrease in expenses related to the ongoing basket trial and the timing of manufacturing activities for DPX-Survivac and DPX-SurMAGE, partly offset by an increase in personnel costs due to an increase in headcount, and pre-clinical development of DPX-COVID-19, which was offset by the increase in government assistance described below.

General and administrative expenses were $3.2 million for the three months ended March 31, 2021 compared with $2.3 million for the three months ended March 31, 2020. This increase of $900,000 was mainly attributable to an increase in insurance premium following rate increases in mid-2020.

Government assistance totaled $1.2 million for the three months ended March 31, 2021 compared with $415,000 in Q1 2020. This increase is mainly explained by various government grants for the development of DPX-COVID-19, reimbursed for eligible development expenditures incurred to date.

The net loss and comprehensive loss of $7.0 million ($0.10 per share) for the three months ended March 31, 2021 was $200,000 lower than the net loss and comprehensive loss of $7.2 million ($0.14 per share) for the three months ended March 31, 2020.

As of May 11, 2021, the number of issued and outstanding common shares was 67,795,933 and a total of 5,072,928 stock options, warrants and deferred share units were outstanding.

The Corporation’s audited annual consolidated results of operations, financial condition and cash flows for the year ended December 31, 2020 and the related management’s discussion and analysis (MD&A) are available on SEDAR at www.sedar.com and on EDGAR at www.sec.gov/edgar as well as the Company’s website at View Source

Selected Upcoming Milestones

Maveropepimut-S

Q2 2021: Beginning of Phase 2B DLBCL trial
Q2 2021: Translational and biomarker clinical update for ovarian cancer
Q3 2021: Initiation of investigator-led study in breast cancer
H2 2021: Meeting with FDA and final design for next clinical study in ovarian cancer
H2 2021: Clinical update basket trial
H1 2022: Clinical update Phase 2B DLBCL trial
DPX-SurMAGE

H2 2021: Initiation of a Phase 1 clinical study in bladder cancer
Conference Call and Webcast Information

Management will host a conference call and webcast today May 12, 2021 at 8:00 a.m. ET. Investment professionals are invited to join the conference call by dialing (866) 211-3204 (U.S. and Canada) or (647) 689-6600 (international) using the conference ID# 9284231

Other interested parties can access the live audio webcast at this link: View Source

On May 12, 2021 EDAP TMS SA (Nasdaq: EDAP) (the "Company"), a global leader in robotic energy-based therapies, reported that unaudited financial results for the first quarter of 2021 and provided an update on strategic and operational developments (Press release, EDAP TMS, MAY 12, 2021, View Source [SID1234579810]).

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Marc Oczachowski, EDAP’s Chairman and Chief Executive Officer, said: "We are thrilled to have completed a successful offering in April which, added to our existing strong cash position, provides funding to achieve potentially value-creating milestones across all aspects of our business. We are acutely focused on driving accelerating growth in the US by building market access and coverage now that our Category 1 CPT code is in place. We also continue to advance our Phase 2 endometriosis program and have now opened a second trial site in France where we commenced patient treatments last week.

"While we continue to experience some effect of COVID-19 on our operations, we are pleased with the global performance of our business as compared to the first quarter of last year. We grew revenue by 35.4%, expanded our gross margin to 42.4%, and generated a profitable quarter for the company. We also saw continued growth in our Focal One and HIFU treatments in the US, reflecting increasing awareness and adoption of our next generation HIFU technology."

"In summary, with our strong balance sheet and US growth initiatives continuing to gain traction, I believe we are well positioned to drive growing adoption of HIFU for the management of prostate cancer in 2021," Mr. Oczachowski concluded.

First Quarter 2021 Results

Total revenue for the first quarter 2021 was EUR 10.3 million (USD 12.4 million), an increase of 35.4% compared to total revenue of EUR 7.6 million (USD 8.4 million) for the same period in 2020.

Total revenue in the HIFU business for the first quarter 2021 was EUR 1.8 million (USD 2.2 million), a decline of 6.2% as compared to EUR 1.9 million (USD 2.1 million) for the first quarter of 2020.

Total revenue in the LITHO business for the first quarter 2021 was EUR 2.9 million (USD 3.5 million), roughly flat with EUR 2.9 million (USD 3.2 million) for the first quarter of 2020.

Total revenue in the Distribution business for the first quarter 2021 was EUR 5.6 million (USD 6.7 million), a 102.5 % increase compared to EUR 2.8 million (USD 3.1 million) for the first quarter of 2020.

Gross profit for the first quarter 2021 was EUR 4.4 million (USD 5.2 million), compared to EUR 3.1 million (USD 3.4 million) for the year-ago period. Gross profit margin on net sales was 42.4% in the first quarter of 2021, compared to 40.2% in the year-ago period. The increase in gross profit year-over-year was due to higher sales effect on fixed costs.

