Eloxx Pharmaceuticals, Inc. · NASDAQ:ELOX
Eloxx Pharmaceuticals — Corporate Update (November 2021)
30 slides from Eloxx Pharmaceuticals's corporate update, Nov 17, 2021
Slide 1: Topline ELX-02 Phase 2 Cystic Fibrosis Results
RARE Thinking for RARE Solutions Topline ELX - 02 Phase 2 Cystic Fibrosis Results November 17, 2021
Slide 2: Forward-looking statements
/ 2 Forward - looking statements This presentation (and the accompanying oral discussion) contains forward - looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 . All statements other than statements of present and historical facts contained in this press re
Slide 3: Opening Remarks
/ 3 Opening Remarks Sumit Aggarwal President and CEO
Slide 4: Today's agenda
/ 4 Today’s agenda Topic Speaker Opening Remarks Sumit Aggarwal, President & CEO Remaining Unmet Need in Cystic Fibrosis & ELX - 02 Topline Phase 2 Results Dr. Eitan Kerem ELX - 02 Next Steps &Program Expansion Dr. Vijay Modur, Head of R&D Key Takeaways & Closing Remarks Sumit Aggarwal, President &
Slide 5: ELX-02 first-in-class drug in Phase 2 for Class I CF
/ 5 ELX - 02 is first - in - class drug in Phase 2 development for Class I CF patients with nonsense mutations ELX - 02 designed as superior readthrough agent to Gentamicin Compelling preclinical activity observed in highly translatable models Phase 2 monotherapy trial designed to evaluate safety an
Slide 6: Key takeaways from Phase 2 monotherapy trials
/ 6 * p value one - sided t - test =0.022 non - parametric =0.026 Key takeaways from ELX - 02 Phase 2 monotherapy trials Significant unmet need remains for Class 1 CF patients ELX - 02 well - tolerated with no treatment - related serious adverse events Results support continued development of ELX -
Slide 7: Professor Eitan Kerem: Key opinion leader in Cystic Fibrosis
/ 7 Professor Eitan Kerem: Globally renowned key opinion leader in Cystic Fibrosis • Former Chairman, Department of Pediatrics at Hadassah University Hospitals, Jerusalem • Principal investigator in many national and international multi - center Cystic Fibrosis clinical trials. Author of key publica
Slide 8: Remaining Unmet Need in Cystic Fibrosis & Top-line Results
/ 8 Remaining Unmet Need in Cystic Fibrosis & ELX - 02 Top - line Results Dr. Eitan Kerem
Slide 9: No available treatments for Class 1 CF patients
/ 9 CI - CI - CI - CI - CI - CI - CI - CI - wt - CFTR I II III IV V VI No Protein No Traffic No Function Less Function Less Protein Less Stable G542X (a) 394delTT (a) 1717 - 1G>A (b) R1066C A561E F508del G551D S549R G1349D R117H R334W A455E 3272 - 26A>G 3849 + 10 kb C >T c.120del23 rF508del No CFTR
Slide 10: Nonsense mutation CF patients have most severe phenotype
/ 10 CF patients carrying nonsense mutations have most severe phenotype Early presentation – usually at the first weeks of life Pancreatic insufficiency with poor nutritional state More severe lung disease • Higher rate of FEV1 decline • Higher rate of respiratory infection with pseudomonas aerugino
Slide 11: Established endpoints for clinical trials in CF
/ 11 Established endpoints for clinical trials in CF Clinical endpoints Surrogates for CFTR function • Pulmonary function (FEV 1 , LCI) • BMI • Number of Exacerbations • Quality of life questionnaire • Patient reported symptoms • Sweat chloride levels • Nasal potential difference
Slide 12: Phase 2 trial design
/ 12 ELX - 02 Phase 2 design ClinicalTrials.gov Identifier: US Trial NCT04135495, EU/IL Trial NCT04126473 * From baseline to Day 7 of treatment periods 1 - 3, and Days 7 and 14 of treatment period 4 ELX - 02 Phase 2 CF trial designed to evaluate safety and sweat chloride reduction 0.3 mg/kg SC QD fo
Slide 13: No related serious adverse events seen: safety summary
/ 13 No ELX - 02 related serious adverse events seen ELX - 02 safety summary Injection site reactions were the most common finding across the patients – Mild erythema or redness – Mild - moderate Injection pain Drug related discontinuations – Tinnitus (mild - moderate) 1 patient at 0.3 mg/kg • Occur
Slide 14: Sweat chloride secondary endpoint analysis methodology
/ 14 * CLSI. Sweat Testing: Third Edition. CLSI document C34 - A3. Wayne, PA: Clinical and Laboratory Standards Institute ; 2009 ** Am J Respir Crit Care Med Jun 3 2016 Sweat chloride secondary endpoint analysis performed using standard criteria Key statistical assessment to ensure appropriate basel
Slide 15: Activity in Phase 2 of treated Class 1 CF patients
