Second Quarter 2017 Results and Pipeline Update Our Value, Our Science, Our Focus

Transcription

1 Secnd Quarter 2017 Results and Pipeline Update Our Value, Our Science, Our Fcus August 8, Achillin Pharmaceuticals. All rights reserved. NASDAQ:ACHN

2 Frward-Lking Statements This presentatin includes frward-lking statements within the meaning f the Private Securities Litigatin Refrm Act f 1995 that are subject t risks, uncertainties and ther imprtant factrs that culd cause actual results t differ materially frm thse indicated by such frward-lking statements. Achillin may use wrds such as expect, anticipate, prject target, intend, plan, aim, believe, seek, estimate, can, culd, fcus, will, lk frward, cntinue, gal, strategy, may and similar expressins t identify such frwardlking statements. These frward-lking statements are abut Achillin Pharmaceuticals, Inc. and its business and prspects, including, withut limitatin, statements regarding drug discvery, research, clinical develpment, timing f anticipated clinical trials and clinical data fr ur prduct candidates, ur expectatins regarding the ptential safety, efficacy and clinical utility fr ur prduct candidates, regulatry apprval prcesses, market pprtunities, strategic gals, ur cllabratin with Janssen in HCV, intellectual prperty, cmpetitin, and financial results. T the extent that statements cntained in this presentatin are nt descriptins f histrical facts, they are frward-lking statements reflecting management s current beliefs and expectatins. Varius imprtant factrs may cause differences between ur frward-lking statements and actual results, including withut limitatin, unexpected r unfavrable safety r efficacy data, lwer than expected enrllment rates in clinical trials, changes in the cmpetitive landscape fr ur prduct candidates, changes in the regulatry envirnment, changes in market cnditins r future demand fr ur drug candidates, the inability t prtect ur intellectual prperty, ur freedm t perate under third party intellectual prperty, the risk that Janssen may nt advance the HCV prgram in the time frames prjected r at all, ur need fr future capital, the risk f litigatin r ther disputes, and general market and ecnmic cnditins. These and ther risks and uncertainties are described in the reprts filed by Achillin with the U.S. Securities and Exchange Cmmissin ( SEC ), including its annual reprt n Frm 10-K and quarterly reprts n Frm 10-Q, and subsequent filings with the SEC frm time t time. Yu shuld read these reprts, including the Risk Factrs cntained in these reprts with the understanding that ur actual future results may be materially different frm what we expect. All frward-lking statements cntained in this presentatin speak nly as f the date heref, and Achillin undertakes n bligatin t update any f these statements, except as required by law. 2

3 FACTOR D INHIBITOR PORTFOLIO ACH-4471: First-in-class Cmplement Inhibitr GOALS FOR INITIAL CLINICAL DEVELOPMENT Demnstrate prf-f-mechanism with a highly innvative apprach Lack f C3 fragment depsitin n PNH red bld cells Reductin in plasma Bb levels Demnstrate prf-f-cncept by shwing clinical efficacy Reductin in LDH Increase in hemglbin Imprvement in fatigue scre (FACIT scre) Increase in PNH RBC clne size Elucidate PK/PD Early develpment prgram serves as a gateway t unlck brader ptential f ACH-4471 Understand plasma cncentratins f ACH-4471 necessary fr ptential efficacy Acceptable safety and tlerability prfile 3

4 FACTOR D INHIBITOR PORTFOLIO Unlcking the Brader Ptential f ACH-4471 PLANS FOR EXPANDING CLINICAL PROGRAM PNH Phase 2: Expand n-ging mntherapy trial in untreated patients Phase 2: Add-n trial t supprt switch-strategy fr patients with subptimal respnse t eculizumab C3G & IC-MPGN Phase 2: 14-day dsing Phase 2: 6-mnth dsing with lng-term dsing extensin Natural histry study: Onging study spnsred by Imperial Cllege f Lndn anticipated t enrll up t 400 patients glbally Pineer best-in-disease factr D inhibitin acrss multiple indicatins 4