Operating expenses were EUR 4.1 million (USD 5.0 million) for the first quarter of 2021, compared to EUR 4.5 million (USD 5.0 million) for the same period in 2020.

Operating profit for the first quarter of 2021 was EUR 0.2 million (USD 0.3 million), compared to an operating loss of EUR 1.5 million (USD 1.6 million) in the first quarter of 2020.

Net income for the first quarter of 2021 was EUR 0.8 million (USD 0.9 million), or EUR 0.03 per diluted share, as compared to a net loss of EUR 1.3 million (USD 1.4 million), or EUR (0.04) per diluted share in the year-ago period.

As of March 31, 2021, the company held cash and cash equivalents of EUR 24.4 million (USD 28.6 million), as compared to EUR 24.7 million (USD 30.2 million) as of December 31, 2020. Subsequent to the end of the first quarter, the completed an underwritten public offering of its American Depository Shares that yielded gross proceeds of approximately $28 million.

Conference Call

An accompanying conference call and webcast will be conducted by management to review the results. The call will be held at 8:30am EDT tomorrow, May 12, 2021. Please refer to the information below for conference call dial-in information and webcast registration.

On May 12, 2021 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a biopharmaceutical company focused on advancing XCART, a personalized CAR T platform technology engineered to target patient- and tumor-specific neoantigens, reported its financial results for the first quarter of 2021 and provided a corporate update (Press release, Xenetic Biosciences, MAY 12, 2021, View Source [SID1234579807]).

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"During the first quarter our focus remained on advancing the development of our XCART platform, which we believe has the potential to provide a personalized CAR T therapy targeting cancers with a patient-and tumor-specific approach. The commencement of our exploratory patient biopsy trial is a key component of our preclinical development strategy and an important step forward toward advancing into a Phase 1 study," commented Jeffrey Eisenberg, Chief Executive Officer of Xenetic.

XCARTTM Platform Technology Overview: Significantly differentiated, proprietary approach to personalized CAR T lymphoma therapy targeting tumor-specific antigens independently of CD19 or other surface antigens that are common to both normal and malignant B-cells. Lead program for Non-Hodgkin lymphoma, an area of significant unmet need, with the potential to address an initial global market opportunity of over $7 billion annually.[1]

Program Highlights:

Collaboration with Pharmsynthez and multiple academic institutions in Eastern Europe to optimize the overall XCART workflow, including clinical manufacturing processes, and ultimately to conduct a first in human study in B-cell Non-Hodgkin lymphoma (NHL) patients.
Research and development collaboration with Scripps Research covering design and implementation of the preclinical development program, as well as method development activities supporting process development for clinical manufacturing.
Recently commenced exploratory patient biopsy study in Eastern Europe. When sufficient experience is gained through this exploratory study, the collaborations being leveraged in the XCART development program may be expanded to include development and qualification of manufacturing processes for producing XCART-designed, tumor-specific autologous CAR T cells. The work being performed under these collaborations is expected to position the Company to conduct IND-enabling studies in the United States.
Upcoming Potential Milestones

Seeking U.S. FDA INTERACT meeting.
Initiating process development for clinical CAR T manufacturing.
PolyXen Platform Technology: Patent-protected platform technology designed for protein or peptide therapeutics, enabling next-generation biological drugs by prolonging a drug’s circulating half-life and potentially improving other pharmacological properties.

Program Highlights:

Exclusive License Agreement with Takeda Pharmaceuticals Co. Ltd. ("Takeda") in the field of blood coagulation disorders.
Takeda currently has one active development program underway.
Royalty payments of approximately $0.2 million were received in the quarter ended March 31, 2021, as Takeda’s sublicensee has now launched the relevant product in multiple global markets.
Company’s partner, Pharmsynthez, announced positive Phase 3 trial results and filed a registration dossier in Russia to obtain approval of Epolong, a polysialylated form of human erythropoietin as a treatment for anemia in patients with chronic kidney disease.
Summary of Financial Results for First Quarter 2021

Net loss for the quarter ended March 31, 2021 was approximately $1.3 million. Research & development expenses for the three months ended March 31, 2021 increased by approximately $0.3 million, or 75.1%, to $0.6 million from $0.4 million in the comparable quarter in 2020. The increase was due to the Company’s increase on spending for the XCART platform technology. General and administrative expenses for the three months ended March 31, 2021 and 2020 were approximately $0.9 million. Increases in consulting and employee related costs during the three months ended March 31, 2021 compared to the same period in 2020 were substantially offset by lower share-based expense and legal and accounting costs. At March 31, 2021, the Company reported working capital was approximately $10.2 million. Working capital decreased by $1.2 million from December 31, 2020 due to the Company’s net loss for the three months ended March 31, 2021. The Company ended the quarter with approximately $10.0 million of cash.

