Press Release
Details
AVROBIO Reports Fourth Quarter and Fiscal Year 2019 Financial Results and Provides Business Update
AVROBIO Reports Fourth Quarter and Fiscal Year 2019 Financial Results and Provides Business Update
Presented positive data at the 16th Annual WORLDSymposium™ on AVR-RD-04 for cystinosis and AVR-RD-01 for Fabry disease, as well as early data on plato™-produced drug product and plasma enzyme activity level
Received orphan drug designation from
Raised
“For AVROBIO, 2019 was an important year, with positive data on a primary clinical endpoint from the first patient in our Fabry Phase 2 trial and the unveiling of our state-of-the-art gene therapy platform, plato™,” said
Program Updates and Milestones
AVR-RD-01 in Fabry disease: Evidence of durability and tolerability across both Phase 1 and Phase 2 trials
- For the first patient in the Phase 1 trial, data continue to demonstrate vector copy number (VCN) stability out to 32 months following treatment, supportive of long-term engraftment. The VCN data profiles were generally consistent across the seven other Phase 1 and Phase 2 trial participants out six to 24 months. The eighth participant has not yet reached the six-month post-treatment date.
-
As of the safety data cut-off date of
Nov. 26, 2019 , there have been no safety events attributed to AVR-RD-01 drug product in either the Phase 1 or Phase 2 trial. Through the safety data cut-off date, two serious adverse events (SAEs) have been reported in the Phase 1 trial and four SAEs have been reported in the Phase 2 trial. The fourth Phase 2 patient, who was dosed after the safety data cut-off date, has reported an SAE, which was not attributed to AVR-RD-01 and subsequently resolved. Across both studies, each of the SAEs has been consistent with the underlying disease or pre-existing conditions, stem cell mobilization or conditioning regimen. Adverse events (AEs) reported did not suggest any unexpected safety signals or trends.
AVR-RD-01 Phase 1 trial in Fabry disease: Interim data continue to support potential first-line use
- Four of five patients had plasma lyso-Gb3 levels reduced between 26 and 47 percent compared to baseline levels measured while on enzyme replacement therapy (ERT). The fifth patient’s plasma lyso-Gb3 level remains within the range for the Fabry disease patients on ERT observed in this study and he remains off ERT.
- Overall, three of the five Phase 1 patients have discontinued ERT and all three remain off ERT at six, 14 and 15 months after dosing.
AVR-RD-01 Phase 2 trial in Fabry disease: Interim data support potential first-line use
- Data show increased leukocyte and plasma enzyme activity in the first three Phase 2 patients, suggesting production of an endogenous supply of functional alpha-galactosidase (AGA) enzyme sustained at nine, 12 and 18 months, respectively, after patient dosing.
- Two of these three patients have also demonstrated associated decreased plasma lyso-Gb3 levels, a key biomarker for monitoring Fabry disease, sustained below their baseline at six and 18 months, respectively, after dosing. As expected, the third Phase 2 patient, a cardiac variant who does not have classic Fabry disease and has very low baseline levels of lyso-Gb3, did not show a substantial decrease in plasma lyso-Gb3 levels.
- Cardiac and kidney function measures in the Phase 2 trial remained within the normal range for patients who had available 12-month data.
AVR-RD-04 Phase 1/2 trial in cystinosis: Three-month data from first patient suggest positive trends across multiple measures
-
No safety events or SAEs attributed to the investigational drug product were reported as of the
Jan. 27, 2020 safety data cut-off date. AEs reported did not suggest any unexpected safety signals or trends and was consistent with the conditioning regimen and the underlying disease. - Three months following administration of the investigational gene therapy, the first patient had a peripheral blood VCN of 2.0.
- Average granulocyte cystine level – one of the trial’s primary endpoints – decreased from 7.8 nmol half cystine/mg protein two weeks after cysteamine discontinuation to 1.5 nmol half cystine/mg protein at three months post-gene therapy.
-
The
U.S. Food and Drug Administration (FDA) granted orphan drug designation for AVR-RD-04 inMarch 2020 .
AVR-RD-02 Phase 1/2 trial in Gaucher disease: Expect to dose first patient in Q2 2020
- Enrolled first patient.
-
Received notice of clearance from the FDA regarding Investigational New Drug (IND) application for AVR-RD-02 for the treatment of Gaucher disease in
January 2020 . -
Recruiting participants in
Australia andCanada , with additionalU.S. sites planned.
plato gene therapy platform debut: Presented one-month data for fourth patient in Phase 2 Fabry trial (FAB-201)
-
Shared preliminary data from the first drug product produced using plato.
- Enzyme activity and transduction efficiency were 2.2 times higher than the mean of these measures for the drug product for the first three patients dosed in FAB-201.