5 FACTOR D INHIBITOR PORTFOLIO ACH-4471: First-in-class Cmplement Inhibitr GOALS FOR INITIAL CLINICAL DEVELOPMENT Demnstrate prf-f-mechanism with a highly innvative apprach Lack f C3 fragment depsitin n PNH red bld cells Reductin in plasma Bb levels Demnstrate prf-f-cncept by shwing clinical efficacy Reductin in LDH Increase in hemglbin Imprvement in fatigue scre (FACIT scre) Increase in PNH RBC clne size Elucidate PK/PD Early develpment prgram serves as a gateway t unlck brader ptential f ACH-4471 Understand plasma cncentratins f ACH-4471 necessary fr ptential efficacy Acceptable safety and tlerability prfile 5

6 ACH-4471 Phase 2 PNH Three-mnth / Lng-term Extensin Trials Interim Results and Next Steps

7 Study Status and Interim Results Phase 2 Trial f ACH-4471 in Untreated PNH Patients Enrllment: 4 t 12 pts Three-mnth Dse Finding Lng-term Extensin KEY INCLUSION / EXCLUSION CRITERIA PATIENT D Classic PNH PATIENT C Classic PNH PATIENT B Aplastic Anemia / PNH PATIENT A Classic PNH PNH clne size > 10% Anemia (Hgb < 12 g/dl) Ttal days n therapy: 9 Days n 200mg TID: -- Ttal days n therapy: 44 Days n 200mg TID: 14 Ttal days n therapy: 126 Days n 200mg TID: 33 Ttal days n therapy: 132 Days n 200mg TID: 40 LDH > 1.5X ULN ANC > 1,000/ mm 3 Platelets > 50,000 μ/l Nrmal ALT Day 1 Day 28 Day 84 Alk Phs 1.5X ULN Part 1 Part 2 Lng-Term Objectives Change in LDH frm baseline Imprvements in Hgb, FACIT Increase PNH RBC clne size Investigatr determines clinical respnse t guide entry int Part 2 Investigatr assessment f benefit determines entry int extensin trial Extensin Study Hgb: hemglbin LDH: lactse dehydrgenase ANC : abslute neutrphil cunt ALT: alanine amintransferase TID: three times daily Initial dse 100 mg TID. Prtcl subsequently amended t allw: - Newly enrlled patients t start at 150 mg TID - Intra-patient dse escalatin thrughut bth studies Available data as f August 8,

8 LDH (U/mL) Phase 2 Trial f ACH-4471 in Untreated PNH Patients Patient A: Classic PNH 36-year-ld male; diagnsed with PNH in 2011 after presentatin with dermal thrmbsis and hemlytic anemia - Otherwise healthy with active lifestyle; n transfusin requirements at baseline HgB (g/dl) LDH (U/L) FACIT PNH clne size (%) C3 fragment depsitin Current Value Negative Baseline Negative Baseline Days n therapy 100mg TID * 13 days 150mg TID * 62 days 175mg TID * 17 days 200mg TID ** 40 days Status: Patient remains n 200 mg TID x ULN 1.5x ULN Hgb: hemglbin LDH: lactse dehydrgenase TID: three times daily * Median values fr LDH and HgB shwn. ** Individual data pints shwn HgB (g/dl) 8

9 LDH (U/mL) Phase 2 Trial f ACH-4471 in Untreated PNH Patients Patient C: Classic PNH 34-year-ld male; diagnsed with PNH in Otherwise healthy with active lifestyle; n transfusin requirements at baseline (BL) Baseline Days n therapy 9 150mg TID * 13 days HgB (g/dl) LDH (U/L) 175mg TID ** 14 days FACIT PNH clne size (%) 200mg TID ** 14 days C3 fragment depsitin Current Value Negative 2x ULN Baseline Negative x ULN Hgb: hemglbin LDH: lactse dehydrgenase TID: three times daily Patient began taper n day 41 fllwing withdrawn cnsent. Patient Withdrew * Median values fr LDH and HgB shwn. ** Individual data pints shwn HgB (g/dl) Status: Patient reprted nncmpliance after initiatin f 200 mg TID dse Patient vluntarily withdrew cnsent fr reasns unrelated t safety n day 41

