AGTC Announces Financial Results and Business Update for the Quarter Ended March 31, 2021
- Reported 50% response rate in visual sensitivity among patients in highest dose groups in ongoing Phase 1/2 clinical trial of its XLRP gene therapy candidate for patients who met inclusion criteria for Skyline and Vista trials -
- Company on track to provide multiple data readouts for its XLRP and ACHM clinical programs in 2021 and 2022 -
- Company plans to lease 21,000 square foot build-to-suit cGMP manufacturing facility adjacent to its
- Company to host conference call and webcast today at 4:30 p.m. ET -
“The positive data from our ongoing Phase 1/2 XLRP clinical trial and planned expansion of our manufacturing and analytics capabilities give us tremendous momentum as we seek to advance our XLRP candidate toward commercialization,” said
X-linked Retinitis Pigmentosa (XLRP)
The Company also reported preliminary 24-month data for three of the seven patients in Group 4 who were evaluable at this time point. Two of these three patients, both of whom were responders at 12 months, continued to be responders at 24 months (one by the 7dB change in at least 5 loci criteria and the other with a statistically significant improvement in retinal sensitivity in the treated eye compared with the untreated eye). These results provide preliminary evidence of continued durability. The third patient was not a responder at 12 or 24 months. To the best of the Company’s knowledge, this is the first XLRP gene therapy clinical trial to demonstrate durability of response at this time point.
Consistent with previously reported 6-month data from Groups 2, 4, 5 and 6, Best Corrected Visual Acuity (BCVA) assessments at 12 months in these groups continued to provide supportive evidence of improved visual acuity in these patients, with a statistically significant difference between the treated and untreated eyes. This type of improvement has not been reported in other XLRP clinical trials and the Company believes that these data, together with the favorable safety profile, differentiate its XLRP candidate from competitors.
The Company’s XLRP candidate, which is administered via subretinal injection, has best-in-class potential that may provide significant benefit to patients with XLRP. The Company expects to:
- Present 12-month trial results from the ongoing Phase 1/2 clinical trial at the
American Academy of OphthalmologyAnnual Meeting in November 2021;
- Provide Skyline trial results from the 3-month masked interim analysis in 4Q 2021;
- Provide Skyline trial results from the 12-month data in 3Q 2022; and
- Provide Vista trial results from the 6-month masked interim analysis in 4Q 2022.
For ACHMA3, of the 16 patients in the four highest dose groups, three patients showed improvements in visual sensitivity in the treated area, as measured by static perimetry. No consistent results were seen in other dose groups. None of these three patients with improvements in visual sensitivity had evaluable mfERGs.
AGTC currently plans to focus on completing enrollment of pediatric patients in the two highest dose groups in its ACHMB3 and ACHMA3 trials, subject to potential delays caused by the COVID-19 pandemic, and to follow all patients through 12 months. The Company expects to:
- Provide 12-month data from the adult patients in both trials in 2Q 2021; and
- Provide preliminary 3-month data from the pediatric patients in both trials in 4Q 2021.
Last week, the Company announced that it had initiated plans to lease a build-to-suit 21,000 square foot current Good Manufacturing Practices (cGMP) manufacturing and quality control facility adjacent to its
Financial Results for the Three and Nine Months Ended
Revenue: There was no revenue for the three and nine months ended
R&D Expenses: Research and development expenses for the three and nine months ended
G&A Expenses: General and administrative expenses for the three and nine months ended
Investment Income, net: Investment income, net for the three and nine months ended
Interest Expense: Interest expense for the three and nine months ended
Net Loss: The Company’s net loss for the three and nine months ended
Financial Guidance: As of
R&D Day Information
AGTC plans to review the data and provide the latest updates on the XLRP and achromatopsia clinical programs at an R&D Day on
Chief Financial Officer Transition
AGTC announced today that
"Bill has been instrumental in leading our finance team through multiple rounds of financings, enabling us to advance our best-in-class clinical and preclinical programs," said
“I am grateful for my time at AGTC and I have really enjoyed working with Sue and the entire team at AGTC,” said
The Company has initiated a search process to identify a new Chief Financial Officer.