- VCN was 1.8 times higher than the mean of this measure for the drug product for the first three patients dosed in FAB-201.
- At one month following administration of the plato-produced investigational gene therapy, plasma AGA enzyme activity level was 4.0 times higher for the first patient dosed using the plato platform in the Company’s FAB-201 clinical trial than the mean plasma enzyme activity level of the first three patients in the same trial at the same timepoint.
Strengthened balance sheet and extended anticipated cash runway into Q2 2022
-
In
February 2020 , the company raised gross proceeds of$100 million through a follow-on common stock offering. -
Based on the company’s current operating plan,
AVROBIO expects its cash and cash equivalents as ofDec. 31, 2019 , together with the net proceeds from theFebruary 2020 follow-on common stock offering, will enable the company to fund its operating expenses and capital expenditure requirements into Q2 2022.
Fourth Quarter and Fiscal Year 2019 Financial Results
Research and development expenses were
General and administrative expenses were
As of
About
Our mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease.
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 prospective product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and 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, the expected safety profile of our investigational gene therapies, and statements regarding our financial and cash position and expected cash runway. 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 will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of
CONDENSED CONSOLIDATED BALANCE SHEETS |
||||||
(In thousands) |
||||||
(Unaudited) |
||||||
|
|
|||||
2019 |
2018 |
|||||
Cash and cash equivalents |
$ |
187,043 |
$ |
126,302 |
||
Prepaid expenses and other current assets |
|
8,658 |
|
3,718 |
||
Property and equipment, net |
|
3,696 |
|
2,634 |
||
Other assets |
|
1,117 |
|
825 |
||
Total assets |
$ |
200,514 |
$ |
133,479 |
||
Accounts payable |
$ |
3,949 |
$ |
2,784 |
||
Accrued expenses and other current liabilities |
|
10,068 |
|
7,822 |
||
Deferred rent, net of current portion |
|
484 |
|
689 |
||
Total liabilities |
|
14,501 |
|
11,295 |
||
|
|
|
||||
Total stockholders’ equity |
|
186,013 |
|
122,184 |
||
Total liabilities and stockholders’ equity |
$ |
200,514 |
$ |
133,479 |
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS |
||||||||||||||||
(In thousands, except share and per share data) |
||||||||||||||||
(Unaudited) |
||||||||||||||||
Three Months Ended |
Twelve Months Ended |
|||||||||||||||
2019 |
2018 |
2019 |
2018 |
|||||||||||||
Operating expenses: |
|
|||||||||||||||
Research and development |
$ |
17,219 |
|
$ |
12,809 |
|
$ |
54,974 |
|
$ |
35,095 |
|
||||
General and administrative |
|
6,214 |
|
|
|
3,867 |
|
|
|
20,835 |
|
|
|
11,148 |
|
|
Total operating expenses |
|
23,433 |
|
|
|
16,676 |
|
|
|
75,809 |
|
|
|
46,243 |
|
|
|
|
|
|
|
|
|
||||||||||
Loss from operations |
|
(23,433 |
) |
|
|
(16,676 |
) |
|
|
(75,809 |
) |
|
|
(46,243 |
) |
|
Total other income (expense), net |
|
771 |
|
|
|
655 |
|
|
|
2,844 |
|
|
|
(118 |
) |
|
Net loss |
$ |
(22,662 |
) |
$ |
(16,021 |
) |
$ |
(72,965 |
) |
$ |
(46,361 |
) |
||||
|
|
|
|
|
|
|
|
|||||||||
Reconciliation of net loss to net loss attributed to common stockholders: Net loss |
$ |
(22,662 |
) |
|
$ |
(16,021 |
) |
|
$ |
(72,965 |
) |
|
$ |
(46,361 |
) |
|
Accretion of issuance costs on redeemable convertible preferred stock |
|
- |
|
|
|
- |
|
|
|
- |
|
|
|
(2,243 |
) |
|
Net loss attributable to common stockholders – basic and diluted |
$ |
(22,662 |
) |
|
$ |
(16,021 |
) |
|
$ |
(72,965 |
) |
|
$ |
(48,604 |
) |
|
|
||||||||||||||||
Net loss per share attributable to common stockholders — basic and diluted |
$ |
(0.72 |
) |
$ |
(0.67 |
) |
$ |
(2.66 |
) |
$ |
(3.62 |
) |
||||
Weighted-average number of common shares used in computing net loss per share attributable to common stockholders—basic and diluted |
|
31,628,930 |
|
|
23,791,495 |
|
|
27,432,489 |
|
|
13,435,478 |
|
View source version on businesswire.com: https://www.businesswire.com/news/home/20200316005179/en/
Investor Contact:
Westwicke, an
339-970-2843
chris.brinzey@westwicke.com
Media Contact:
Ten
857-559-3397
tom@tenbridgecommunications.com
Source: