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

On May 12, 2021 Sensei Biotherapeutics, Inc. (NASDAQ: SNSE), a clinical-stage immunotherapy company focused on the discovery and development of next generation therapeutics for cancer, reported financial results for the first quarter ended March 31, 2021 and provided a business update (Press release, Sensei Biotherapeutics, MAY 12, 2021, View Source [SID1234579804]).

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"The first quarter was a productive time for Sensei with the successful execution of our IPO, continued progress with our pipeline, and expansion of our headcount to support anticipated growth," said John Celebi, President and Chief Executive Officer of Sensei Biotherapeutics. "As we approach several near-term events, we continue to believe in the strength of our proprietary ImmunoPhage platform to potentially generate multiple immune activating therapeutics and our monoclonal antibody and nanobody platform targeted to key immune pathways. We are looking forward to the presentation of more mature data from the Phase 1/2 clinical trial of SNS-301 at ASCO (Free ASCO Whitepaper), and to further explore the potential breadth and depth of our programs in collaboration with leading researchers. With the proceeds raised from our recent IPO, we are well capitalized to advance our current programs and our drug discovery operations."

Sensei Biotherapeutics is developing a pipeline of medicines designed to fulfill the substantial potential of the immune system to defeat cancer and other diseases. The company has developed two unique approaches – the ImmunoPhage platform, which leverages its proprietary knowledge and expertise in bacteriophage (widespread viruses that infect bacteria but not mammalian cells) immunology, and its monoclonal antibody and nanobody platform, which are comprised of unique human monoclonal antibodies and alpaca-derived nanobodies that are selectively active in the tumor microenvironment. Together, these platforms are designed to create a new class of personalized immuno-oncology medicines.

First Quarter and Recent Highlights:

Strengthened Immuno-Oncology Advisory Board and Board of Directors – In May 2021, Sensei announced that it appointed Maura Gillison, M.D., Ph.D., Professor of Medicine, Thoracic/Head and Neck Medical Oncology at MD Anderson Cancer Center, and Richard Ulevitch, Ph.D., Professor of Immunology and Chairman Emeritus at The Scripps Research Institute to its Immuno-Oncology Advisory Board. In April 2021, Sensei appointed Jessie M. English, Ph.D. to its Board of Directors.
Completed Upsized Initial Public Offering (IPO)– In February 2021, Sensei completed its initial public offering of 8,030,295 shares of common stock, inclusive of the exercise by the underwriters of their option to purchase 1,030,243 shares, at a public offering price of $19.00 per share. Gross proceeds were $152.6 million.
Anticipated Pipeline Milestones and Events:

ImmunoPhage Platform Updates

SNS-301: a first-in-class, bacteriophage-based immune activating agent targeting human aspartate β-hydroxylase (ASPH), a tumor associated antigen overexpressed in multiple tumor types – SNS-301 is being evaluated in a signal-seeking Phase 1/2 study in combination with pembrolizumab. The study is designed to have two cohorts and enroll 60 patients with locally advanced unresectable or metastatic SCCHN who did not achieve a response to prior treatment with PD-1 blockade or patients who did not receive prior therapy with PD-1 blockade. The company expects to add a third cohort by the end of 2021 to evaluate SNS-301 with HPV-specific E6/E7 ImmunoPhage.

American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 conference: An abstract titled: "Update on Safety and Efficacy of a Phase 1/2 Study of SNS-301 and Pembrolizumab in Patients with Advanced Squamous Cell Carcinoma of the Head and Neck (SCCHN)" has been accepted for poster presentation at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting taking place virtually from June 4-8, 2021.
Phase 1/2 Topline Data Readout on Track: Sensei remains on track to report a substantial subset of data from the Phase 1/2 study by the end of 2021.
Enrollment Ongoing in Frontline Therapy Trial: Sensei announced that patient dosing has been initiated in the second cohort of the Phase 1/2 study evaluating SNS-301 as a first-line therapy in combination with pembrolizumab. The company continues to evaluate early enrollment trends in this cohort and the impact of COVID-19 on this clinical program.
Evaluation of New Opportunities for SNS-301 in the Neoadjuvant Setting: Sensei’s current Phase 1/2 studies in head and neck cancer are focusing only on SNS-301 in combination with pembrolizumab (Keytruda). Sensei has concluded its clinical trial collaboration with AstraZeneca for durvalumab (Imfinzi). Sensei is currently evaluating additional opportunities to study SNS-301 in the neoadjuvant setting following the results in February from AstraZeneca’s Phase 3 KESTREL trial of durvalumab for the treatment of first line recurrent or metastatic head and neck squamous cell carcinoma.
SNS-401: a first-in-class personalized ImmunoPhage cocktail being developed in collaboration with the University of Washington – SNS-401 is in preclinical development for the treatment of Merkel Cell Carcinoma, an aggressive form of skin cancer commonly caused by the Merkel Cell Carcinoma Polyoma Virus.

