AVROBIO, Inc. Reports Second Quarter 2018 Financial Results and Provides Business Update
“2018 has been a very productive year thus far for AVROBIO,” commented
Second Quarter and Recent Business Highlights
- Successfully completed IPO. In
June 2018, AVROBIOsuccessfully completed an initial public offering (IPO) of 6,035,151 shares of common stock at a public offering price of $19.00 per share, including the full exercise by the underwriters of their overallotment option to purchase 787,193 additional shares of common stock. AVROBIO received gross proceeds of $114.7 millionin the offering. The IPO was preceded by a Series B financing in January 2018raising gross proceeds of $60.5 million. These two financings have strengthened the Company’s balance sheet and provided it with funding to significantly advance its programs.
- Initiated patient enrollment and dosing in a Phase 2 clinical trial of AVR-RD-01 for the treatment of Fabry disease. AVR-RD-01 is AVROBIO’s first product candidate from the Company’s pipeline of gene therapies for the treatment of lysosomal storage disorders (LSDs). The Company has dosed the first patient with Fabry disease in the Phase 2 study. The Phase 2 clinical trial is an open-label, single-arm clinical trial evaluating AVR-RD-01 in 8 to 12 male patients who have not previously received treatment with enzyme replacement therapy (ERT) or other therapies for Fabry disease. The trial will assess safety and efficacy as measured by multiple indicators, including changes in globotriaosylsphingosine (Gb3), a lipid that abnormally builds up in multiple tissues of patients with Fabry disease, as well as other clinical endpoints. All enrolled patients in the Phase 2 study will receive a single treatment with AVR-RD-01 and will be followed for 48 weeks to measure safety and efficacy.
- Held pre-Investigational New Drug (pre-IND) meeting to expand enrollment of the Phase 2 clinical trial of AVR-RD-01 for the treatment of Fabry disease to U.S. patients. The Company recently held a pre-IND meeting to discuss the requirements to commence clinical trials in the U.S. The Company plans to open a U.S. site for its ongoing Phase 2 clinical trial of AVR-RD-01 in 2019.
- Continued enrollment in Phase 1 investigator-sponsored trial with the dosing of a third patient in this trial on
July 11th. The Phase 1 trial is designed to assess the safety of AVR-RD-01 in up to six patients with Fabry disease who have been treated with ERT for at least six months. Patients participating in the Phase 1 clinical trial temporarily suspend ERT prior to receiving AVR-RD-01 and then resume ERT treatment at bi-weekly intervals one month after dosing with AVR-RD-01.
AVROBIOwill provide an interim update on both the Phase 1 investigator-sponsored and Phase 2 company sponsored clinical trials in the second half of 2018.
- Remain on track to file Clinical Trial Application (CTA) for AVR-RD-02 in Gaucher disease in second half of 2018.
AVR-RD-02, AVROBIO’s second pipeline program, is being developed as a potential cure for patients with Gaucher disease. AVR-RD-02 is designed to maximize the likelihood of sustained production of glucocerebrosidase (GCase), the genetically deficient enzyme in Gaucher disease, in hematopoietic stem cells and their progeny. The Company plans to initiate a Phase 1/2 clinical trial of AVR-RD-02 in patients with Type 1 Gaucher disease and begin dosing patients in 2019.
- AVR-RD-03 (Pompe disease) and AVR-RD-04 (cystinosis) continue to advance as planned. AVR-RD-03, a gene therapy candidate being investigated for Pompe disease, continues making progress in preclinical development. AVR-RD-04, a gene therapy candidate being investigated for the treatment of patients with cystinosis, remains on track to enroll the first patient in a
University of California, San Diegoinvestigator-sponsored Phase 1/2 study in 2019.
- Appointed two new members to the board of directors with deep financial, clinical development and regulatory experience.
Phillip Donenberghas 23 years of leadership in finance, mergers and acquisitions and operations focused in the pharmaceutical and healthcare industries, recently serving as Chief Financial Officer at AveXis. Annalisa Jenkins has 25 years of global pharmaceuticals industry experience, previously serving as Executive Vice President and Head of Global Researchand Development for Merck Serono and Chief Executive Officer of Dimension Therapeutics. Each director brings to AVROBIOa deep understanding of both biotechnology and gene therapy industries, as well as finance expertise and corporate development experience.
Second Quarter Financial Results
Research and development expenses for the second quarter 2018 were
General and administrative expenses were
Based on its current operating plan,
Various express or implied statements in this release concerning AVROBIO’s future expectations, plans and prospects, including without limitation, its expectations regarding the contributions of any member of its board of directors, including such member’s ability to affect AVROBIO’s development or growth plans, statements regarding the development and the continued progress of AVROBIO’s programs, including the commencement of clinical trials or expansion of trial sites, and the therapeutic potential of its product candidates, and statements regarding the Company’s cash position and expected runway, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Any forward-looking statements in this press release are based on management’s current 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 will not be successfully developed or commercialized, the risk of cessation or delay of any of AVROBIO’s ongoing or planned clinical trials, and the risk that the results of previously conducted studies will not be repeated or observed in ongoing or future studies involving AVROBIO’s 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 the final prospectus related to AVROBIO’s initial public offering filed with the
The Yates Network
CONDENSED CONSOLIDATED BALANCE SHEETS
(In thousands, except share data)
|June 30,||December 31,|
|Cash and cash equivalents||$||155,015||$||5,963|
|Prepaid expenses and other current assets||1,132||345|
|Property and equipment, net||2,050||349|
|Accrued expenses and other current liabilities||5,438||2,098|
|Warrant to purchase redeemable convertible preferred stock||-||35|
|Deferred rent, net of current portion||781||126|
|Other long-term liability||-||500|
|Redeemable convertible preferred stock||-||26,500|
|Total stockholders’ equity (deficit)||148,318||(23,135||)|
|Total liabilities, redeemable convertible preferred stock
and stockholders’ equity (deficit)
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(In thousands, except share and per share data)
|Three Months Ended June 30,|
|Research and development||$||7,407||$||1,881|
|General and administrative||2,140||661|
|Total operating expenses||9,547||2,542|
|Loss from operations||(9,547||)||(2,542||)|
|Total other income (expense), net||(960||)||8|
|Net loss per share attributable to common stockholders — basic and diluted||$||(2.98||)||$||(1.15||)|
|Weighted-average number of common shares used in computing net loss per share attributable to common stockholders—basic and diluted||3,529,269||2,202,735|
Source: AVROBIO, Inc.