Background: Immune thrombocytopenia (ITP) is characterized by immune-mediated platelet destruction and impairment of platelet production, leading to downstream thrombocytopenia, a predisposition to bleeding, and adverse impact on patient quality of life. Unmet needs in relapsed or refractory ITP are to improve remission rates and durability through targeting underlying disease mechanisms. PRN1008 is an oral, reversible, covalent inhibitor of Bruton tyrosine kinase (BTK) that modulates immune-mediated processes in ITP. Preclinical PRN1008 data showed inhibition of B-cell receptor-mediated activation of human B cells, Fc receptor (Fc-gamma and Fc-epsilon)-mediated activation of immune cells, and dose-dependent reduction in platelet loss in a mouse ITP model. In platelets from normal healthy volunteer and ITP patients, clinically-relevant concentrations of PRN1008 showed no platelet aggregation or interference with other platelet agonists, in contrast to ibrutinib (Langrish et al. ASH 2017:1052). Methods: This is an ongoing open-label, adaptive, intra-patient dose-escalation, phase I/II study of PRN1008 in adult patients with relapsed or refractory ITP (primary or secondary) who previously responded to ≥ 1 prior ITP therapy and have no available therapeutic options (NCT03395210). Eligible patients have two platelet counts < 30,000/µL within 15 days prior to treatment. Oral PRN1008 starting doses were 200 mg QD, 400 mg QD, 300 mg BID (total 600 mg daily), and 400 mg BID (total 800 mg daily), with intra-patient dose escalation allowed every 4 weeks (maximum 400 mg BID) as needed for efficacy. Stable doses of concomitant corticosteroids and thrombopoietin-receptor agonists (TPO-RA) are permitted. The primary end point is the proportion of patients with ≥ 2 consecutive platelet counts (separated by ≥ 5 days) of ≥ 50,000/µL and increased by ≥ 20,000/µL from baseline without requiring rescue medication. Results: A total of 21 patients have been enrolled to date at starting doses of 200 mg QD (n=9), 400 mg QD (n=1), 300 mg BID (n=5), and 400 mg BID (n=6). As of 15 July 2019 data cut-off, 11 patients were receiving ongoing treatment, 4 completed the study, and 6 patients withdrew (2 due to patient decision, 2 from non-treatment-related adverse events [AEs], 1 erroneously enrolled, and 1 because of rescue medication use). Patients had a median age of 54 y (range, 30-65), 4 (19%) had a prior splenectomy, 19 (90%) were diagnosed with primary ITP, and 2 (10%) with secondary ITP. Patients had ITP for a median of 8.3 years (range, 0.5-42.4) and had received a median of 4 prior ITP therapies. Median platelet count at study entry was 14,173/µL (range, 2,670-27,000/µL). During the study, 6 (29%) patients received PRN1008 monotherapy; 15 (71%) patients were on ≥ 1 concomitant ITP medication. Related treatment-emergent AEs (TEAEs) were reported by 4 (19%) patients; all were grade 1 or 2. The most frequent related TEAEs were nausea, diarrhea, and abdominal distension. There were no treatment-related bleeding or thrombotic events, and no significant changes in the ITP-BAT bleeding scale between baseline and the last visit. There were no dose limiting toxicities (DLT). Patients had received treatment for a median of 10.1 weeks (range, 0.1-31.0). Overall, 7 (33%) patients achieved the primary endpoint across all doses (Table). Patient responses were improved at the 2 higher doses. In 10 patients who had reached ≥ 12 weeks of treatment, ≥ 50% of patients had platelet counts of ≥ 50,000/µL and ≥ 20,000/µL increases from baseline. Conclusion: Overall, PRN1008 was active in 33% of ITP patients who were refractory to multiple treatments with no alternative therapeutic options. This result was demonstrated despite the limited duration of treatment and including patients at all dose levels. In