Apellis Company Presentation November
|
|
- Alan Hines
- 5 years ago
- Views:
Transcription
1 Apellis Company Presentation November
2 Forward looking statements Statements in this presentation about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of These statements include, but are not limited to, statements relating to the implications of preliminary clinical data and planned or future clinical trials and the timing thereof. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, should, target, will, would and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: whether preliminary or interim results from a clinical trial will be predictive of the final results of the trial; whether results obtained in preclinical studies and clinical trials such as the results referenced in this presentation will be indicative of results that will be generated in future clinical trials; whether APL-2 will successfully advance through the clinical trial process on a timely basis, or at all, and receive approval from the United States Food and Drug Administration or equivalent foreign regulatory agencies; whether, if Apellis products receive approval, they will be successfully distributed and marketed; and other factors discussed in the Risk Factors section of Apellis Annual Report on Form 10-K filed with the Securities and Exchange Commission on July 31, 2018, and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. 2
3 Key milestones for 2018 GA: Phase 2: 18 month safety & efficacy data GA: Start of Phase 3 program PNH: Phase 1b: monotherapy expansion Phase 1b: Soliris weaning in addon study Start of Phase 3 program AIHA: Phase 2: POC data in CAD Phase 2: POC data in wa-aiha AIHA: Preliminary data in CAD & wa-aiha 3
4 What we do C3 Pioneers in innate immunity & complement immunology By regulating its core component C3 Value & patient outcomes at the center of our programs Initially focused on AMD & PNH Broad potential in other immune conditions 4
5 Pipeline Product Area Disease Pre-clinical Phase 1 Phase 1b/2 Phase 3 APL-2 (intravitreal) Ophthalmology Geographic Atrophy (GA) APL-2 (subcutaneous) Hematology Paroxysmal Nocturnal Hemoglobinuria (PNH) Auto-immune Hemolytic Anemia (AIHA) Nephrology Complement-dependent Nephropathies (CDN) APL-9 (intravenous) Other Undisclosed 5
6 Apellis lead molecule: APL-2 Cyclic peptide Cyclic peptide Polyethyleneglycol (PEG) Peptides of the APL-2 family bind to a pocket of C3 and inhibit activation* Subcutaneous or intravitreal injections * Janssen, J. Biol. Chem., 282(40), ,
7 Central inhibition of complement Lectin Pathway APL-2 C3a Inflammation Classical Pathway C3b C5 C5a Inflammation Alternative Pathway Cell removal, Antigen uptake by APCs C5b MAC Cell death, secretion, lysis, or proliferation 7
8 Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare, life-threatening blood disease PNH characterized by uncontrolled hemolysis Intravascular hemolysis Red blood cell rupture in the circulation Extravascular hemolysis Red blood cell destruction by macrophages in spleen and liver ~12,000 prevalent patients in US, EU & Japan ~4,700 patients in US 35% 5-year mortality if untreated (thrombosis, severe anemia) Alexion s Soliris (eculizumab) is only approved therapy Treats only intravascular hemolysis Many Soliris treated patients remain anemic and transfusion dependent due to extravascular hemolysis 8
9 Number of patients Hemoglobin levels in patients with PNH receiving eculizumab (n=141; all hemolytic) 8 >4 g/dl 6 PNH patients on eculizumab Normal individuals Hemoglobin (gm/dl) Source: Peter Hillmen, Professor of Experimental Haematology, University of Leeds 9
10 Only 30% of patients on Soliris have bone marrow function strong enough to keep patients from experiencing anemia and/or transfusion ~30% of Patients ~40% of Patients ~20% of Patients ~10% of Patients Transfusion - v Transfusion - v Transfusion ++ Breakthrough ++ Hb >12 Hb <12 Hb <10 Hb = any 10
11 PHAROAH: APL-2 shows potential to improve eculizumab outcomes as add-on therapy in PNH 270 mg/day, N=6 Mean Hemoglobin g/dl normal treatment started Off APL-2 for 3 Weeks BMI 59 Under-dosed? Months dosing Patient transfusions treatment started Mean LDH Patient 1 Patient 4 ULN Patient 2 Patient 5 Patient 3 Patient normal Interim data as reported June
12 PHAROAH: APL-2 shows potential to improve eculizumab outcomes as add-on therapy in PNH 270 mg/day, N= normal Mean Hemoglobin treatment started g/dl Months dosing Patient transfusions treatment started Mean LDH Patient 1 Patient 4 ULN Patient 2 Patient 5 Patient 3 Patient normal Interim data as reported June
13 Hemoglobin (g/dl) LDH (xuln) PHAROAH: APL-2 add-on to Soliris - all four patients successfully transitioned to APL-2 monotherapy normal treatment started Soliris Soliris Patient transfusions Soliris Soliris Months 0 Interim data as reported Sept 4,
14 PHAROAH: APL-2 add-on to Soliris - all four patients successfully transitioned to APL-2 monotherapy Eculizumab APL-2 + Monotherapy i Eculizumab ii APL-2 Monotherapy iii Hemoglobin (g/dl) * Annual Transfusions (avg.) LDH (ULN) * 1.0x 0.8x 0.9x Reticulocytes (ULN)* 2.7x 1.2x 0.8x Patient Years (Total) NA 5.9 Years 1.9 Years Multiple of Eculizumab Label Dose (900mg x 2wk.) 1.6x 1.0x - *Average last available reading for all four patients on each dosing regimen i) last reading during eculizumab monotherapy prior to co-treatment with APL-2 (ii) last reading during co-treatment and prior to APL-2 monotherapy (iii) last reading while on APL-2 monotherapy Interim data as reported Sept 4,