10 LDH (U/mL) Phase 2 Trial f ACH-4471 in Untreated PNH Patients Patient D: Classic PNH 54-year-ld male; diagnsed in 2012 with PNH - N histry f transfusin-dependence HgB (g/dl) LDH (U/L) FACIT PNH clne size (%) C3 fragment depsitin Current Value n/a n/a n/a 2x ULN 1.5x ULN Baseline n/a 36 Negative Baseline Days n therapy 150mg TID * 9 days Hgb: hemglbin LDH: lactse dehydrgenase TID: three times daily 175mg TID mg TID -- * Individual data pints shwn HgB (g/dl) Status: Patient recently enrlled Currently receiving 150 mg TID dse and will be evaluated fr intra-patient dse escalatin 10

11 Phase 2 Trial f ACH-4471 in Untreated PNH Patients Patient B: Aplastic Anemia PNH 41-year-ld male; diagnsed with AA in 2008; subsequently diagnsed with PNH in Treated with ATG, ral prednisne and cyclsprine; ending in Baseline marrw functin: platelets range 30-60K, ANC and requires Q3-4 weekly RBC transfusins t maintain HgB 8 g/dl HgB (g/dl) LDH (U/L) FACIT PNH clne size (%) C3 fragment depsitin Current Value Baseline Negative AA: Aplastic Anemia Hgb: hemglbin LDH: lactse dehydrgenase ANC: abslute neutrphil cunt TID: three times daily 11

12 Understanding PNH Classificatins Segment Bne Marrw (BM) Status Classic PNH vs. PNH + Aplastic Anemia (AA) Classic PNH PNH/AA Overlap Syndrme AA w/ subclinical PNH Near-Nrmal Evidence f BM Failure Evidence f BM Failure Significant Mderate - Minimal Minimal t Nne Degree f Hemlysis Tw mst clinically vert presentatins f PNH are: Classic PNH: > hemlysis withut bne marrw failure AA: Overlap f bth PNH and bne marrw failure Bth are assciated with elevated risk f thrmbsis, chrnic kidney disease, and fatigue Estimated 25% PNH patients have verlap Surce: Data n file. Achillin Pharmaceuticals, Inc. 12

13 LDH (U/mL) Phase 2 Trial f ACH-4471 in Untreated PNH Patients Patient B: Aplastic Anemia PNH 41-year-ld male; diagnsed with AA in 2008; subsequently diagnsed with PNH in Treated with ATG, ral prednisne and cyclsprine; ending in Baseline marrw functin: platelets range 30-60K, ANC and requires Q3-4 weekly RBC transfusins t maintain Hgb 8 g/dl HgB (g/dl) LDH (U/L) FACIT PNH clne size (%) C3 fragment depsitin Current Value Negative 2x ULN Baseline Negative Baseline 1.5x ULN Days n therapy mg TID * 13 days 150mg TID * 58 days 175mg TID * 22 days AA: Aplastic Anemia Hgb: hemglbin LDH: lactse dehydrgenase ANC: abslute neutrphil cunt TID: three times daily 200mg TID ** 33 days * Median values fr LDH and HgB shwn. ** Individual data pints shwn HgB (g/dl) Status: Patient remains n 200 mg TID Patient cntinued t receive RBC transfusins during therapy

14 Phase 2 Trial f ACH-4471 in Untreated PNH Patients Summary f Interim Data ACH-4471 has been well tlerated with n SAEs. N clinically significant elevatins in liver enzymes At 200 mg TID, clinically significant imprvements bserved include: Imprvements in hemglbin: g/dl increase and a 1.1g/dL increase fr the 2 classic PNH patients at dse f 200mg TID - Increase in PNH type III RBC clne size Clinically meaningful reductins in LDH Patient interviews and increase in bjective fatigue scres shw meaningful imprvements in well being N evidence f C3 fragment depsitin n PNH RBCs Increases in clne size demnstrated PNH RBC prtectin frm hemlysis Sustained decreases in plasma Bb level indicated targeted inhibitin f factr D (data nt shwn) 14