addition, patients treated for longer periods of time have substantially improved response rates that support continued interest in this ongoing study. The safety profile was tolerable at all studied doses whether given as a monotherapy or with allowed concomitant ITP therapy. Importantly, TEAEs were grade 1 or 2 with no thrombotic events. The dose-escalation portion of the study is complete; enrollment is expanding at the 400 mg BID starting dose for a duration of 24 weeks to further characterize treatment benefit and for continued treatment beyond 24 weeks in patients who have responded. Disclosures Kuter: Dova: Consultancy, Honoraria; Kyowa-Kirin: Consultancy, Honoraria; Caremark: Consultancy, Honoraria; Pfizer: Consultancy, Honoraria; Kezar: Research Funding; Argenx: Consultancy, Honoraria, Research Funding; Novartis: Consultancy, Honoraria; Platelet Disorder Support Association: Consultancy, Honoraria; Principia: Consultancy, Honoraria, Research Funding; Alnylam: Consultancy, Honoraria, Research Funding; Bristol Myers Squibb (BMS): Consultancy, Honoraria, Research Funding; Agios: Consultancy, Honoraria, Research Funding; Sanofi: Consultancy, Honoraria; Genzyme: Consultancy, Honoraria; Shinogi: Consultancy, Honoraria; Shire: Consultancy, Honoraria; Merck Sharp Dohme: Consultancy, Honoraria; Momenta: Consultancy, Honoraria; Protalex: Consultancy, Honoraria, Research Funding; Protalix: Consultancy, Honoraria; Rigel: Consultancy, Honoraria, Research Funding; Takeda (Bioverativ): Consultancy, Honoraria, Research Funding; UCB: Consultancy, Honoraria; Up-to-Date: Consultancy, Honoraria, Patents & Royalties: 3 Up-to-Date chapters; Zafgen: Consultancy, Honoraria; Daiichi Sankyo: Consultancy, Honoraria; Actelion (Syntimmune): Consultancy, Honoraria, Research Funding; Amgen: Consultancy, Honoraria, Research Funding. Boccia:AstraZeneca: Speakers Bureau; Celgene: Speakers Bureau; Amgen: Speakers Bureau; AMAG: Consultancy; Genentech: Speakers Bureau; DSI: Speakers Bureau. Lee:Weill Cornell Medical College: Employment. Tzvetkov:UMHAT Georgi Stranski: Employment; DCC Pleven: Consultancy. Mayer:AOP Orphan Pharmaceuticals AG: Research Funding. Trněný:Abbvie: Consultancy, Honoraria; Gilead Sciences: Consultancy, Honoraria; Takeda: Consultancy, Honoraria; Bristol-Myers Squibb: Consultancy, Honoraria; MorphoSys: Consultancy, Honoraria; Incyte: Consultancy, Honoraria; Janssen: Consultancy, Honoraria; Celgene: Consultancy; F. Hoffmann-La Roche: Consultancy, Honoraria; Amgen: Consultancy, Honoraria. Kostal:Novartis: Honoraria; AOP: Honoraria; University Hospital in Hradec Kralove, Czech Republic: Employment. Hajek:Janssen: Consultancy, Honoraria, Research Funding; Amgen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Celgene: Consultancy, Honoraria, Research Funding; AbbVie: Consultancy; BMS: Consultancy, Honoraria, Research Funding; Novartis: Consultancy, Research Funding; PharmaMar: Consultancy, Honoraria; Takeda: Consultancy, Honoraria, Research Funding, Speakers Bureau. McDonald:Bayer: Honoraria; Amgen: Honoraria; Novartis: Honoraria. Bandman:Principia Biopharma: Employment, Equity Ownership, Patents & Royalties: Institutional with Incyte and Portola, no royalties. Burns:Principia BioPharma: Employment. Neale:Principia BioPharma: Employment, Equity Ownership. Thomas:Principia Biopharma: Employment, Equity Ownership; BMS: Equity Ownership; Pfizer: Equity Ownership. Cooper:Principia: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Rigel: Consultancy, Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Amgen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees. Off Label Disclosure: Yes, this was an investigational clinical phase I/II study of PRN1008 in patients with relapsed/refractory ITP. Phase I dose escalation phase is now complete and expanded phase II studies ongoing.