IND submission planned for first half of 2022: Sensei plans to submit an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) for its SNS-401 program in the first half of 2022.
ImmunoPhage Platform and Phortress Library – Phortress is Sensei’s proprietary library of ImmunoPhages, derived from antigens found across multiple patient populations and tumor types. Sensei has manufactured more than 25 ImmunoPhages and continues to add inventory to this "off-the-shelf" library as well as drive technologic innovations to the ImmunoPhage platform itself.

Monoclonal Antibody Platform Update

VISTA (V-domain Ig suppressor of T cell activation) is an immune checkpoint that inhibits anti-tumor immune responses – SNS-VISTA is in preclinical development in collaboration with Adimab LLC.

IND-Enabling Studies on Track: Sensei announced it is on track to initiate IND-enabling studies for its SNS-VISTA program by year-end 2021.
Corporate Update

Sensei Biotherapeutics announced that it will move its corporate headquarters to the Seaport Innovation District in Boston, MA. The new space more than doubles the size of the company’s current research and development footprint. The company’s manufacturing operations will remain in Rockville, MD.

First Quarter 2021 Financial Results

Cash Position – Cash and cash equivalents were $169.4 million as of March 31, 2021, as compared to $16.6 million as of December 31, 2020. Sensei expects the current cash balance to fund operations at least into the second half of 2023.

Research and Development (R&D) Expenses – R&D expenses were $3.4 million for the quarter ended March 31, 2021, compared to $2.2 million for the quarter ended March 31, 2020. The increase in R&D expenses was primarily attributable to increased headcount to support Sensei’s research, development, and manufacturing activities.

General and Administrative (G&A) Expenses – G&A expenses were $4.6 million for the quarter ended March 31, 2021, compared to $1.9 million for the quarter ended March 31, 2020. The increase was primarily attributable to higher personnel costs, including stock-based compensation expense, to support Sensei’s business.

Net Loss – Net loss was $8.0 million, for the quarter ended March 31, 2021, compared to $4.7 million for the quarter ended March 31, 2020.

KEYTRUDA is a registered trademark of Merck, and IMFINZI is a registered trademark of the AstraZeneca group of companies.

On May 12, 2021 Sanofi reported that New pivotal data and clinical analyses from portfolio of investigational therapies for immune-mediated rare blood disorders will be featured at the 26th Annual European Hematology Association (EHA) (Free EHA Whitepaper) Virtual Congress, June 9-17, 2021 (Press release, Sanofi, MAY 12, 2021, View Source [SID1234579803]).

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"The data being presented at EHA (Free EHA Whitepaper) 2021 demonstrate Sanofi’s commitment to providing first-in-class and potentially transformative treatments for people with immune-mediated rare blood disorders, where significant unmet needs persist," said Karin Knobe, Head of Development, Rare and Rare Blood Disorders, Sanofi. "We are excited to share pivotal data from the placebo-controlled CADENZA Phase 3 study of sutimlimab in people with CAD with no recent history of transfusion as well as interim data from our rilzabrutinib Phase 1/2 study in patients with ITP."

Working to break barriers in the treatment of cold agglutinin disease

Cold agglutinin disease (CAD) is a rare, chronic autoimmune hemolytic anemia that causes the body’s immune system to mistakenly attack healthy red blood cells and cause their destruction (hemolysis) via activation of the classical complement pathway. CAD patients may experience chronic anemia, profound fatigue, acute hemolytic crisis, and other potential complications, including an increased risk of thromboembolic events and early death.1,2,3 CAD impacts the lives of an estimated 12,000 people in the U.S., Europe, and Japan.4 Currently there are no approved therapies for CAD.

Sutimlimab is an investigational, potential first-in-class monoclonal antibody designed to selectively target C1-activated hemolysis in CAD.

New data from pivotal Phase 3 CADENZA and CARDINAL studies

Abstract #S291: C1s-Targeted Inhibition of Classical Complement Pathway by Sutimlimab in Cold Agglutinin Disease (CAD): Efficacy and Safety Results from the Randomized, Placebo (PBO)-Controlled Phase 3 CADENZA Study. Oral Presentation.
Abstract #S290: Sutimlimab, a Targeted Complement C1s Inhibitor, Improves Quality of Life (QOL) in Patients with Cold Agglutinin Disease (CAD): Results from the Randomized, Placebo-Controlled Phase 3 CADENZA Study. Oral Presentation.
Abstract #S312: Sustained Improvements in Patient-Reported Outcomes with Sutimlimab in Patients with Cold Agglutinin Disease: 1-Year Follow-Up Interim Results from the CARDINAL Study. Oral Presentation.
Abstract #EP1179: Clinically Important Change in FACIT-Fatigue Score for Patients with Cold Agglutinin Disease: An Analysis Using the Phase 3 CARDINAL and CADENZA Studies. Poster Presentation.
Abstract #EP689: Inhibition of Complement C1s with Sutimlimab in Patients with Cold Agglutinin Disease (CAD): 1-Year Interim Results of the Phase 3 CARDINAL Study Long-term Follow-up (adaptation). Poster Presentation.