Conference Call and Webcast
AGTC will host a conference call and webcast to discuss financial results for the quarter ended March 31, 2021 today at 4:30 p.m. ET. To access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US). A live webcast will be available in the Events and Presentations section of AGTC’s Investor Relations site at http://ir.agtc.com/events-and-presentations. Please log in approximately 10 minutes prior to the scheduled start time.
The archived webcast will be available in the Events and Presentations section of the Company's website.
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.
XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th or 5th decade of life. AGTC was granted
ACHM is an inherited retinal disease, which is present from birth and is characterized by the lack of cone photoreceptor function. The condition results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by ACHM, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind.
This press release contains forward-looking statements that reflect AGTC's plans, estimates, assumptions and beliefs, including statements regarding the projected timing for its planned Vista (Phase 2/3 XLRP) and Skyline (Expanded Phase 1/2 XLRP) clinical trials, the timing for reporting data in both its Skyline and Vista trials, the potential of its ACHM clinical programs and its ability to enroll pediatric patients, and AGTC’s planned build-to-suit lease, the expected timing for the build-out of the facility subject to the lease and its potential to support AGTC’s pipeline programs. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic, including the impact on AGTC’s ability to enroll patients. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as "anticipates," "believes," "could," "seeks," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," "would" or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: risks related to new construction; gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on the Company’s business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading "Risk Factors" in AGTC’s most recent annual and subsequently filed quarterly reports filed with the
|CONDENSED BALANCE SHEETS|
|In thousands, except per share data|
|Cash and cash equivalents||$||89,531||$||38,463|
|Prepaid and other current assets||1,997||2,506|
|Total current assets||113,024||82,964|
|Property and equipment, net||4,019||4,311|
|Intangible assets, net||1,291||1,098|
|Right-of-use assets – operating leases||3,158||3,422|
|Right-of-use asset – finance lease||46||80|
|LIABILITIES AND STOCKHOLDERS’ EQUITY|
|Accrued and other liabilities||13,504||10,502|
|Lease liabilities – operating||1,072||1,058|
|Lease liability – finance||51||48|
|Total current liabilities||16,553||12,963|
|Lease liabilities – operating, net of current portion||3,540||4,070|
|Lease liability – finance, net of current portion||—||38|
|Long-term debt, net of debt discounts and deferred financing fees||9,929||9,677|
|Additional paid-in capital||324,302||252,519|
|Total stockholders’ equity||97,000||71,016|
|Total liabilities and stockholders’ equity||$||129,666||$||100,319|
|CONDENSED STATEMENTS OF OPERATIONS|
|In thousands, except per share data||2021||2020||2021||2020|
|Collaboration and milestone revenue||$||—||$||—||$||—||$||2,297|
|Research and development||10,960||8,308||34,397||25,325|
|General and administrative and other||3,528||3,134||10,268||9,490|
|Total operating expenses||14,488||11,442||44,665||34,815|
|Loss from operations||(14,488||)||(11,442||)||(44,665||)||(32,362||)|
|Other income (expense), net:|
|Investment income, net||13||281||106||1,063|
|Total other income (expense), net||(317||)||285||(891||)||1,063|
|Loss before provision for income taxes||(14,805||)||(11,157||)||(45,556||)||(31,299||)|
|Provision for income taxes||21||21||62||63|
|Loss before equity in net losses of an affiliate||(14,826||)||(11,178||)||(45,618||)||(31,362||)|
|Equity in net losses of an affiliate||(25||)||(11||)||(75||)||(27||)|
|Weighted average shares outstanding:|
|Net loss per common share:|
Chief Financial Officer
T: (617) 843-5728
Chief Business Officer
T: (617) 413-2754
Source: Applied Genetic Technologies Corporation