15 x ULN x ULN PADDOCK (interim): APL-2 shows potential to reach normal LDH levels as monotherapy in treatment in naïve PNH patients 270 mg/day LDH (mean±se) LDH (mean±se) 12.0x 3.0x 10.0x APL-2 2.5x 8.0x 2.0x 6.0x 1.5x 4.0x 1.0x normal 2.0x 0.5x 0.0x normal Weeks 0.0x Weeks Week Week n n=13 n=13 n=13 n=10 n=8 n=5 n=5 n=4 Not Multiple 9.54x 1.76x 0.80x 0.66x 0.79x 0.95x 0.94x Taken 0.91x of ULN n n=13 n=13 n=13 n=10 n=8 n=5 n=5 n=4 Not Multiple 9.54x 1.76x 0.80x 0.66x 0.79x 0.95x 0.94x Taken 0.91x of ULN Excludes results from one of the original three patients, who had underlying metastatic ovarian cancer with a chronic low gastrointestinal bleed, unknown at the time of screening. Excludes results from one of the original three patients, who had underlying metastatic ovarian cancer with a chronic low gastrointestinal bleed, unknown at the time of screening. Interim data as reported June 26, 2018 at Apellis R&D day 15
16 PADDOCK (interim): Mean hemoglobin improvement was 3.5 g/dl (n=13) showing an impact on both intravascular and extravascular hemolysis Hemoglobin 2/13 patients had transfusions, one at day 2 and a noncompliant patient at day 14; it is believed that neither patient at day 14 had yet reached sufficient exposure to APL-2 for hematological benefit 2/13 patients had transfusions, one at day 2 and a non-compliant patient It is believed that neither patient had yet reached sufficient exposure to APL-2 for hematological benefit At last measure; excludes one patient who had underlying metastatic ovarian cancer with a chronic low gastrointestinal bleed, unknown at the time of screening, which resulted in artificially low Hb and high LDH levels that were determined to be unrelated to PNH. At last measure; excludes one patient who had underlying metastatic ovarian cancer with a chronic low gastrointestinal bleed, unknown at the time of screening, which resulted in artificially low Hb and high LDH levels that were determined to be unrelated to PNH Interim data as reported June 26, 2018 at Apellis R&D day 16
17 10^9/L umol/l PADDOCK (interim): other measures of anemia meaningfully improved with APL-2 including reticulocytes and bilirubin Reticulocytes (mean±se) Total Bilirubin (mean±se) normal 10 5 normal Weeks Weeks Week Week n n=13 n=13 n=13 n=11 n=8 n=5 n=5 n=4 Not Mean Retics Taken 92 (10^9/L) n n=13 n=13 n=13 n=11 n=8 n=5 n=5 n=4 Not Mean Bilirubin Taken 13 (umol/l) Interim data as reported June 26, 2018 at Apellis R&D day 17
18 PEGASUS Phase 3 Head to Head Vs. Soliris Eculizumab maintenance Screening 1 Month Run-in 4 Month Randomized controlled period Open-label APL-2 period Follow up* On Eculizumab (stable for 3 ) Meet include/exclude criteria APL-2 + Eculizumab (All Patients) Group 2 (n=35) Eculizumab Group 1 (n=35) APL-2 Group 2 Ecul. + APL-2 Group 1+2 APL-2 Group 1+2 APL-2 *Open Label extension may be offered to all participants if clinical benefit is evident Baseline 1:1 Randomized Parameter Population Primary Endpoint Design Posology Sample size PNH patients on eculizumab who continue to be anemic (Hb < 10.5 g/dl at screening) Week 16 change from baseline in hemoglobin level Randomized 1:1 APL-2:eculizumab. 1 Month Run In; 4 Month Randomization; 6 Month Open Label; 1 Month Follow Up Dose 1080 mg 2/week, SC infusion via injection pump 70 patients (35/group) randomized 1:1 provides 90% power with 2-sided=0.05 to detect a difference of 1 g/dl 18
19 STUDY 2 Phase 3 in Treatment Naïve PNH Patients Masked, controlled APL- 2 N = 32 Open label Screening N = 48 R: 1:2 26 weeks APL- 2 N = 48 No Drug N = 16 8 Parameter Population 1 Endpoint Patients with PNH (not on eculizumab) Hb stabilization, defined as avoidance of a decrease in Hb levels from baseline in the absence of transfusion through Week 26 Design Randomized 2:1 APL-2:No-treatment t 26 weeks No-treatment controlled period followed by 8 open label period Posology Sample size Dose 1080 mg, twice/week SC injection via injection pump 48 patients randomized 2:1 (32:16) to APL-2:No-treatment provides 90% power with 2-sided =0.05 to detect a 45% difference in Hb stabilization (assuming a Notreatment rate of 5% and an APL-2 rate of 50%). 19
20 APL-2 PNH program early data suggests potentially differentiated efficacy C3 APL-2 blocks C3 and inhibits intravascular & extravascular hemolysis LDH reduction appears to be equivalent to or better than C5 inhibitors Significant increase in HgB levels to normal ranges in most patients Other hematological measures meaningfully improved including reticulocytes and bilirubin Ameliorates transfusion dependence in sub-optimal responding patients on high dose C5 inhibitors (PHAROAH) Favorable product profile with convenient sub-q dosing & stability at room temperature for several While the LDH corrections seen with APL-2 monotherapy in these patients with PNH are truly remarkable, it is the significant hemoglobin correction that is most clinically meaningful. Dr. Peter Hillmen Elevated reticulocytes and bilirubin are important markers of anemia resulting from extravascular hemolysis and are not known to improve in patients treated with eculizumab or other C5 inhibitors. Dr. Anita Hill 20
21 Autoimmune hemolytic anemia (AIHA) is a group of rare autoimmune disorders characterized by the premature hemolysis of red blood cells (RBCs) by autoantibodies AIHA presents in two common forms ~25,000 AIHA patients in US AIHA patients present with anemic symptoms similar to PNH Cold Agglutinin Disease Typically associated with IgM autoantibodies 20-25% of cases Warm Antibody AIHA Typically associated with IgG autoantibodies % of cases Overall mortality of 11% IgG and IgM antibodies are the main cause of AIHA resulting in RBC phagocytosis and lysis Corticosteroids are first line therapy Many patients progress to splenectomy or Rituxan (off-label) 21
22 g/dl 10 9 /l APL-2 in Cold Agglutinin Disease (n=2) Preliminary Data Hemoglobin Reticulocytes mg/d CAD mg/d CAD mg/d CAD mg/d CAD Days Days Interim data as reported Nov 1,
23 g/dl 10 9 /l APL-2 in Warm Antibody AIHA (n=2) Preliminary Data Hemoglobin Reticulocytes mg/d wa-aiha mg/d wa-aiha mg/d wa-aiha mg/d wa-aiha Days Days Interim data as reported Nov 1,
24 Geographic Atrophy Impacts One Million People in the U.S. Alone 24