Introduction: Rilzabrutinib is an oral, reversible, covalent inhibitor of Bruton tyrosine kinase (BTK) that targets underlying disease mechanisms of platelet destruction without inhibiting platelet aggregation (common with ibrutinib). The mechanisms of BTK inhibition provide a new approach for treating patients with immune thrombocytopenia (ITP). Completion of dose-escalation study phase determined that the minimally-effective dose was rilzabrutinib 400 mg given twice daily (BID; Kuter. ASH 2019). Methods: This open-label phase I/II study evaluated rilzabrutinib in adults with relapsed ITP who had at least two platelet counts <30×109/L in the 14 days prior to the first dose of rilzabrutinib, and included patients who had inadequate response to prior corticosteroids (CS)/thrombopoietin receptor agonists (TPO-RA) but were allowed to continue receiving stable doses of these medications. The primary endpoint was ≥2 consecutive platelet counts of ≥50×109/L and an increase of ≥20×109/L from baseline without requiring rescue medication. Subgroup analyses included assessing the impact of select prior therapies on reaching the primary endpoint, as well as the ability of rilzabrutinib to maintain durable responses and safety in the long-term extension (LTE) period. Results: As of May 5, 2020, 32 patients initiated treatment with rilzabrutinib 400 mg BID. The median baseline age was 50 years (range, 21-74) and 97% were classified as having primary ITP. Patients had a median baseline platelet count of 13×109/L, had ITP for a median duration of 7.3 y (range, 0.4-52.5), and were heavily pretreated with a median of 6 prior therapies (range, 1-53; 28% prior splenectomy). Median duration of rilzabrutinib 400 mg BID treatment was 18.0 wk (range, 1.4-24.6). Overall, 14/32 patients (44%) achieved the primary endpoint, and responders maintained platelet counts ≥50×109/L for a median of 71% (range, 33%-100%) of weekly counts. Primary endpoint responses were achieved despite prior splenectomy or lack of response to prior ITP therapies (Table). Independent of the primary response, 67% of all patients were able to achieve clinically meaningful benefit of platelet counts ≥30×109/L. Nine patients continued rilzabrutinib 400 mg BID into the LTE period for an additional median of 20 wk (range, 4-36) of treatment. Four of these LTE patients were on rilzabrutinib monotherapy and 5 on rilzabrutinib with concomitant ITP therapy (n=3 corticosteroids, n=2 romiplostim). Baseline characteristics for the 9 LTE patients were a median duration of ITP of 2.6 y (1.2-14.3) and a median of 5 prior therapies (range, 1-8; 1 patient had a prior splenectomy). Platelet counts of ≥50×109/L in the LTE period were maintained for a median of 100% (range, 36%-100%) of weekly counts (Figure). Treatment-related, treatment-emergent adverse events (TEAEs) were all grade 1/2 in patients initiating rilzabrutinib 400 mg BID; 1 patient each experienced grade 1 diarrhea and grade 1 hypophosphatemia in the LTE period. Conclusions: Oral rilzabrutinib treatment achieved clinically significant platelet responses (≥50×109/L) in patients with heavily pretreated ITP irrespective of splenectomy or lack of response to prior ITP therapy, and maintained responses for the majority of time. In addition, most patients (67%) achieved a clinically meaningful response (platelet counts ≥30×109/L). In patients treated beyond 6 months in the LTE, responses remained consistently reliable (median 100% of weeks). Rilzabrutinib was well tolerated with only grade 1/2 treatment-related TEAEs overall, with only 2 related grade 1 events observed in the LTE period. Continued study is warranted to further demonstrate the magnitude and durability of rilzabrutinib's clinical benefit. Disclosures Kuter: Zafgen: Consultancy, Honoraria; Takeda (Bioverativ): Consultancy, Honoraria, Other, Research Funding; Merck Sharp Dohme: Consultancy, Honoraria; Kyowa-Kirin: Consultancy, Honoraria; UCB: Consultancy, Honoraria; Platelet Disorder Support Association: Consultancy, Honoraria; Incyte: Consultancy, Honoraria; Shionogi: Consultancy, Honoraria; Dova: Consultancy, Honoraria; Shire: Consultancy, Honoraria; Sanofi (Genzyme): Consultancy, Honoraria; Protalix