UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d)
of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): May 18, 2023
(Exact name of registrant as specified in its charter)
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(State or other jurisdiction of incorporation)
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(Commission File Number)
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(I.R.S. Employer Identification No.)
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(Address of principal executive offices, including zip code)
(617 ) 914-8420
(Registrant’s telephone number, including area code)
Not Applicable
(Former Name or Former Address, if Changed Since Last Report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following
provisions:
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Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
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Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
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Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
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Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
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Securities registered pursuant to Section 12(b) of the Act:
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Title of each class
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Trading
symbol(s)
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Name of each exchange
on which registered
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2
of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company ☒
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised
financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
| Item 7.01 |
Regulation FD Disclosure.
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On May 18, 2023, AVROBIO, Inc. (the “Company”) issued a press release titled “AVROBIO Announces Positive Data from Phase
1/2 Clinical Trial of Investigational Gene Therapy for Cystinosis at the ASGCT 26th Annual Meeting.” A copy of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K.
On May 18, 2023, the Company updated its corporate presentation for use in meetings with investors, analysts and others. A copy of the slide presentation is filed as
Exhibit 99.2 to this Current Report on Form 8-K.
The information in Item 7.01 of this Current Report on Form 8-K, including Exhibits 99.1 and 99.2 attached hereto, shall not be deemed “filed” for purposes of the
Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the
Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing.
| Item 9.01 |
Financial Statements and Exhibits.
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(d) Exhibits
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AVROBIO, Inc. press release, dated May 18, 2023.
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AVROBIO, Inc. slide presentation, dated May 18, 2023.
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104
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The cover page from this Current Report on Form 8-K, formatted in Inline XBRL.
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SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto
duly authorized.
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AVROBIO, INC.
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Date: May 18, 2023
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By:
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/s/ Erik Ostrowski
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Erik Ostrowski
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President, Interim Chief Executive Officer, Chief Financial Officer and Treasurer
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Exhibit 99.1
AVROBIO Announces Positive Data from Phase 1/2 Clinical Trial of
Investigational Gene Therapy for Cystinosis at the ASGCT 26th Annual Meeting
All patients remain off oral cysteamine up to 36 months post gene therapy
Sustained engraftment and durable reduction in leukocyte cystine levels across all patients
Received positive regulatory feedback from US and UK agencies
CAMBRIDGE, Mass.—(BUSINESS WIRE)—May 18, 2023—AVROBIO, Inc. (Nasdaq: AVRO), a leading
clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced follow-up data demonstrating a durable treatment effect across key measures out to 36 months from a collaborator-sponsored Phase 1/2
clinical trial1 evaluating an investigational gene therapy for the treatment of cystinosis. These data are being presented at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles,
California, on May 18, 2023.
Cystinosis is a rare, progressive disease with a high treatment burden and unmet need. The fully enrolled Phase 1/2 clinical trial is monitoring long-term
safety and efficacy in six adult patients affected by the most severe and common form of cystinosis who previously had been treated with the standard of care (SOC), cysteamine. The patients’ own hematopoietic stem cells (HSCs) were genetically
modified to express a functional version of cystinosin, the protein that is deficient in people living with cystinosis. Preliminary data suggest that post-gene therapy, functional cystinosin is produced throughout the body as evidenced by clinical
measures in multiple tissues, including the eyes, skin and gastrointestinal mucosa, as well as by neurocognitive tests suggestive of activity in the central nervous system. No adverse events (AEs) related to the drug product or serious adverse
events have been reported to date.
“These data show that genetically modifying a patient’s own HSCs has the potential to restore functional cystinosin and systemically reduce the
accumulation of cystine, laying the foundation for a registration-enabling clinical trial,” said AVROBIO Chief Medical Officer Essra Ridha, M.D., MRCP, FFPM. “We are excited about moving this investigational gene therapy closer to patients.”
In addition to the data presented, the Company also announced positive and productive
meetings with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and U.S. Food and Drug Administration (FDA) designed to align on regulatory paths and obtain feedback on this program.