25 Geographic Atrophy - the leading cause of blindness Intermediate AMD Presence of drusen. No serious vision loss. Wet AMD Rapid, serious vision loss if untreated. First-line treatment with VEGF inhibitors. Up to 98% of chronic anti-vegf patients progress to GA. Geographic Atrophy Risk of blindness when central vision is affected. ~1M patients in US alone. No approved therapies. Source: American Academy of Ophthalmology; The Lancet; Ophthalmology; L.E.K. interviews and analysis 25
26 FILLY - Phase 2 study of APL-2 in Geographic Atrophy Sham group, n=81 (pooled) APL-2 EOM, n=79 APL-2 Monthly, n=86 APL-2 0 mg Sham injections APL-2 injections every other month APL-2 15 mg APL-2 15 mg APL-2 injections every month 26
27 FILLY timeline and endpoints Primary efficacy endpoint is the primary registration endpoint Change in geographic atrophy (GA) lesion size from baseline to month 12. images taken at treatment period 12 no injections 18 Primary safety endpoint Number and severity of local and systemic treatment emergent adverse events (TEAEs). 27
28 Filly baseline characteristics Sham injections N=81 APL-2 every other month N=79 APL-2 monthly N=86 Bilateral GA, n (%) 72 (90.0%) 64 (82.1%) 71 (85.5%) History of CNV in Fellow Eye, n (%) 29 (35.8%) 28 (35.4%) 36 (41.9%) GA lesion size, mean, mm 2 (SD) 8.2 (4.1) 8.9 (4.5) 8.0 (3.8) BCVA score, mean letters (SD) 59.8 (17.2) 58.4 (16.0) 59.8 (15.7) BCVA score (Snellen equivalent) 20/63 20/80 20/63 LL-BCVA score, mean letters (SD) 33.6 (17.8) 31.4 (17.1) 36.3 (16.6) Groups were well balanced as to age, gender and race 28
29 Lesion growth (mm) APL-2 slowed GA growth at 12 (square root) primary endpoint sham injections APL-2 every other month APL-2 monthly % lesion growth difference p=0.067 vs Sham % lesion growth difference p=0.008 vs Sham Modified Intent to Treat population (mitt), Observed, Mixed-Effect Model 29
30 Lesion growth (mm) Lesion growth by six-month periods (square root) - post hoc analysis Sham group Sham injections APL-2 EOM APL-2 injections every other month APL-2 Monthly APL-2 injections every month % lesion growth difference vs sham p= % lesion growth difference vs sham p < Data from subjects with a measurable GA lesion size at both Months 6 & 12 30
31 Lesion growth (mm) Lesion growth by six-month periods (square root) - post hoc analysis Sham group Sham injections APL-2 EOM APL-2 injections every other month APL-2 Monthly APL-2 injections every month % lesion growth difference vs sham p >0.5 12% lesion growth difference vs sham p = 0.47 Data from subjects with a measurable GA lesion size at both Months 6 & 12 & 18 31
32 Lesion growth (mm) After cessation of treatment at 12, GA growth resumes but treatment effect is maintained through 18 (square root) sham injections APL-2 every other month APL-2 monthly % lesion growth difference p=0.097 vs Sham 20% lesion growth difference p=0.044 vs Sham Modified Intent to Treat population (mitt), Observed, Mixed-Effect Model 32
33 Lesion growth (mm) GA growth comparison: fellow eye vs study eye - post hoc analysis Sham group Sham injections APL-2 EOM APL-2 injections every other month APL-2 Monthly APL-2 injections every month 0.4 N=72 N=63 Difference N= p > % 23% Difference p = Fellow Study Fellow Study Fellow Study Includes patients from the Bilateral GA Population 33
34 New onset wet AMD Sham group Sham injections APL-2 EOM APL-2 injections every other month APL-2 Monthly APL-2 injections every month 1% 8% 18% 9% 21% 12-month outcomes 18-month outcomes 34
35 New onset wet AMD FILLY: 38% of enrolled patients had wet AMD in the non-study eye (fellow eye), balanced between the three groups 6 patients developed wet AMD in the month non-treatment period (5/6 had fellow eye wet AMD) Sham group Sham injections DRY APL-2 EOM every other month DRY Fellow eye WET APL-2 Monthly every month Fellow eye DRY Fellow eye WET Expected based on natural history ~2%/yr for Dry fellow eye patients (Sunness et al 1999) and ~10%/yr for Wet fellow eye patients (Marques et al. 2013) 35
36 FILLY phase 2 trial C3 Preventing complement activation by blocking C3 No specific genotype driving results Statistically significant data in largest Phase 2 in GA (n=246) Further confidence in results from intra-patient control Results correlated to treatment frequency with increasing effect size over time Phase 3 program initiated Risk benefit profile observed at 18 supporting decision to move to Phase 3 Upon discontinuation of APL-2, treatment effect is maintained through 18 36
37 DERBY & OAKS - Phase 3 Program Overview Population 1 o Endpoint 2 Global Studies Patients with Geographic Atrophy secondary to AMD Change in total area of GA lesion(s) based on FAF at Month 12 Each study will have the following design: Screening - R:2:1:2:1 2 years APL-2 Monthly N = 200 Design Double Masked, Randomized 2:1:2:1 APL-2 EOM N = 200 Treatment 15 mg/0.1 ml Intravitreal Injection vs. Sham Injection Sham Monthly N = 100 Sham EOM N = 100 Sample size 600 Subjects from approx. 100 multinational sites per study 37
38 Key milestones for 2018 GA: Phase 2: 18 month safety & efficacy data GA: Start of Phase 3 program PNH: Phase 1b: monotherapy expansion Phase 1b: Soliris weaning in addon study Start of Phase 3 program AIHA: Phase 2: POC data in CAD Phase 2: POC data in wa-aiha AIHA: Preliminary data in CAD & wa-aiha 38
39 39
40 Thank you design by 40
Apellis Company Presentation October
Apellis Company Presentation October 218 1 Forward looking statements Statements in this presentation about future expectations, plans and prospects, as well as any other statements regarding matters that
More informationJefferies Complement Therapeutics Summit April
Jefferies Complement Therapeutics Summit April 218 1 Forward looking statements Statements in this presentation about future expectations, plans and prospects, as well as any other statements regarding
More informationApellis Company Presentation March 2019
Apellis Company Presentation March 219 Forward looking statements Statements in this presentation about future expectations, plans and prospects, as well as any other statements regarding matters that