Biotherapeutics: Consultancy; Up-To-Date: Consultancy, Honoraria, Patents & Royalties; Protalex: Consultancy, Honoraria, Research Funding; Shionogi: Consultancy; Genzyme: Consultancy, Honoraria; Immunovant: Consultancy, Honoraria; Pfizer: Consultancy, Honoraria; Actelion (Syntimmune): Consultancy, Honoraria, Other: Travel Expenses, Research Funding; Protalex: Consultancy, Honoraria, Other, Research Funding; Rigel: Consultancy, Honoraria, Other, Research Funding; Daiichi Sankyo: Consultancy, Honoraria; Agios: Consultancy, Honoraria, Other: Travel Expenses, Research Funding; Caremark: Consultancy, Honoraria; CRICO: Consultancy, Honoraria; Kezar Life Sciences, Inc: Other, Research Funding; Principia Biopharma: Consultancy, Honoraria, Other, Research Funding; Amgen: Consultancy, Honoraria, Other: Travel Expenses, Research Funding; Alnylam: Consultancy, Honoraria, Other: Travel Expenses, Research Funding; Immunovant: Other: Travel Expenses, Research Funding; Principia: Consultancy, Research Funding; Momenta: Consultancy, Honoraria; Bristol-Myers Squibb: Consultancy, Honoraria, Other: Travel Expenses, Research Funding; Argenx: Consultancy, Honoraria, Other: Travel Expenses, Research Funding; Novartis: Consultancy, Honoraria. Efraim:UMHAT"ST.MARINA": Consultancy, Current Employment, Current equity holder in private company. Mayer:AbbVie: Research Funding; Principia Biopharma: Research Funding. McDonald:Bayer: Consultancy, Honoraria; Rigel: Research Funding; Novartis: Consultancy, Honoraria, Other: Travel Expenses; Amgen: Consultancy, Honoraria, Other: Travel Expenses. Bird:Sanofi: Consultancy, Other: (pls note personal honoraria declined for ad board); Principia Biopharma: Other: investigator in clinical studies; Amgen: Consultancy, Other: (pls note personal honoraria declined for all), Speakers Bureau; Novartis: Consultancy, Other: (pls note personal honoraria declined for all), Speakers Bureau; Rigel: Other: investigator in clinical studies; CSL-Behring: Other: investigator in clinical studies; Bristol-Myers Squibb Company: Other: investigator in clinical studies; Ablynx: Other: investigator in clinical studies. Regenbogen:Roche: Current equity holder in publicly-traded company; Gilead: Current equity holder in publicly-traded company; Amgen: Current equity holder in publicly-traded company; Moderna: Current equity holder in publicly-traded company; AbbVie: Current equity holder in publicly-traded company. Garg:Takeda: Consultancy. Kaplan:Celgene: Honoraria; Novartis: Honoraria. Bandman:Principia Biopharma: Current Employment. Burns:Principia Biopharma: Current Employment. Neale:Principia Biopharma: Current Employment. Thomas:Principia Biopharma: Current Employment, Current equity holder in publicly-traded company. Cooper:Novartis: Consultancy, Honoraria; Amgen: Consultancy, Honoraria; Rigel: Consultancy, Honoraria; Principia: Consultancy, Honoraria. OffLabel Disclosure: Rilzabrutinib is an investigational therapy being evaluated in a clinical study for the treatment of patients with immune thrombocytopenia.

INTRODUCTION: Hypomethylating agents have become a standard therapy for high-risk myelodysplastic syndromes (MDS) and elderly patients with acute myeloid leukemia (AML). AIM:The aim of the study was to assess the efficacy of azacitidine treatment in patients with MDS and AML followed for 18 months. MATERIALS AND METHODS: Twenty-seven patients with MDS and AML treated in theClinic of Hematology at St. Marina University Hospital, Varna were included in the study. Azacitidine was administered subcutaneously at a dose of 75 mg/m 2 for 7 days. Disease assessment was performed on the 3 rd month, 6 th month, and at progression. RESULTS: Twenty-seven patients were analyzed. Their median age was 71.5 years. Nine had refractory anemia with excess of blasts II (RAEB II), 5 had chronic myelomonocytic leukemia II (CMML II), 1 was with unclassifiable MDS (MDS-U), and 12 with AML. The median number of administered cycles was 6 (1-19). Eleven patients completed 6 cycles of azacitidine. Partial response was achieved in 9 patients (33%) (7 MDS and 2 AML), stable disease in 8 (29%) (5 MDS and 3 AML). Progressive disease was observed in 10 patients (37%). The response correlated with the type of the disease (p=0.03), cytogenetic risk (p=0.01), and survival (p=0.000). At 18 months, 60% of MDS patients were alive compared to 41.7% in the AML group. The median time to death in the AML patient group was 2.5 months. The mean overall survival was 10.4 months (12.6 months for MDS patients and 5.4 months for AML patients). CONCLUSION:The therapy with azacitidine is an option for elderly patients with high-risk MDS. In patients with AML a rapid progression is observed during the first two cycles with mortality rate of 58.3%. Scr Sci Med. 2018;50(1):31-35