Data on the potential impact of sutimlimab on QOL and fatigue in people with CAD

Abstract #EP709: Inflammation and Fatigue in Patients with Cold Agglutinin Disease (CAD): Analysis from the Phase 3 CARDINAL Study (adaptation). Poster Presentation.

New analyses on disease burden and healthcare resource utilization by people living with CAD

Abstract #EP1180: Increased Antidepressant Use Among Newly Diagnosed Patients with Cold Agglutinin Disease Compared with Other Patients in a Large US Healthcare System. Poster Presentation.
Abstract #EP1193: Healthcare Resource Utilization Among Patients with Cold Agglutinin Disease in Denmark (adaption). Poster Presentation.
Aiming to address unmet needs in immune thrombocytopenic purpura

Immune thrombocytopenic purpura (ITP) is an acquired autoimmune blood disorder characterized by immune-mediated platelet destruction and impairment of platelet production, which leads to thrombocytopenia, a predisposition to bleeding, and altered quality of life for patients. There remains a need in relapsed/refractory ITP for patients failing to maintain their platelet counts with the current therapies.

Rilzabrutinib is an investigational peripheral Bruton’s tyrosine kinase (BTK) inhibitor with Tailored Covalency technology intended to treat ITP.

Abstract #S299: Phase I/II Updated Safety and Efficacy Results of Oral Bruton Tyrosine Kinase (BTK) Inhibitor Rilzabrutinib in Relapsed/Refractory Immune Thrombocytopenia (ITP). Oral Presentation.
Abstract #PB1733: Phase III Multicenter, Double-Blind, Randomized, Placebo-Controlled Study of Rilzabrutinib, Oral BTK Inhibitor, in Adults and Adolescents with Persistent or Chronic Immune Thrombocytopenia. Abstract-only.
Additional presentation

Abstract #EP1145: Epidemiology, Treatment Patterns, and Clinical Outcomes Among Patients with Acquired Thrombotic Thrombocytopenic Purpura (aTTP) in the United States: an Electronic Health Records Analysis

Editor’s Note:
About sutimlimab: Sutimlimab is an investigational, humanized monoclonal antibody that is designed to selectively target and inhibit C1s in the classical complement pathway, which is part of the innate immune system. By blocking C1s, sutimlimab inhibits the activation of the classical complement pathway with the goal of halting C1-activated hemolysis in CAD to prevent the abnormal destruction of healthy red blood cells. By selectively inhibiting the classical pathway upstream at C1s, sutimlimab does not inhibit the lectin and alternative complement pathways.

Sutimlimab has been granted Breakthrough Therapy by the U.S. Food and Drug Administration (FDA) and Orphan Drug status by the FDA, European Medicines Agency and the Pharmaceuticals and Medical Devices Agency in Japan. Sutimlimab is currently under clinical investigation and has not been approved by any regulatory authority.

About rilzabrutinib: Rilzabrutinib is an oral, peripheral Bruton’s tyrosine kinase inhibitor with Tailored Covalency technology being investigated for the treatment of immune-mediated diseases, including immune thrombocytopenic purpura. BTK is involved in innate and adaptive immune responses and is a signalling molecule in immune mediated diseases. Rilzabrutinib has the potential to target the underlying disease pathogenesis and has not been shown to alter platelet aggregation5. The clinical significance of these mechanisms is currently under investigation and have not been approved by any regulatory authority. A Phase 3 clinical trial to evaluate rilzabrutinib for the treatment of ITP is currently underway with the first patient dosed in April 2021. Rilzabrutinib has been granted orphan drug designation and fast track designation by the U.S. FDA for ITP.

Rilzabrutinib is also being investigated in a Phase 3 trial for pemphigus, an immune- mediated disease characterized by blisters in mucous membranes and skin as well as a Phase 2 study in the autoimmune condition IgG4 disease. Three additional Phase 2 studies in immunological diseases are planned to start in 2021.

_______________________________

*Tailored Covalency is a registered trademark of Principia Biopharma Inc., a Sanofi company

Broome C, et al. Increased risk of thrombotic events in cold agglutinin disease: A 10-year retrospective analysis. Res Pract Thromb Haemost. 2020;00:1–8.
Quentin A. Hill, Rajeshwari Punekar, Jaime Morales Arias, Catherine M Broome, Jun Su; Mortality Among Patients with Cold Agglutinin Disease in the United States: An Electronic Health Record (EHR)-Based Analysis. Blood 2019; 134 (Supplement_1): 4790.
Lauren C. Bylsma, Anne Gulbech Ording, Adam Rosenthal, Buket Öztürk, Jon P. Fryzek, Jaime Morales Arias, Alexander Röth, Sigbjørn Berentsen; Occurrence, thromboembolic risk, and mortality in Danish patients with cold agglutinin disease. Blood Adv 2019; 3 (20): 2980–2985.
Berentsen S, et al. Haematologica. 2006;91(4):460-466
Langrish CL et al – Preclinical Efficacy and Anti-Inflammatory Mechanisms of Action of the Bruton Tyrosine Kinase Inhibitor Rilzabrutinib for Immune-Mediated Disease – J Immunol published online 5 March 2021