More informationJPM Presentation January 9, 2018
JPM Presentation January 9, 18 Forward looking statements Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not
More informationInternational PNH Interest Group Meeting Dec 8, 2017
International PNH Interest Group Meeting Dec 8, 2017 APL-2 is a C3 inhibitor APL-2 Subcutaneous for PNH, AIHA & CDN Intravitreal for GA Peptides of the APL-2 family bind to a pocket of C3 and inhibit activation*
More informationNew phase 2 Clinical Trial Enrolling Now
New phase 2 Clinical Trial Enrolling Now Warm Autoimmune Hemolytic Anemia (waiha) and Cold Agglutinin Disease (CAD) Designed for patients Age 18 or older With a primary diagnosis of waiha who had a recurrence,
More informationWelcome to Apellis at the Apella R&D Day 2018
Welcome to Apellis at the Apella R&D Day 2018 Welcome & Today s Objectives Cedric Francois, MD, PhD, Founder and Chief Executive Officer, Apellis Pharmaceuticals Forward Looking Statements Statements in
More informationNew Phase III Clinical Trial Enrolling Now
New Phase III Clinical Trial Enrolling Now Paroxysmal Nocturnal Hemoglobinuria (PNH) Designed for Patients 1. At least 18 years of age 2. With a primary diagnosis of PNH confirmed by high-sensitivity flow
More informationNew phase 2 Clinical Trial Enrolling Now
New phase 2 Clinical Trial Enrolling Now Warm Autoimmune Hemolytic Anemia (waiha) and Cold Agglutinin Disease (CAD) Designed for patients Age 18 or older With a primary diagnosis of waiha who had a recurrence,
More informationTransforming Complement Therapeutics. October 2018
Transforming Complement Therapeutics October 2018 Forward Looking Statements This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of
More informationComplement Focused. Patient Driven.
ACH-4471: Factor D Complex Complement Focused. Patient Driven. Interim Data and Strategic Update December 17, 2018 NASDAQ:ACHN 2018. All rights reserved. Cautionary Note Regarding Forward-Looking Statements
More informationParoxysmal Nocturnal Hemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria Barry Skikne MD, FACP, FCP(SA) Professor of Hematology Division of Hematologic Malignancies and Cellular Therapeutics Cardinal Clinical Manifestations PNH Clonal disease
More information3/31/2014 PNH. Jack Goldberg MD FACP. Clinical Professor of Medicine University of Pennsylvania
PNH Jack Goldberg MD FACP Clinical Professor of Medicine University of Pennsylvania 1 2 3 4 1 5 6 Historically Viewed as a Hemolytic Anemia Normal red blood cells are protected from complement attack by
More informationNext Generation Therapeutics for Disorders of Complement Regulation
Next Generation Therapeutics for Disorders of Complement Regulation March 2018 Forward Looking Statements This presentation contains "forward-looking statements" within the meaning of the Private Securities
More informationTransforming Complement Therapeutics. June 2018
Transforming Complement Therapeutics June 2018 Forward Looking Statements This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995,
More informationSOLIRIS is a Complement Inhibitor Indicated for the Treatment of Patients With PNH to Reduce Hemolysis
SOLIRIS (eculizumab) SOLIRIS is a Complement Inhibitor Indicated for the Treatment of Patients With PNH to Reduce Hemolysis SOLIRIS is the First and Only Approved Therapy for PNH SOLIRIS (eculizumab) [package
More information7/14/2014. SOLIRIS (eculizumab) SOLIRIS PNH Clinical Studies. SOLIRIS Blocks Terminal Complement. 86% Reduction in LDH: TRIUMPH and SHEPHERD
Proximal Terminal Lactate Dehydrogenase (U/L) 7/1/1 SOLIRIS (eculizumab) Humanized First in Class Anti - C5 Antibody SOLIRIS (eculizumab) Human Framework Regions No mutations Germline SOLIRIS is a Complement
More informationPNH PNH PNH 3/22/2016 PNH. Paroxysmal Nocturnal Hemoglobinuria (PNH): Current Thinking on the Disease PATHOGENESIS OF PNH
3//1 PATHGENESIS F PIG-A gene Bone Marrow Failure Disease Scientific Symposium Rockville, March 17-1, 1 1 71 9 3 Paroxysmal Nocturnal Hemoglobinuria (): Current Thinking on the Disease 119 5 15 PRTEIN
More informationTransforming Complement Therapeutics. August 2018
Transforming Complement Therapeutics August 2018 Forward Looking Statements This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of
More informationMANIFEST Phase 2 Enhancement / Expansion
MANIFEST Phase 2 Enhancement / Expansion Investor Conference Call Stellar Science, Breakthrough Medicine October 11, 2018 Forward-Looking Statements This presentation contains forward-looking statements
More informationTransforming Complement Therapeutics. April 2019
Transforming Complement Therapeutics April 2019 Forward-Looking Statements This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of
More informationBrrrr, It s Cold In Here
Brrrr, It s Cold In Here Kate Grogan, MD Transfusion Medicine Fellow Physician, BloodworksNW 4/24/15 Patient 20 year old female Viral illness Presents one week later with severe hemolysis Hgb 5.6 Increased
More informationInarigivir ACHIEVE Trial Results and HBV Clinical Program Update. August 2, 2018
Inarigivir ACHIEVE Trial Results and HBV Clinical Program Update August 2, 2018 FORWARD LOOKING STATEMENT This presentation includes forward-looking statements within the meaning of the Private Securities
More informationOrphan Focused Patient Driven
Orphan Focused Patient Driven June 21, 2018 2018 Achillion Pharmaceuticals. All rights reserved. NASDAQ:ACHN Cautionary Note Regarding Forward-Looking Statements This presentation includes forward-looking
More informationeculizumab, 300mg concentrate for solution for infusion (Soliris ) SMC No. (436/07) Alexion Pharma UK Ltd
eculizumab, 300mg concentrate for solution for infusion (Soliris ) SMC No. (436/07) Alexion Pharma UK Ltd 8 October 2010 (Amended 11 July 2011) The Scottish Medicines Consortium (SMC) has completed its
More informationWhat is PNH? PNH: What it is Not 9/11/2015. What is Paroxysmal Nocturnal Hemoglobinuria?