On May 12, 2021 Sierra Oncology, Inc. (SRRA), a late-stage biopharmaceutical company on a quest to deliver targeted therapies that treat rare forms of cancer, reported abstracts on new analyses from the previously completed SIMPLIFY-1 and SIMPLIFY-2 studies have been accepted into the program for the 2021 Annual Meeting of the European Hematology Association (EHA) (Free EHA Whitepaper) (Press release, Sierra Oncology, MAY 12, 2021, View Source [SID1234579805]). An abstract highlighting the association between transfusion independence and improved overall survival in myelofibrosis patients treated with momelotinib has been selected for oral presentation.

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"Overall survival remains the gold standard benchmark for oncology therapies. We are excited to showcase these new analyses in an oral presentation demonstrating a strong correlation between achieving or maintaining transfusion independence at Week 24 and overall survival for momelotinib patients in SIMPLIFY-1. A similar trend was seen in SIMPLIFY-2," said Barbara Klencke, MD, Chief Development Officer of Sierra. "Higher rates of transfusion independence at Week 24 were observed in anemic patients receiving momelotinib, regardless of the degree of baseline anemia, and in all subgroups of patients when analyzed either by baseline platelet count or baseline transfusion status as we describe in a separate accepted poster. Together, these data support the potential long-term benefit of achieving or maintaining transfusion independence by inhibiting ACVR1 / ALK2 in addition to JAK1 and JAK2 with momelotinib in myelofibrosis patients, regardless of baseline platelet count, transfusion status or degree of anemia. We look forward to the presentation of the full data sets at the EHA (Free EHA Whitepaper) annual meeting next month."

Transfusion Independence is Associated with Improved Overall Survival in Myelofibrosis Patients Receiving Momelotinib

Efficacy data examining the association between transfusion independence and overall survival for momelotinib patients from SIMPLIFY-1 (JAKi-naïve) and SIMPLIFY-2 (JAKi-exposed) will be presented in an oral presentation by Ruben Mesa, MD, Director of the Mays Cancer Center, UT Health San Antonio, MD Anderson Cancer Center.

Previously published data from both SIMPLIFY studies demonstrate robust overall survival for momelotinib-treated patients (median not yet reached in SIMPLIFY-1 and 34.3 months in SIMPLIFY-2). Additionally, previously reported week 24 transfusion independence rates were higher in the momelotinib arms of SIMPLIFY-1 (67% vs. 49%) and SIMPLIFY-2 (43% vs. 21%). The new analyses suggest JAKi-naïve patients receiving momelotinib who maintain or achieve transfusion independence at week 24 have favorable overall survival compared to non-responders, with a similar trend observed in SIMPLIFY-2.

Presentation Details

Abstract: S202
Title: Transfusion Independence is Associated with Improved Overall Survival in Myelofibrosis Patients Receiving Momelotinib
Presenter: Ruben Mesa, MD, Director of the Mays Cancer Center, UT Health San Antonio, MD Anderson Cancer Center
Session Title: Novel Therapies and Targets in MPN
Virtual Presentation Available: Friday, June 11, 2021 beginning at 9:00 am CEST
Live Q&A Discussion: Wednesday, June 16, 2021, 4:00 – 4:45 pm CEST

Improved Transfusion Independence Rates for Momelotinib vs. Ruxolitinib in Anemic JAKi Naïve Myelofibrosis Patients Independent of Baseline Platelet or Transfusion Status

Progressive anemia is a common occurrence in myelofibrosis, with nearly all patients requiring transfusions as their disease advances. As described above, the analyses in Abstract S202 show that JAKi-naïve patients receiving momelotinib who maintain or achieve transfusion independence at week 24 have favorable overall survival compared to non-responders, with a similar trend observed in SIMPLIFY-2. Therefore, it is important to understand which patients are most likely to achieve transfusion independence at week 24.

Abstract EP1081 will highlight new analyses from SIMPLIFY-1 to demonstrate that the prognostically important week 24 transfusion independence rates in JAK inhibitor-naïve myelofibrosis patients were consistently higher in anemic patients receiving momelotinib compared to ruxolitinib, regardless of the platelet count, transfusion status or degree of anemia at baseline. Data to be featured further support the potential benefits of inhibiting ACVR1 / ALK2 in addition to JAK1 and JAK2 in myelofibrosis patients, and will be presented by Jean-Jacques Kiladjian, MD, PhD, Professor of Clinical Pharmacology, Paris Diderot University; Consultant Hematologist, Head, Clinical Investigation Center, Saint Louis Hospital, Paris, France.

Presentation Details

Abstract: EP1081
Title: Improved Transfusion Independence Rates for Momelotinib vs. Ruxolitinib in Anemic JAKi Naïve Myelofibrosis Patients Independent of Baseline Platelet or Transfusion Status
Presenter: Jean-Jacques Kiladjian, MD, PhD
Session Name: Myeloproliferative Neoplasms – Clinical
Virtual Presentation Available: Friday, June 11, 2021 beginning at 9:00 am CEST