CAMBRIDGE, Mass.–()–AVROBIO, Inc. (Nasdaq: AVRO), a number one clinical-stage gene remedy firm with a mission to free individuals from a lifetime of genetic illness, right this moment reported monetary outcomes for the quarter ended March 31, 2021 and offered a enterprise replace.

“Through the first quarter, we met with the U.S. Meals and Drug Administration (FDA) to debate the regulatory path for AVR-RD-01, our investigational gene remedy for Fabry illness. We plan to interact with FDA within the second half of 2021 to hunt suggestions on our proposal for a registration trial,” mentioned Geoff MacKay, president and CEO of AVROBIO. “As well as, we began the 12 months with sturdy knowledge updates from all three of our medical applications – Fabry illness, Gaucher illness and cystinosis – on the WORLDSymposium™. These knowledge present sustained enhancements throughout necessary biomarkers and useful metrics, which we consider help the potential of our lentiviral gene remedy platform to deal with life-limiting lysosomal problems. We’ve got been gratified by the sturdy curiosity in our medical trials, and we’re happy with our progress in enrolling and dosing new sufferers, together with three sufferers dosed in simply the final three months in our FAB-GT trial. As we advance AVR-RD-01, we plan to proceed to execute throughout our pipeline, and we sit up for offering future updates.”

Program Updates and Milestones

Held assembly with FDA to debate the regulatory path for AVR-RD-01 for Fabry illness within the first quarter of 2021

  • Primarily based on a current U.S. regulatory growth for Fabry illness therapies, AVROBIO intends to debate with FDA a possible registration trial with a proposed main efficacy surrogate endpoint of clearance of Gb3 inclusions in biopsied renal peritubular capillaries as the premise for potential full approval. See full update right here.
  • Topic to FDA suggestions, AVROBIO intends to pursue full approval for investigational AVR-RD-01 as a first-line remedy for Fabry illness by conducting a single, head-to-head registration trial versus Fabrazyme® (agalsidase beta)1 utilizing a kidney biopsy surrogate endpoint much like the FAB-GT Section 2 trial, the place the corporate has seen 100% and 87% substrate reductions at one 12 months post-gene remedy within the two sufferers with evaluable kidney biopsies. The corporate anticipates proposing to FDA a registration trial design with a scope, measurement and period akin to different gene remedy trials.
  • Though FDA steerage gives {that a} surrogate endpoint that was the premise for approval in a selected medical growth program shouldn’t be assumed to be applicable to be used in a special program, AVROBIO believes Fabrazyme’s full approval based mostly on an analogous kidney biopsy surrogate endpoint might doubtlessly help the usage of such an endpoint in a registration trial of AVR-RD-01.

Three further sufferers dosed in three months and plans to amend FAB-GT trial protocol

  • Three sufferers have now been dosed over the previous three months within the FAB-GT Section 2 trial. Seven sufferers of the as much as 14 deliberate trial members have been dosed so far.
  • AVROBIO expects to amend the FAB-GT trial protocol within the second quarter of 2021 to assist help the potential use of AVR-RD-01 in a broad Fabry illness inhabitants. The corporate plans to enroll feminine members, eradicate antibody-status exclusions and add the gathering of information on further parameters which are acknowledged to be limitations of enzyme alternative remedy (ERT), reminiscent of endpoints to evaluate the gene remedy’s potential capability to deal with cardiovascular and central nervous system manifestations.

4 knowledge abstracts accepted for presentation on the 24th digital Annual Assembly of the American Society of Gene & Cell Remedy (ASGCT), Could 11-14, 2021

  • New knowledge updates from an investigator-sponsored Section 1/2 trial2in cystinosis and from AVROBIO’s preclinical program in Pompe illness, in addition to encore knowledge from AVROBIO’s medical program in Gaucher illness sort 1, have been offered.
  • Encore knowledge from AVROBIO’s medical program in Fabry illness might be offered on Friday, Could 14, 2021.
  • See extra data here.

Offered sturdy knowledge updates for Fabry illness, cystinosis and Gaucher illness sort 1 throughout WORLDSymposium™ in February 2021

  • Introduced 100% kidney substrate discount at 12 months post-gene remedy within the first affected person dosed with the plato® platform within the FAB-GT trial, along with further knowledge from a number of biomarkers and useful cardiac measurements throughout each the Section 1 and Section 2 Fabry trials.
  • Confirmed enchancment throughout a number of measures for first two sufferers dosed within the investigator-sponsored Section 1/2 trial in cystinosis, and substantial enchancment in photophobia within the first affected person at 12 months post-gene remedy.
  • Offered early knowledge from the Section 1/2 medical trial in Gaucher illness sort 1 displaying plasma chitotriosidase ranges decreased 49% and poisonous metabolite lyso-Gb1 ranges decreased 44% within the first affected person at six months post-gene remedy, in comparison with the affected person’s pre-treatment ranges whereas on ERT.
  • Extra particulars on the info offered through the seventeenth annual WORLDSymposium™ will be discovered here.

First Quarter 2021 Monetary Outcomes

AVROBIO reported a web lack of $26.9 million for the primary quarter of 2021 as in comparison with a web lack of $26.0 million for the comparable interval in 2020. This enhance was pushed by elevated analysis and growth bills and a lower in different (expense) revenue, web.

Analysis and growth bills have been $18.5 million for the primary quarter of 2021 as in comparison with $18.3 million for the comparable interval in 2020. This enhance was pushed by elevated program growth actions associated to the development of the corporate’s pipeline, in addition to elevated personnel-related prices ensuing from a rise in worker headcount, which incorporates the impression of non-cash stock-based compensation.

Normal and administrative bills have been $8.4 million for the primary quarter of 2021 as in comparison with $8.3 million for the comparable interval in 2020. This enhance was primarily as a consequence of a rise in worker headcount, which incorporates the impression of non-cash stock-based compensation, which was offset by a lower in consulting prices {and professional} charges.

Different (expense) revenue, web was $0.02 million in expense for the primary quarter of 2021 as in comparison with different (expense) revenue, web of $0.6 million in revenue for the comparable interval in 2020. This lower was pushed by a discount in curiosity revenue.

As of March 31, 2021, AVROBIO had $233.0 million in money and money equivalents, as in comparison with $259.7 million in money and money equivalents as of Dec. 31, 2020. Primarily based on the corporate’s present working plan, AVROBIO expects its money and money equivalents as of March 31, 2021, will allow the corporate to fund its working bills and capital expenditure necessities into the primary quarter of 2023.


Our imaginative and prescient is to deliver personalised gene remedy to the world. We intention to stop, halt or reverse illness all through the physique with a single dose of gene remedy designed to drive sturdy expression of therapeutic protein, even in hard-to-reach tissues and organs together with mind, muscle and bone. Our ex vivo lentiviral gene remedy pipeline contains medical applications in Fabry illness, Gaucher illness sort 1 and cystinosis, in addition to preclinical applications in Hunter syndrome, Gaucher illness sort 3 and Pompe illness. AVROBIO is powered by our trade main plato® gene remedy platform, our basis designed to ship gene remedy worldwide. We’re headquartered in Cambridge, Mass., with an workplace in Toronto, Ontario. For extra data, go to, and observe us on Twitter and LinkedIn.

Ahead-Trying Statements

This press launch accommodates forward-looking statements, together with statements made pursuant to the protected harbor provisions of the Non-public Securities Litigation Reform Act of 1995. These statements could also be recognized by phrases and phrases reminiscent of “goals,” “anticipates,” “believes,” “might,” “designed to,” “estimates,” “expects,” “forecasts,” “objective,” “intends,” “might,” “plans,” “attainable,” “potential,” “seeks,” “will,” and variations of those phrases and phrases or comparable expressions which are meant to determine forward-looking statements. These forward-looking statements embrace, with out limitation, statements concerning our enterprise technique for and the potential therapeutic advantages of our product candidates, the design, graduation, enrollment and timing of ongoing or deliberate medical trials, medical trial outcomes, product approvals and regulatory pathways, our plans and expectations with respect to the event of AVR-RD-01, together with timing and design of our potential registration trial, the meant use of such trial as our registration trial for this product candidate, anticipated interactions with regulatory businesses and the deliberate use of surrogate endpoints in future growth of AVR-RD-01, anticipated advantages of our gene remedy platform together with potential impression on our commercialization actions, timing and probability of success, the anticipated advantages and outcomes of our implementation of the plato platform in our medical trials and gene remedy applications, the anticipated security profile of our investigational gene therapies, and statements concerning our monetary and money place and anticipated money runway. Any such statements on this press launch that aren’t statements of historic truth could also be deemed to be forward-looking statements. Ends in preclinical or early-stage medical trials will not be indicative of outcomes from later stage or bigger scale medical trials and don’t guarantee regulatory approval. You shouldn’t place undue reliance on these statements, or the scientific knowledge offered.

Any forward-looking statements on this press launch are based mostly on AVROBIO’s present expectations, estimates and projections about our trade in addition to administration’s present beliefs and expectations of future occasions solely as of right this moment and are topic to various dangers and uncertainties that might trigger precise outcomes to vary materially and adversely from these set forth in or implied by such forward-looking statements. These dangers and uncertainties embrace, however will not be restricted to, the chance that anybody or extra of AVROBIO’s product candidates is not going to be efficiently developed or commercialized, the chance of cessation or delay of any ongoing or deliberate medical trials of AVROBIO or our collaborators, the chance that regulatory businesses might disagree with our anticipated growth method for our product candidates reminiscent of AVR-RD-01, together with that we might not have the ability to make the most of our deliberate registration trial of AVR-RD-01 for full approval however as an alternative be required to conduct further testing, that we could also be required to conduct our deliberate testing in a extra time-consuming, costly, difficult or in any other case completely different method than we envision or have carried out for our present trials, significantly in mild of the FDA’s choice for medical trials to be double-blinded and doubtlessly embrace sham controls, the chance that we might not have the ability to make the most of our envisioned surrogate endpoint to help full approval of AVR-RD-01 however as an alternative be required to measure a special endpoint reminiscent of a medical final result, the chance that AVROBIO might not efficiently recruit or enroll a ample variety of sufferers for our medical trials, the chance that AVROBIO might not notice the meant advantages of our gene remedy platform, together with the options of our plato® platform, the chance that our product candidates or procedures in reference to the administration thereof is not going to have the protection or efficacy profile that we anticipate, the chance that prior outcomes, reminiscent of indicators of security, exercise or sturdiness of impact, noticed from preclinical or medical trials, is not going to be replicated or is not going to proceed in ongoing or future research or trials involving AVROBIO’s product candidates, the chance that we’ll be unable to acquire and preserve regulatory approval for our product candidates, the chance that the scale and development potential of the marketplace for our product candidates is not going to materialize as anticipated, dangers related to our dependence on third-party suppliers and producers, dangers concerning the accuracy of our estimates of bills and future income, dangers regarding our capital necessities and wishes for added financing, dangers regarding medical trial and enterprise interruptions ensuing from the COVID-19 outbreak or comparable public well being crises, together with that such interruptions might materially delay our enrollment and growth timelines and/or enhance our growth prices or that knowledge assortment efforts could also be impaired or in any other case impacted by such crises, and dangers regarding our capability to acquire and preserve mental property safety for our product candidates. For a dialogue of those and different dangers and uncertainties, and different necessary components, any of which might trigger AVROBIO’s precise outcomes to vary materially and adversely from these contained within the forward-looking statements, see the part entitled “Danger Elements” in AVROBIO’s most up-to-date Annual or Quarterly Report, in addition to discussions of potential dangers, uncertainties and different necessary components in AVROBIO’s subsequent filings with the Securities and Trade Fee. AVROBIO explicitly disclaims any obligation to replace any forward-looking statements besides to the extent required by regulation.


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1 Fabrazyme® (agalsidase beta) is a registered trademark owned by Sanofi Genzyme.

2 Collaborator-sponsored Section 1/2 medical trial of AVR-RD-04 is funded partially by grants to UCSD from the California Institute for Regenerative Medicine (CIRM), Cystinosis Research Foundation (CRF) and Nationwide Institutes of Well being (NIH).

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