EX-99.2 3 brhc20053135_ex99-2.htm EXHIBIT 99.2
Exhibit 99.2

 ASGCT 2023 cystinosis update 
 

 Disclaimer  2     This presentation has been prepared by AVROBIO, Inc. (“AVROBIO”) for informational purposes only and not for any other purpose. Certain information contained in this presentation and statements made orally during this presentation relate to or are based on studies, publications, surveys, and other data obtained from third-party sources and AVROBIO’s own internal estimates and research. Although AVROBIO believes these third-party sources to be reliable as of the date of this presentation, they have not been independently verified, and AVROBIO makes no representation as to the adequacy, fairness, accuracy. or completeness of any information obtained from third-party sources. Although AVROBIO believes its internal research is reliable, such research has not been verified by any independent source.   This presentation may contain forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as “aims,” “anticipates,” “believes,” "continue," “could,” “designed to,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” "predicts," "projects," “seeks,” "strives," "should," and “will,” as well as variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our current and prospective product candidates, including our AVR-RD-04 investigational gene therapy for the treatment of cystinosis; regulatory pathways; our plans and expectations with respect to the development of our clinical and preclinical product candidates, the timing of anticipated clinical and regulatory updates; the timing of patient recruitment and enrollment activities, clinical trial results and product approvals; the anticipated benefits of our gene therapy platform including the potential impact on our commercialization activities, timing and likelihood of success; the expected benefits and results of our manufacturing technology, including the implementation of our plato®platform in our clinical trials and gene therapy programs; and the expected safety profile of our investigational gene therapies. Any such statements in this presentation that are not statements of historical fact may be deemed to be forward-looking statements.  Any forward-looking statements in this presentation are based on our current expectations, estimates, and projections about our industry as well as management’s current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of our product candidates will not be successfully developed or commercialized; the risk that regulatory agencies may disagree with our anticipated development approach for any one or more of our product candidates; the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators; the risk that we may not successfully recruit or enroll a sufficient number of patients for our clinical trials; the risk that we may not realize the intended benefits of our gene therapy platform, including the features of our plato® platform; the risk that our product candidates or procedures in connection with the administration thereof, including our use of busulfan as a conditioning agent, will not have the safety or efficacy profile that we anticipate; the risk that prior results, such as signals of safety, activity, or durability of effect, observed from preclinical or clinical trials, will not be replicated orwill not continue in ongoing or future studies or trials involving our product candidates; the risk that we will be unable to obtain and maintain regulatory approval for our product candidates; the risk that the size and growth potential of the market for our product candidates will not materialize as expected; risks associated with our dependence on third-party suppliers and manufacturers; risks regarding the accuracy of our estimates of expenses and future revenue; risks relating to our capital requirements and needs for and availability of additional financing including the risk that failure to obtain additional funding may force us to delay, limit or terminate our product development efforts or other operations; risks relating to our identification and pursuit of any strategic opportunities with respect to one or more of our programs, our technology or our plato platform; risks relating to clinical trial and business interruptions resulting from the ongoing COVID-19 pandemic or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises; and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIO’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in AVROBIO’s most recent Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIO’s subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.  Note regarding trademarks: plato® is a registered trademark of AVROBIO. Other trademarks referenced in this presentation are the property of their respective owners.  © Copyright 2023 AVROBIO, Inc. All rights reserved.
 

 ASGCT 2023 cystinosis update  3  Summary of key points  Continued positive trends across multiple biomarkers and neurocognitive measures seen in Phase 1/2 collaborator-sponsored trial  All patients remain off oral cysteamine, up to 36 months post-gene therapy  Safety and tolerability profile remains strong  Positive interactions with U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and U.S. Food and Drug Administration (FDA) in Q1 2023 
 Phase 1/2  Objectives  Patients  Safety and tolerability   Hypothesis generation of clinical efficacy endpoints  6 patients  Adults and adolescents  Cohorts 1-2 >18 years; Cohort 3 >14 years  Male and female  Oral and ophthalmic cysteamine  Cystinosis Phase 1/2 dosing complete  4  Clinical trial funded in part by grants to UCSD from the California Institute for Regenerative Medicine (CIRM), Cystinosis Research Foundation (CRF) and National Institutes of Health (NIH)  Collaborator-sponsored  University of California, San Diego 
 