Introduction: Key characteristics of immune thrombocytopenia (ITP) include immune-mediated platelet destruction/impaired production, with resultant thrombocytopenia and increased bleeding risk. Durable response to current therapies remains an unmet need, particularly in the relapsed/refractory setting. Rilzabrutinib is the first oral, reversible, covalent inhibitor of Bruton tyrosine kinase designed to target immune-mediated pathways in ITP without inhibiting normal platelet aggregation. Initial phase I/II results in ITP demonstrated rapid and durable efficacy with rilzabrutinib that was well-tolerated at all dose levels, including the optimal 400 mg bid dose. Interim results on rilzabrutinib effects in patients with relapsed/refractory ITP were previously reported. Here we present long-term data from a larger group of patients who initiated rilzabrutinib at 400 mg bid and are continuing in the long-term extension (LTE) period. Methods: This ongoing, global phase I/II study (NCT03395210) enrolled adult patients from 8 countries with relapsed or refractory ITP who previously responded to ≥1 prior ITP therapy. Eligible patients with 2 baseline platelet counts of <30×10 9/L no less than 7 days apart in the 15 days before baseline received oral rilzabrutinib at starting doses of 200 or 400 mg QD, 300 or 400 mg bid for 24 weeks; intrapatient dose escalation was permitted to improve efficacy. Stable doses of concomitant corticosteroids and thrombopoietin receptor agonists were allowed for patients with inadequate platelet response. The primary endpoints were safety and achievement of ≥2 consecutive platelet counts ≥50×10 9/L and increased ≥20×10 9/L from baseline without rescue medication. Responding patients could continue in the LTE period with rilzabrutinib at the optimal 400 mg bid dose. Results: As of June 1, 2021 in 60 patients, 45 patients initiated rilzabrutinib 400 mg bid and to date, 16 patients with durable, stable response proceeded to LTE at 400 mg bid. At enrollment for patients initiating rilzabrutinib 400 mg bid, median age was 49 y (range, 19-74), median duration of ITP was 6.1 y (range, 0.4-52.5), and median platelet count was 15×10 9/L (range, 2-33×10 9/L). Patients were heavily pretreated with a median of 6 prior treatment events (range, 1-53), including 24% with prior splenectomy. Overall, 18/45 patients (40%) achieved the primary endpoint. In primary responders, platelet counts of ≥30×10 9/L, ≥30×10 9/L with ≥20×10 9/L above baseline, and ≥50×10 9/L were maintained for a median of 95%, 86%, and 72% of weeks, respectively. Median time to first platelet counts of ≥30×10 9/L, ≥30×10 9/L with ≥20×10 9/L above baseline, and ≥50×10 9/L were 8.5 (7-134), 11.5 (7-134), and 12.5 (8-142) days, respectively. According to subgroup analyses, primary platelet responses were consistently >30% irrespective of baseline platelet counts, the use of concomitant therapy, duration of ITP, or number of prior therapies (Table). LTE patients received rilzabrutinib for an overall median duration of 462 days (range, 303-764). At LTE entry, patients had a median platelet count of 87×10 9/L (range, 16-321×10 9/L). In addition to all LTE patients achieving the primary endpoint during the main treatment period, these patients maintained platelet counts of ≥30×10 9/L, ≥30×10 9/L with ≥20×10 9/L above baseline, and ≥50×10 9/L, for a median of 100%, 96%, and 90%, of weeks, respectively, during the LTE period (Figure). Treatment-related treatment-emergent adverse events (TEAEs; all grade 1/2) were reported in 27/45 patients (60%); the most common were 36% diarrhea and 31% nausea, and all others were <10%. Only 3 