15 Unlcking the Brader Ptential f ACH-4471 PLANS FOR EXPANDING CLINICAL PROGRAM PNH Phase 2: Expand n-ging mntherapy trial in untreated patients Phase 2: Add-n trial t supprt switch-strategy fr patients with subptimal respnse t eculizumab C3G & IC-MPGN Phase 2: 14-day dsing Phase 2: 6-mnth dsing with lng-term dsing extensin Natural histry study: Onging study spnsred by Imperial Cllege f Lndn anticipated t enrll up t 400 patients glbally Interim results generated with ACH-4471 have been shared with regulatry agencies in rder t supprt planned expansin f PNH and C3G prgrams 15

16 Measures f Clinical Efficacy & Safety in PNH Lactse dehydrgenase (LDH) Hemglbin (Hgb) C3 fragment depsitin Fatigue (FACIT scale) PNH RBC Clne Size Safety GOAL Clinically meaningful reductin in LDH GOAL Stabilize / increase hemglbin GOAL Observe n C3 fragment depsitin n PNH RBCs GOAL Imprvement ver time in bjective measures f patient fatigue GOAL Increase percentage f PNH RBC clnes frm baseline GOAL Gd tlerability prfile Clinical data generated t date highlight the ptential rle f factr D inhibitin in PNH 16

17 Extended-Release Activities fr ACH-4471 ONGOING EXTENDED-RELEASE (XR) FORMULATION Objective Develp an extended release tablet frmulatin t allw fr: Optimized trugh expsures Reduced dsing frequency ACH-4471 has high permeability with animal and mdeling data reprting gd absrptin thrughut the GI tract Human biavailability study fr extended release tablet is planned fr 4Q

18 FACTOR D INHIBITOR PORTFOLIO ACH-5228: Next-Generatin Oral Factr D Inhibitr Plasma Cnc. (ng/ml) Next-generatin factr D inhibitr platfrm can be leveraged t create additinal strategic ptins fr value creatin Structure alteratin in next-generatin factr D inhibitrs target imprvements in ptency and pharmackinetic prperties ACH-5228: GLP studies are n-ging with gal f initiating clinical studies in 4Q17 AP Hemlysis Cmpund ID IC 50 (nm) IC 90 (nm) ACH ACH ACH ACH ACH Plasma Cncentratins after an Oral Dsing in Beagle Dgs ACH-4471 (20 mg/kg DN 5 mg/kg) ACH-5228 (5 mg/kg) ACH-5548 (5 mg/kg) ACH-5628(5 mg/kg) Time (h) 18

19 GEOGRAPHIC ATROPHY (GA) AND DRY AMD Factr D Inhibitrs in Ophthalmlgical Disease Surces: Rche Gegraphic Atrphy (GA) is an advanced frm f dry Age-related Macular Degeneratin (dry AMD) GA is a leading cause f impaired visin and blindness that affects mre than 2.6 millin peple in US & EU and mre than 5 millin wrld-wide Factr D is a validated target as demnstrated by Genentech s lampalizumab prgram Phase 3 trial with lampalizumab underway evaluating intra-vitreal injectins every 4 r 6 weeks Achillin is targeting treatment duratin f 3 mnths r lnger with diverse small mlecule factr D inhibitrs Fur diverse cmpunds and distinct delivery appraches are advancing this year with the gal f initiating IND enabling studies in