9/11/15 PNH: Current Thinking on the Disease, Diagnosis, and Treatment Joseph H. Antin, MD Professor of Medicine Harvard Medical School Jock and Bunny Adams Chair in Hematology Dana-Farber/Brigham and
More informationThe use of the complement inhibitor eculizumab (Soliris R ) for treating Korean patients with paroxysmal nocturnal hemoglobinuria
VOLUME 45 ㆍ NUMBER 4 ㆍ December 2010 THE KOREAN JOURNAL OF HEMATOLOGY ORIGINAL ARTICLE The use of the complement inhibitor eculizumab (Soliris R ) for treating Korean patients with paroxysmal nocturnal
More informationNot So Benign Hematology. Robert A. Brodsky, MD Johns Hopkins Family Professor of Medicine and Oncology Division Chief Hematology
Not So Benign Hematology Robert A. Brodsky, MD Johns Hopkins Family Professor of Medicine and Oncology Division Chief Hematology Disclosures Dr. Brodsky serves as a Scientific Advisory Board member to:
More informationDiagnostic Approach to Patients with Anemia
J KMA Special Issue Diagnostic Approach to Patients with Anemia Seonyang Park, MD Department of Internal Medicine, Seoul National University College of Medicine E mail : seonpark@snu.ac.kr J Korean Med
More informationPathophysiology 7/18/2012 PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
PAROXYSMAL NOCTURNAL HEMOGLOBINURIA OUTLINE OF DISCUSSION WHAT IS IT WHO GETS IT NATURAL HISTORY TYPES RISKS COURSE TREATMENTS SYMPTOMS PREGNANCY Pathophysiology Acquired hematopoietic stem cell disorder
More informationSoliris Medical Policy Prior Authorization Program Summary
Soliris Medical Policy Prior Authorization Program Summary Precertification/Prior Authorization may be required under certain plans. Please verify each member s benefits. OBJECTIVE The intent of the Soliris
More informationLJPC-401 Phase 1 Results and Development Update. September 7, 2016
LJPC-401 Phase 1 Results and Development Update September 7, 2016 Forward-Looking Statements These slides contain "forward-looking" statements within the meaning of the Private Securities Litigation Reform
More informationADAPTIMMUNE INVESTOR PRESENTATION. August 2016
ADAPTIMMUNE INVESTOR PRESENTATION August 2016 DISCLAIMER This presentation contains forward-looking statements, as that term is defined under the Private Securities Litigation Reform Act of 1995 (PSLRA),
More informationNot So Benign Hematology Aplastic anemia, Paroxysmal Nocturnal Hemoglobinuria, atypical Hemolytic Uremic Syndrome, Thrombotic Thrombocytopenic Purpura
Not So Benign Hematology Aplastic anemia, Paroxysmal Nocturnal Hemoglobinuria, atypical Hemolytic Uremic Syndrome, Thrombotic Thrombocytopenic Purpura Robert A. Brodsky, MD Johns Hopkins Family Professor
More informationUnderstanding the Complement Cascade and Its Role in Cold Agglutinin Disease. 1 M-CAgD-US-3006 February 2018
Understanding the Complement Cascade and Its Role in Cold Agglutinin Disease 1 February 2018 Instructions This information is provided as an educational resource for healthcare providers. It is not intended
More informationSoliris in NMOSD Phase 3 PREVENT Study Topline Results September 24, 2018
Soliris in NMOSD Phase 3 PREVENT Study Topline Results September 24, 2018 Forward-Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation
More informationESTABLISH 2 Top Line Data Release
The Future of Anti-Infectives ESTABLISH 2 Top Line Data Release March 25, 2013 1 Forward Looking Statements Statements contained in this data release regarding matters that are not historical facts are
More informationNot So Benign Hematology. Robert A. Brodsky, MD Johns Hopkins Family Professor Director of Adult Hematology
Not So Benign Hematology Robert A. Brodsky, MD Johns Hopkins Family Professor Director of Adult Hematology Disclosures Dr. Brodsky serves as a Scientific Advisory Board member to: Alexion Pharmaceuticals
More informationEdasalonexent (CAT-1004) Program
Edasalonexent (CAT-1004) Program Oral small molecule designed to inhibit NF-κB for the treatment of Duchenne muscular dystrophy Joanne M. Donovan, MD, PhD Chief Medical Officer, Catabasis Pharmaceuticals
More informationUtrophin Modulation: A Universal Treatment Approach to DMD. End Duchenne Tour April 2018
Utrophin Modulation: A Universal Treatment Approach to DMD April 2018 Legal Disclaimer Statements in this presentation, other than statements of historical fact, constitute forward-looking statements within
More informationBuilding a Premier Oncology Biotech
Wells Fargo Securities Healthcare Conference Building a Premier Oncology Biotech Dr. Helen Torley, President and CEO September 2018 Forward-Looking Statements All of the statements in this presentation
More informationSoliris (eculizumab) DRUG.00050
Market DC Soliris (eculizumab) DRUG.00050 Override(s) Prior Authorization Approval Duration 1 year Medications Soliris (eculizumab) APPROVAL CRITERIA Paroxysmal Nocturnal Hemoglobinuria I. Initiation of
More informationIdera Pharmaceuticals
Idera Pharmaceuticals ILLUMINATE-204 Clinical Data Update December 2018 Forward Looking Statements and Other Important Cautions This presentation contains forward-looking statements within the meaning
More informationXP23829 PHASE 2 PSORIASIS TRIAL PRELIMINARY TOPLINE DATA PRESENTATION SEPTEMBER 15, 2015 COPYRIGHT 2015 XENOPORT, INC. ALL RIGHTS RESERVED.