patients had grade 1/2 treatment-related TEAEs during the LTE period, supporting a favorable safety profile with longer treatment. There were two related grade 1 bleeding events (conjunctival hemorrhage and contusion), two related grade 2 infections that resolved on treatment (erysipelas during the main treatment period and upper respiratory tract infection during the LTE), but no related thrombotic events or deaths. Conclusion: Oral rilzabrutinib 400 mg bid was well-tolerated and had durable, clinically significant platelet responses across subgroups and with extended treatment in patients with heavily pretreated ITP. Continued study in the ongoing, randomized phase III LUNA3 trial (NCT04562766) will further assess the magnitude and durability of rilzabrutinib's clinical benefit in ITP. Figure 1 Figure 1. Disclosures Kuter: Actelion (Syntimmune), Agios, Alnylam, Amgen, Argenx, Bristol Myers Squibb (BMS), Immunovant, Kezar, Principia, Protalex, Rigel, Takeda (Bioverativ), UCB: Research Funding; Up-to-Date: Patents & Royalties: Up-To-Date; Rubius: Current equity holder in publicly-traded company; Actelion (Syntimmune), Agios, Alnylam, Amgen, Argenx, BioCryst, Bristol Myers Squibb (BMS), Caremark, CRICO, Daiichi Sankyo, Dova, Genzyme, Immunovant, Incyte, Kyowa-Kirin, Merck Sharp Dohme, Momenta, Novartis, Pfizer, Principia, Protalex, Protalix, Rigel: Consultancy, Other: grant support and consulting fees; Platelet Disorder Support Association: Membership on an entity's Board of Directors or advisory committees. Mayer: Principia: Research Funding. Jansen: 3SBIO, Novartis: Other: Travel, accomodations, expenses; Advisory Board Novartis: Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Other: Travel, Accommodations, Expenses. McDonald: Bayer, Sobi, Novartis, Amgen, argenx: Honoraria; Grifols: Research Funding. Baker: Roche, Janssen-Celeg: Membership on an entity's Board of Directors or advisory committees; Bayer, Takeda, Pfizer, Daiichi Sankyo, CSL Behring, Roche, Amgen, Celgene, Rigel Pharmaceuticals, Abbvie, Sanofi, MorphoSys AG, Acerta Pharma, Jansen-Cileg, Bristol-Myers Squibb, Boehringer Ingelheim, Portola, Technoclone, Alexion: Research Funding. Bird: Novartis, Amgen: Speakers Bureau. Garg: Amgen Janssen Novartis Sanofi Takeda: Honoraria; Takeda Janssen Novartis Sanofi: Other: Travel Accommodations, Expenses; University Hospital Leicester: Current Employment. Gernsheimer: Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: personal fees ; Amgen: Honoraria; Cellphire: Consultancy; Dova: Consultancy, Honoraria; Principia: Membership on an entity's Board of Directors or advisory committees, Research Funding; Rigel: Membership on an entity's Board of Directors or advisory committees, Research Funding; NHLBI: Research Funding. Ghanima: Amgen, Novartis, Pfizer, Principia Biopharma Inc- a Sanofi Company, Sanofi, SOBI, Griffols, UCB, Argenx: Consultancy; Amgen, Novartis, Pfizer, Bristol Myers Squibb, SOBI, Griffols, Sanofi: Honoraria; Bayer, BMS/Pfizer: Research Funding. Bandman: Sanofi: Ended employment in the past 24 months. Arora: Principia Biopharma Inc, a Sanofi Company: Ended employment in the past 24 months. Burns: Sanofi: Ended employment in the past 24 months. Yao: Sanofi: Current Employment. Daak: Sanofi: Current Employment. Sourdille: Sanofi: Current Employment. Thomas: Chinook and Equillium Biopharma: Current holder of individual stocks in a privately-held company; Chinook: Membership on an entity's Board of Directors or advisory committees; Equillium Biopharma: Current Employment; Principia, a Sanofi Company: Ended employment in the past 24 months; Equillium Biopharma: Current equity holder in publicly-traded company. Neale: Principia Biopharma/Sanofi: Ended employment in the past 24 months; Principia Biopharma: Divested equity in a private or publicly-traded company in the past 24 months. Cooper: Sanofi and Principia: Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: travel, accommodations expenses; Principia and Sanofi: Consultancy. OffLabel Disclosure: Rilzabrutinib is an investigational therapy being evaluated in a clinical study for the treatment of patients with immune thrombocytopenia.

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