Data show investigational HSC gene therapy durably and systemically impacts neurocognitive measures and reduces cystine levels in the
blood, and crystal accumulation in the skin and gastrointestinal mucosa
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Follow-up data suggest that after receiving HSC gene therapy, patients can produce
functional cystinosin protein throughout the body. As a result, leukocyte cystine levels in the blood have decreased below baseline in all six patients, and stabilized up to 36 months out from treatment. Skin and gastrointestinal mucosa biopsies
reveal a decline in tissue cystine crystals below baseline in the first four patients, who have been observed for at least 12 months, with two patients observed up to 24 months.
Patients with cystinosis typically do not see an improvement in visual spatial or visual motor function over time in standardized tests evaluating the
ability of the brain to interpret and translate visual information into an exact motor response. The first four patients treated with gene therapy have shown an improvement or stabilization of scores on the Beery – Buktenica Developmental Test of
visual motor integration, up to 36 months out, suggesting a potential impact on the neuropathology of the disease.
These data represent an extension of trends that have previously been measured, confirming the durability of the treatment effect up to 36 months.
Safety and tolerability profile remains strong
Preliminary data from this trial suggest that this HSC gene therapy is well tolerated, with no AEs related to the drug product
to date. All AEs were related to myeloablative conditioning, stem cell mobilization, the underlying disease, co-morbid or pre-existing conditions. The majority of AEs were mild or moderate and resolved without clinical sequelae.
An oral presentation by Dr. Cherqui on these data, “Phase 1/2 Clinical Trial of Autologous
Hematopoietic Stem and Progenitor Cell Gene Therapy for Cystinosis,” will occur today at 3:45 PM PT in the session Metabolic, Storage, Endocrine, Liver and Gastrointestinal Disease II of the ASGCT Annual Meeting. Further details on the Phase 1/2
trial (NCT03897361) are available on clinicaltrials.gov.
About cystinosis
Cystinosis is a rare, progressive disease marked by the accumulation of cystine in cellular
organelles known as lysosomes. This buildup causes progressive organ damage and debilitating corneal damage, swallowing dysfunction, chronic kidney disease leading to end-stage renal disease and muscle wasting leading to a shortened lifespan.
Currently, more than 90% of treated cystinosis patients require a renal transplant in the second or third decade of life. The current standard of care for cystinosis is cysteamine, a treatment regimen that can require dozens of pills per day,
does not prevent overall disease progression and carries side effects, such as breath and body odor and gastrointestinal complications, which may be difficult to tolerate.
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About AVROBIO
Our vision is to bring personalized gene therapy to the world. We target the root cause of genetic disease by introducing a functional copy of the affected gene into
patients’ own hematopoietic stem cells (HSCs), with the goal of durably expressing the therapeutic protein throughout the body, including the central nervous system. Our first-in-class pipeline includes clinical programs for Gaucher disease,
cystinosis and Hunter syndrome, as well as a preclinical program for Pompe disease. Our proprietary plato® gene therapy platform is scalable for planned global commercialization. We are headquartered in Cambridge, Mass. For additional
information, visit avrobio.com, and follow us on Twitter and LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements, including 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,” “could,” “designed to,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,”
“potential,” “seeks,” “will,” and 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 product candidates, including our AVR-RD-04 investigational gene therapy for the treatment of cystinosis, regulatory pathways, anticipated benefits of our gene therapy platform including
potential impact on our commercialization activities, timing and likelihood of success, the expected benefits and results of our implementation of the 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 press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative
of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.
Any forward-looking statements in this press release are based on AVROBIO’s 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 AVROBIO’s product candidates, including AVR-RD-04 for the treatment of cystinosis, will not be successfully developed or
commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk
that AVROBIO 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 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 or will not continue in ongoing
or future studies or trials involving AVROBIO’s 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 additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our enrollment and
development timelines 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 materially and adversely 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.
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Investor Contact:
Christopher F. Brinzey
Westwicke, an ICR Company
339-970-2843
chris.brinzey@westwicke.com
Media Contact:
Kit Rodophele
Ten Bridge Communications
617-999-9620
krodophele@tenbridgecommunications.com
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Exhibit 99.2