XP23829 PHASE 2 PSORIASIS TRIAL PRELIMINARY TOPLINE DATA PRESENTATION SEPTEMBER 15, 2015 COPYRIGHT 2015 XENOPORT, INC. ALL RIGHTS RESERVED. SAFE HARBOR DISCLAIMER These slides and the accompanying oral
More informationCapricor Therapeutics
Therapeutics Conference Call to Discuss the HOPE-2 Clinical Trial NASDAQ: CAPR November 29, 2017 Forward-Looking Statements Statements in this presentation regarding the efficacy, safety, and intended
More informationSoliris. Soliris (eculizumab) Description
Federal Employee Program 1310 G Street, N.W. Washington, D.C. 20005 202.942.1000 Fax 202.942.1125 5.85.11 Subject: Soliris Page: 1 of 5 Last Review Date: September 20, 2018 Soliris Description Soliris
More informationLiving with PNH 7/3/2013. Paroxysmal Nocturnal Hemoglobinuria (PNH): A Chronic, Systemic, and Life- Threatening Disease
Living with PNH Laurence A. Boxer, MD University of Michigan Case Study 15 year old awakened in the morning with chest pain and a sore throat. She experienced chest pain all day accompanied with coughing
More informationSTUDY 1 PHASE 3 TOP-LINE RESULTS. September 2017
STUDY 1 PHASE 3 TOP-LINE RESULTS September 2017 Forward Looking Statement Zogenix cautions you that statements included in this presentation that are not a description of historical facts are forward-looking
More informationBuilding a Premier Oncology Biotech
Corporate Deck Building a Premier Oncology Biotech Dr. Helen Torley, President and CEO November 2018 Forward-Looking Statements All of the statements in this presentation that are not statements of historical
More informationmicrorna Therapeutics Harnessing the power of micrornas to target multiple pathways of disease
microrna Therapeutics Harnessing the power of micrornas to target multiple pathways of disease January 2018 Safe Harbor Statement Statements contained in this presentation regarding matters that are not
More informationDetermined to realize a future in which people with cancer live longer and better than ever before Q Conference Call
Reimagining Cancer Treatment Determined to realize a future in which people with cancer live longer and better than ever before Q1 2016 Conference Call 1 Forward-Looking Statements Disclosure This presentation
More informationCorporate Overview June 2014 Jefferies Healthcare Conference NASDAQ: GLYC
Corporate Overview June 2014 Jefferies Healthcare Conference NASDAQ: GLYC Forward-Looking Statements To the extent that statements contained in this presentation are not descriptions of historical facts
More informationGrowth Conference. October 19, May 2013
Dawson Corporate James Securities Presentation 3rd Annual Small Cap Growth Conference October 19, 2017 May 2013 Safe Harbor This document contains forward-looking statements within the meaning of the "safe
More information8 of 21 (38.1%) Achieved RECIST v1.1 Durable Complete Response (CR) in Predicted Anti-PD-1 Non-Responder Melanoma Patients at 24 Weeks
October 19, 2017 OncoSec Presents Positive Phase 2 Data for ImmunoPulse IL-12 in Combination with Pembrolizumab Demonstrating a Best Overall Response Rate (BORR) of 50% in Predicted Anti-PD-1 Non- Responder
More informationDr.Rajeshwari Basavanna, Dr.Biju George, Dr.Dolly Daniel Transfusion Medicine and Immunohaematology Department of Haematology CMC Vellore
Dr.Rajeshwari Basavanna, Dr.Biju George, Dr.Dolly Daniel Transfusion Medicine and Immunohaematology Department of Haematology CMC Vellore Background Clinical Severity of disease Autoimmune Haemolytic anaemia
More informationThird Quarter 2015 Earnings Call. November 9, 2015
Third Quarter 2015 Earnings Call November 9, 2015 Forward-Looking Statements All of the statements in this presentation that are not statements of historical facts constitute forward-looking statements
More information-- Edasalonexent Substantially Slowed Duchenne Muscular Dystrophy Disease Progression through 36 Weeks --
Catabasis Pharmaceuticals Reports Positive Results from Open-Label Extension of Phase 2 MoveDMD Trial Evaluating Edasalonexent in Duchenne Muscular Dystrophy and Plans to Initiate Phase 3 Clinical Trial
More informationIdenix Pharmaceuticals Building a Leading Antiviral Franchise. Cowen & Company 27 th Annual Healthcare Conference March 13, 2007 Boston
Idenix Pharmaceuticals Building a Leading Antiviral Franchise Cowen & Company 27 th Annual Healthcare Conference March 13, 2007 Boston Safe Harbor This presentation includes forward-looking statements
More informationParoxysmal Nocturnal Hemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria Current Thinking On the Disease Diagnosis and Treatment Ilene Ceil Weitz, MD Associate Clinical Professor of Medicine Jane Anne Nohl Division of Hematology Keck-USC
More informationPRO 140. First self-administered antibody therapy for HIV in late-stage clinical development. March
PRO 140 First self-administered antibody therapy for HIV in late-stage clinical development March 2018 Forward-Looking Statements This presentation includes forward-looking statements and forward-looking
More informationMyeloid Differentiation Observed, Including Induction of CD38 in 85% of Evaluable Patients
December 10, 2017 Syros Announces Initial Clinical Data from Ongoing Phase 2 Trial of SY-1425 Showing Biological and Clinical Activity as Single Agent in Genomically Defined AML and MDS Patients Clinical
More informationOpthea Initiates OPT-302 Diabetic Macular Edema Clinical Trial
ASX and Media Release 3 January 2018 Opthea Initiates OPT-302 Diabetic Macular Edema Clinical Trial Melbourne, Australia; January 3 2018 Opthea Limited (ASX:OPT), a late stage biopharmaceutical company
More informationAnemia (3).ms4.25.Oct.15 Hemolytic Anemia. Abdallah Abbadi
Anemia (3).ms4.25.Oct.15 Hemolytic Anemia Abdallah Abbadi Case 3 24 yr old female presented with anemia syndrome and jaundice. She was found to have splenomegaly. Hb 8, wbc 12k, Plt 212k, retics 12%, LDH
More informationBayer and Regeneron start additional Phase 3 Study for VEGF Trap-Eye in Wet Age-related Macular Degeneration
Investor News Bayer AG Investor Relations 51368 Leverkusen Germany www.investor.bayer.com Bayer and Regeneron start additional Phase 3 Study for VEGF Trap-Eye in Wet Age-related Macular Degeneration International
More informationMedical Policy. MP Eculizumab (Soliris) Related Policies None. Last Review: 01/24/2019 Effective Date: 04/25/2019 Section: Prescription Drug
Medical Policy Last Review: 01/24/2019 Effective Date: 04/25/2019 Section: Prescription Drug Related Policies None DISCLAIMER Our medical policies are designed for informational purposes only and are not
More informationDairion Gatot, Soegiarto Ganie, Savita Handayani. Divisi Hematologi & Onkologi Medik Departemen Ilmu Penyakit Dalam FK-USU/RS H.Adam Malik Medan 2009
HAEMOLYTIC ANAEMIA Dairion Gatot, Soegiarto Ganie, Savita Handayani. Divisi Hematologi & Onkologi Medik Departemen Ilmu Penyakit Dalam FK-USU/RS H.Adam Malik Medan 2009 WHEN BY THEN Hb 9 g% transfusion
More informationPNH Glossary of Terms
AA Absolute neutrophil count Alendronate Allergen ALT Anemia Antibodies Anticoagulant Anticoagulation Antigen Antithymocyte globulin (ATG) Aplastic Aplastic anemia Band Bilirubin Blast cells Bone marrow
More informationPNH ahus gmg. Dosing and Administration Guide
Injection for Intravenous Use PNH ahus gmg For Paroxysmal Nocturnal Hemoglobinuria (PNH) and atypical Hemolytic Uremic Syndrome (ahus), and generalized Myasthenia Gravis (gmg) patients Dosing and Administration
More informationJuly, ArQule, Inc.
July, 2012 Safe Harbor This presentation and other statements by ArQule may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act with respect to clinical
More informationPNH ahus. Dosing and Administration. For Paroxysmal Nocturnal Hemoglobinuria (PNH) and atypical Hemolytic Uremic Syndrome (ahus) patients
For Paroxysmal Nocturnal Hemoglobinuria (PNH) and atypical Hemolytic Uremic Syndrome (ahus) patients PNH ahus Dosing and Administration Soliris is indicated for the treatment of patients with paroxysmal
More informationSAVARA CORPORATE PRESENTATION (NASDAQ: SVRA) NOVEMBER 2018
SAVARA CORPORATE PRESENTATION (NASDAQ: SVRA) NOVEMBER 2018 SAFE HARBOR STATEMENT Savara Inc. ( Savara or the Company ) cautions you that statements in this presentation that are not a description of historical
More informationImmunohematology (Introduction)
Modified from Serotonin version Immunohematology (Introduction) References: -Blood Groups and Red Cell Antigens (Laura Dean) -Cellular and molecular immunology, 8 th edition Introduction to replace blood
More informationMaking Hope A Reality December 10, Nasdaq : BLUE
Making Hope A Reality December 10, 2014 Nasdaq : BLUE Forward Looking Statement These slides and the accompanying oral presentation contain forward-looking statements and information. The use of words
More informationSoliris (eculizumab) Inhibits TMA and Improves Renal Function in Pediatric and Adult Patients with atypical Hemolytic Uremic Syndrome (ahus)
Soliris (eculizumab) Inhibits TMA and Improves Renal Function in Pediatric and Adult Patients with atypical Hemolytic Uremic Syndrome (ahus) New Data from the Largest Prospective Trial of Adult Patients
More informationJefferies Healthcare Conference
Jefferies Healthcare Conference June 7, 2016 NASDAQ: CHMA Forward-Looking Statements These slides contain forward-looking statements and information. The use of words such as may, might, will, should,
More informationAVEO and Astellas Report Final Overall Survival Results from TIVO-1
AVEO and Astellas Report Final Overall Survival Results from TIVO-1 - Median Overall Survival of 28.8 Months Reported for Tivozanib in Patients with Advanced Kidney Cancer - CAMBRIDGE, Mass. and TOKYO,
More informationDr. Shiva Nazari Assistant Professor of Pediatric Oncologist & Hematologist Shahid Beheshti Medical Science University Mofid Children s Hospital
Dr. Shiva Nazari Assistant Professor of Pediatric Oncologist & Hematologist Shahid Beheshti Medical Science University Mofid Children s Hospital Reduction in the normal red cell survival (120 days) RBC
More informationLeerink Immuno-Oncology Roundtable Conference
Leerink Immuno-Oncology Roundtable Conference September 28, 2017 NASDAQ:FPRX Forward-Looking Statements Disclaimer This presentation contains forward-looking statements within the meaning of the Private
More informationBuilding a Premier Oncology Biotech
Building a Premier Oncology Biotech August 208 Forward-Looking Statements All of the statements in this presentation that are not statements of historical facts constitute forward-looking statements within
More informationTreatment of visual impairment due to macular edema secondary to branch retinal vein occlusion (BRVO):
Investor News Not intended for U.S. and UK Media Bayer AG Investor Relations 51368 Leverkusen Germany www.investor.bayer.com Treatment of visual impairment due to macular edema secondary to branch retinal
More informationSAKURA 3 Open-Label Phase 3 Safety Study with DaxibotulinumtoxinA for Injection (RT002) for the Treatment of Moderate to Severe Glabellar Lines
SAKURA 3 Open-Label Phase 3 Safety Study with DaxibotulinumtoxinA for Injection (RT002) for the Treatment of Moderate to Severe Glabellar Lines Presented by Dan Browne, Co-Founder, President & CEO, and
More informationPNH. PNH A study case 5/9/2012. PNH Current Thinking on the Disease, Diagnosis, and Treatment. Where have we been, where are we going?