 VCN: Vector Copy Number; PBCs: Peripheral Blood Cells; dg: Diploid Genome  *Average of 2 drug products  Note: Patient 2 was lost to follow-up after 12 months  Drug Product VCN/dg  Patient 1  2.1  Patient 2  1.3  Patient 3  1.6  Patient 4  0.6  Patient 5  2.5  Patient 6  2.9*  VCN trending as expected, indicating sustained engraftment  CYSTINOSIS PHASE 1/2: PATIENTS 1-6  NEW DATA POINT  5 
 
 Therapeutic range is <1.0 Half Cystine (nmol/mg protein). Measure of 1 is level of healthy heterozygote.; For Patient 1, Leukocyte Cystine Quantification was initiated at approximately week 20 ; *Patient 1: Hemolyzed sample which may potentially lead to lower results; mo=month; Note: Patient 2 was lost to follow-up after 12 months  NEW DATA   6  Sustained leukocyte cystine level reduction  CYSTINOSIS PHASE 1/2: PATIENTS 1-6  Lift updated data from next slide and put on this slide  NEW DATA POINT  * 
 
 7  Skin and gastrointestinal mucosa cystine crystal reduction  CYSTINOSIS PHASE 1/2: PATIENTS 1-5  Rectal Biopsy  Average intracytoplasmic crystals per cell   Skin Biopsy  8.4  Put on AVRO slide in AVRO format  Note: Patient 2 was lost to follow-up after 12 months  NEW DATA POINT 
 
 Improvement or stabilization in motor coordination and visual perception  8  The Beery – Buktenica Developmental Test of Visual Motor Integration (Beery VMI) [6th edition] is a standardized test evaluating the ability of the brain to interpret and translate visual information into an exact motor response; Patient 2 did not complete the examination  CYSTINOSIS PHASE 1/2: PATIENTS 1-5  Patient 1  Patient 3  Patient 4  Patient 5  NEW DATA POINT 
 
 All patients continue to be oral cysteamine-independent  9  Patient #1 out 3 years  CYSTINOSIS PHASE 1/2: PATIENTS 1-6  Patient  Months off cysteamine pills and eye drops post   Ctns-rd-04 infusion   Current status  Cysteamine  Pills  Patient 1  OFF  Patient 2  Lost to follow-up  Patient 3  OFF  Patient 4  OFF  Patient 5  OFF  Patient 6  OFF  Cysteamine Eye Drops  Patient 1  OFF  Patient 2  Lost to follow-up  Patient 3  OFF  Patient 4  Was not on cysteamine eye drops prior to infusion  OFF  Patient 5  OFF  Patient 6  OFF  Note: Patients 2, 3 and 5 stopped cysteamine eye drops 1-month post-transplant (per protocol); Patient 1 stopped cysteamine eye drops prior to baseline; Data as of May 8, 2023. Patient 2 has elected not to return since the 12-month follow-up visit.   NEW DATA POINT 
 
 eGFR: Estimated Glomerular Filtration Rate; eGFR calculated using CKD-EPI formula  Note: Patient 2 was lost to follow-up after 12 months   10  eGFR data reinforce need for early intervention  CYSTINOSIS PHASE 1/2: PATIENT 1-6  Lift updated data from next slide and put on this slide  CYSTINOSIS PHASE 1/2: PATIENTS 1-5 
 
 No adverse events related to drug product  11  No SAEs or AEs related to drug product  AE: Adverse Event; SAE: Serious Adverse Event  No SAEs reported  Preliminary AEs reported (as of May 8, 2023)  N=46 for patient 1; N=22 for patient 2; N=8 for patient 3; N=29 for patient 4; N=37 for patient 5; N=41 for patient 6  Majority of AEs are mild or moderate  1 severe AE for subject 1  Appendicitis (resolved) – unrelated to study treatment or procedures  AEs are generally consistent with myeloablative conditioning, study procedures, underlying disease or co-morbid or pre-existing conditions:  Pre-gene therapy treatment and prior to conditioning (not all events listed)  Diarrhea, hypokalemia, hypomagnesemia, thrombocytopenia, dizziness, dehydration, vomiting, bone pain, headache  Post-treatment (not all events listed)  Pancytopenia, deep vein thrombosis, Staphylococcus sepsis, Coronavirus infection, alopecia, rash, mucositis  Intermittent: diarrhea, vomiting, loss of appetite, epistaxis, blurry vision, febrile neutropenia, hypomagnesemia, hypokalemia  No adverse events related to drug product 
 
 ASGCT 2023 cystinosis update