/ 15 Activity in Phase 2 of ELX - 02 treated Class 1 CF patients † p value did not reach significance * p value one - sided t - test =0.022 non - parametric =0.026 ** Results for patients receiving variable doses up to 3.0 mg/kg were not significant. Moreover, inconsistent dosing (as seen in pharmac
Slide 16: Post-hoc subset analysis of 1.5mg/kg/day completers
/ 16 Post - hoc subset analysis of sweat chloride change in 1.5mg/kg/day completers Stronger evidence of dose response in subset analysis of 1.5mg/kg dose completers -14 -12 -10 -8 -6 -4 -2 0 2 4 6 8 0.8 † - 2.6 † ELX - 02 (0.3 mg/kg) ELX - 02 (0.75 mg/kg) - 5.4 ELX - 02 (1.5 mg/kg) Sweat chloride c
Slide 17: Activity in Class 2 patients in Orkambi trials
/ 17 Activity in Class 2 Hom delF508 patients in Phase 2 Orkambi trials Data from Lancet Respir Med . 2014 Jul;2(7):527 - 38 * Approved Orkambi dose Class 2 CF patients on Lumcaftor had a 4.1 to 4.8 mmol/L mean sweat chloride reduction at 2 weeks in Phase 2 - 4.1 8 weeks: Lum/Iva (400mgqd/250mg bid)
Slide 18: Activity in Class 2 patients in Symdeko trials
/ 18 Activity in Class 2 Hom delF508 patients in Phase 2 Symdeko trials Data from Am J Respir Crit Care Med. 2018 Jan 15;197(2):214 - 224 † p value did not reach significance * Approved Symdeko dose Class 2 CF patients on Symdeko had a 1.8 to 5.2 mmol/L mean sweat chloride reduction at 4 weeks in Ph
Slide 19: Summary: monotherapy well tolerated and met sweat chloride endpoint
/ 19 *Note no head - to - head comparison has been conducted between ELX - 02 and these agents Summary: ELX - 02 monotherapy was well tolerated and met the key secondary endpoint of sweat chloride reduction Strong activity with high responder proportion despite short treatment duration and small sam
Slide 20: Next Steps & Program Expansion
/ 20 ELX - 02 Next Steps & Program Expansion Dr. Vijay Modur Head of R&D
Slide 21: Phase 2 results support combination trial with Ivacaftor and Phase 3 planning
/ 21 *Oral Sessions / Journal of Cystic Fibrosis WS03 - 18S1 (2019) S1 – S38 ELX - 02 Phase 2 monotherapy results support planned combination trial with Ivacaftor and Phase 3 planning Phase 2 extension study initiated evaluating combination of ELX - 02 and ivacaftor: On Track for end of Phase 2 meet
Slide 22: Expanded combination trial study design includes longer dosing
/ 22 Expanded combination trial study design includes longer dosing Expanding current combination study across all sites and all Class 1 nonsense mutation CF patients Primary Endpoint: Safety , tolerability and pharmacokinetics Key Secondary endpoints: Sweat chloride and FEV1 N = up to 30 Dose : 1.5
Slide 23: Lung vs plasma exposures with inhaled vs subcutaneous dosing
/ 23 Lung vs Plasma exposures with inhaled vs. subcutaneous dosing Phase 2 results support potential for higher efficacy with inhaled delivery of ELX - 02 13 Lung Plasma 2,010 159X 18.8X Rat dose of 60 - 69 mg/kg equivalent to 1.5 mg/kg human sc dose ELX - 02 Rat drug exposure 69mg/kg inhaled (AUC 0
Slide 24: Class 1 CF program milestones
/ 24 ELX - 02 Class 1 CF program milestones x Completed enrollment of ELX - 02 monotherapy arms in ongoing Phase 2 trials x Report data from ongoing Phase 2 trial of ELX - 02 monotherapy arms in CF x First patient dosed in combination arm of Phase 2 trial of ELX - 02 x Initiated IND - enabling studi
Slide 25: Key Takeaways & Closing Remarks
/ 25 Key Takeaways & Closing Remarks Sumit Aggarwal President and CEO
Slide 26: Key takeaways from Phase 2 monotherapy trials
/ 26 * p value one - sided t - test =0.022 non - parametric =0.026 Key takeaways from ELX - 02 Phase 2 monotherapy trials Significant unmet need remains for Class 1 CF patients ELX - 02 well - tolerated with no treatment - related serious adverse events Results support continued development of ELX -
Slide 27: Deep pipeline of synergistic potential first-in-class therapies
/ 27 * ELX - 02 has received Fast Track Designation from the FDA for the treatment of CF patients with nonsense mutations Class 1 CF: Cystic fibrosis patients with class1 mutations; RDEB/JEB: Recessive Dystrophic/Junctional Epidermolysis Bullosa; FAP : Familial adenomatous polyposis; CRC: Colorectal
Slide 28: Positioned to transform Eloxx and create significant value for shareholders
/ 28 We are positioned to transform Eloxx and create significant value for shareholders Three rare disease programs with over $5B market potential Right new leadership with a track record of success Platform company focused on novel small molecule Genetic Therapies targeting rare diseases and cancer
Slide 29: Questions?
Questions? Answers. Eloxx
Slide 30: PHARMACEUTICALS
i PHARMACEUTICALS TURBO-ZM" Eloxx
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