PNH Current Thinking on the Disease, Diagnosis, and Treatment Where have we been, where are we going? Carlos M. de Castro, MD Duke University Medical Center PNH Case What is PNH? What causes PNH? Relationship
More informationAcute haemolysis and appearance of PNH-like clones in patients with vitamin B12 deficiency and iron deficiency after iron dextran administration
Acute haemolysis and appearance of PNH-like clones in patients with vitamin B12 deficiency and iron deficiency after iron dextran administration Chun-Liang Lin 1, Chin-Chan Lin 1,Wen-Jyi Lo 2,Yu-Chien
More informationDetermined to realize a future in which people with cancer live longer and better than ever before
Determined to realize a future in which people with cancer live longer and better than ever before 3Q 2018 EARNINGS PRESENTATION NOVEMBER 2018 1 Forward-looking statements disclosure This presentation
More informationZIOPHARM / Intrexon Graft-Versus-Host Disease Exclusive Channel Collaboration SEPTEMBER 28, 2015
ZIOPHARM / Intrexon Graft-Versus-Host Disease Exclusive Channel Collaboration SEPTEMBER 28, 2015 1 Forward-looking Statements This presentation contains certain forward-looking information about ZIOPHARM
More informationBuilding a Fully Integrated Biopharmaceutical Company. June 2014
Building a Fully Integrated Biopharmaceutical Company June 2014 Forward-Looking Statements This presentation contains forward-looking statements within the meaning of The Private Securities Litigation
More informationEfficacy of eculizumab in paroxysmal nocturnal hemoglobinuria patients with or without aplastic anemia: prospective study of a Korean PNH cohort
BLOOD RESEARCH VOLUME 52 ㆍ NUMBER 3 September 2017 ORIGINAL ARTICLE Efficacy of eculizumab in paroxysmal nocturnal hemoglobinuria patients with or without aplastic anemia: prospective study of a Korean
More informationParoxysmal Nocturnal Hemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria Jun Ho Jang, M.D., Ph.D. Professor, Division of Hematology-Oncology, Samsung Medical Center Sungkyunkwan University School of Medicine, Seoul, Korea Contents 1. Disease
More informationSynergy Pharmaceuticals TRULANCE (Plecanatide) Receives U.S. FDA Approval for the Treatment of Adults with Chronic Idiopathic Constipation
January 19, 2017 Synergy Pharmaceuticals TRULANCE (Plecanatide) Receives U.S. FDA Approval for the Treatment of Adults with Chronic Idiopathic Constipation NEW YORK--(BUSINESS WIRE)-- Synergy Pharmaceuticals
More informationAcknowledgments. Michael Brown Petra Muus for case reports
Current treatment strategy for PNH Jeff Szer Professor/Director, Department of Clinical Haematology & BMT Service The Royal Melbourne Hospital Australia Acknowledgments Michael Brown Petra Muus for case
More informationCommitted to Transforming the Treatment Paradigm for Migraine Prevention
June 14, 2018 Committed to Transforming the Treatment Paradigm for Migraine Prevention September 6, 2018 Forward-Looking Statements This presentation and the accompanying commentary contains certain forward-looking
More informationAnemia (3).ms Hemolytic Anemia. Abdallah Abbadi Feras Fararjeh
Anemia (3).ms4.26.2.18 Hemolytic Anemia Abdallah Abbadi Feras Fararjeh Case 3 24 yr old female presented with anemia syndrome and jaundice. She was found to have splenomegaly. Hb 8, wbc 12k, Plt 212k,
More informationAnnual Results 2017 & Business Update 13 April 2018
Annual Results 2017 & Business Update 13 April 2018 1 Disclaimer These slides and the accompanying oral presentation contain forward-looking statements and information. Forward-looking statements are subject
More informationQuarterly Update & ASH 2017 Abstract Conference Call
Quarterly Update & ASH 2017 Abstract Conference Call November 1, 2017 Nasdaq : BLUE 1 Forward Looking Statements These slides and the accompanying oral presentation contain forward-looking statements and
More informationJP Morgan Healthcare Conference. January 8, 2007
JP Morgan Healthcare Conference January 8, 2007 Safe Harbor Statement Except for the historical information set forth herein, the matters set forth in this presentation,including without limitation statements
More informationAVEO and Astellas Announce Positive Findings from TIVO-1 Superiority Study of Tivozanib in First-Line Advanced RCC
FOR IMMEDIATE RELEASE AVEO and Astellas Announce Positive Findings from TIVO-1 Superiority Study of Tivozanib in First-Line Advanced RCC - Tivozanib is the First Agent to Demonstrate Greater than One Year
More informationImmunohematology (Introduction) References: -Blood Groups and Red Cell Antigens (Laura Dean) -Cellular and molecular immunology, 8 th edition
Immunohematology (Introduction) References: -Blood Groups and Red Cell Antigens (Laura Dean) -Cellular and molecular immunology, 8 th edition Introduction to replace blood lost by hemorrhage or to correct
More informationGeneral Characterisctics
Anemia General Characterisctics Definition: anemia is a decrease in red blood cells. Happens due to underproduction, increased destruction or loss of red cells. Diagnosis of anemia: Hgb < 135 (men) Hgb
More informationCoding... 5 Benefit Application... 5 Description of Services... 6 Clinical Evidence... 7
TABLE OF CONTENTS Product Variations.... 1 Policy Statement.... 1 Related Policies.... 4 Policy Guidelines..... 4 Coding.... 5 Benefit Application........ 5 Description of Services..... 6 Clinical Evidence.......
More information