20 Achillin Develpment Prtfli PROGRAM DELIVERY DISCOVERY : PRECLINICAL CLINICAL Discvery DMPK & Safety Preclinical Phase 1 Phase 2 Phase 3 HEPATITIS C JNJ-4178 (dalasvir+al-335+simeprevir) Oral 6- and 8-week treatment regimen COMPLEMENT FACTOR D PLATFORM Parxysmal Ncturnal Hemglbinuria (PNH) ACH-4471: Factr D Inhibitr Oral C3 Glmerulpathy (C3G) & IC - MPGN ACH-4471: Factr D Inhibitr Oral AP-mediated diseases ACH-5228: Next-Generatin fd inhibitr Oral Gegraphic Atrphy (GA), advanced frm f dry AMD Next-generatin fd inhibitrs Ophthalmic fd: Factr D DMPK: Drug Metablism/Pharmackinetics AP: Alternative Pathway 20

21 Financial Summary: Capitalizatin and Ownership Balance Sheet Metrics As f 6/30/2017 Cash, cash equivalents, marketable securities and interest receivable Debt bligatins Shares utstanding Tp Sharehlders $369.9 millin $0.5 millin millin Psitin Jhnsn & Jhnsn Develpment Crp millin (13%) RA Capital 13.6 millin (10%) Orbimed Advisrs 13.1 millin (10%) Blackrck, Inc. 9.9 millin (7%) Vanguard Grup 9.6 millin (7%) Janus Capital Management 4.4 millin (3%) State Street Glbal Advisrs 4.3 millin (3%) Gldman Sachs & C. 3.7 millin (3%) Bitechnlgy Value Fund (BVF) 3.3 millin (2%) Numeric Investrs 3.0 millin (2%) Based upn mst recent SEC filings. 21

22 Strength t Achieve ur Gals Near-term Develpment Plan Cmpund Indicatin Next steps ACH-4471 PNH Phase 2: Expand n-ging mntherapy Phase 2: Add-n trial t supprt switch strategy fr subptimal respnders ACH-5228 C3G & IC-MPGN Extended Release Tablet AP-mediated diseases (acute/chrnic) Phase 2: 14-day dsing Phase 2: Lng-term (6-mnth dsing) Phase 1 Biavailability Phase 1 SAD/MAD Ophthalmlgy Gegraphic Atrphy Cmplete Preclinical Assessment $369.9 millin in cash, cash equivalents, and interest receivable t supprt expansin HCV: wrldwide cllabratin with Janssen fr JNJ

23 C3 GLOMERULOPATHY (C3G) A Rare Disease with N Available Treatment Renal Survival (%) C3G includes bth Dense Depsit Disease (DDD) and C3 glmerulnephritis (C3GN) Estimated prevalence f 8 12 peple affected per millin in majr markets 100 DDD AND C3GN IMPACT ON RENAL SURVIVAL Incidence rate f 1 2 per millin patients diagnsed with C3G n an annual basis 75 There are n apprved treatments fr patients with C3G Nn-specific treatment appraches include bld pressure cntrl and brad immunsuppressin Significant unmet medical need as nearly half f C3G patients prgress t end-stage renal disease 30-50% prgress t ESRD within 10 years ~70% f patients experience disease recurrence pst renal transplant, with a 50% chance f graft lss Years frm Diagnsis C3GN (n=49) DDD (n=26) Barbur et al. (2015); NICE C3G Evidence Summary (2015); Surces: Servais et al (2013); Medjeral-Thmas et al (2014); Data n File. Achillin Pharmaceuticals

24 C3 GLOMERULOPATHY (C3G) Patient-Fcused Drug Develpment (PFDD) Meeting Achillin was lead spnsr f externally-led PFDD meeting fcused n C3G First renal disease discussed at a PFDD meeting n August 4, 2017 Led by the Natinal Kidney Fundatin Gal is t understand the patient perspective f their disease PFDD meeting prvides an imprtant pprtunity t us and t the FDA t hear directly frm patients / caregivers Understand the impact f the disease n patients daily lives Input may infrm FDA s decisins thrughut the drug develpment prcess Patient experiences shared at the meeting highlight the unmet need and the urgency t develp transfrmative therapies 24

25 Q&A

26 Secnd Quarter 2017 Results and Pipeline Update Our Value, Our Science, Our Fcus August 8, Achillin Pharmaceuticals. All rights reserved. NASDAQ:ACHN