Sangamo Therapeutics Reports Recent Business Highlights and Fourth Quarter and Full Year 2022 Financial Results
Conference Call and Webcast Scheduled for
“2022 was a year of important clinical and non-clinical milestones across our pipeline. We strengthened that momentum today by releasing compelling data from our Phase 1/2 Fabry disease study, supporting a potential best-in-class product profile,” said
Fourth Quarter Updates and Recent Business Highlights
Fabry disease – Reported compelling updated preliminary Phase 1/2 data showing extended clinical benefit; reported kidney biopsy data demonstrating 78% globotriaosylceramide (Gb3) substrate clearance at 6-months; Phase 3 trial anticipated to start by end of 2023.
-
Presented updated preliminary data as of the
October 20, 2022 cutoff date from the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, our wholly owned gene therapy product candidate for the treatment of Fabry disease at the 19th Annual WORLDSymposium. -
Sustained, elevated expression of alpha-galactosidase A (α-Gal A) activity observed in 13 patients for over two years for the longest treated patient as of the
November 15, 2022 supplemental cutoff date. - No prophylactic corticosteroids or other immune modulating agents have been administered.
- All five patients who began the dose escalation phase on enzyme replacement therapy (ERT) had been successfully withdrawn from ERT and remain off ERT today.
- Announced first available kidney biopsy data demonstrating 78% Gb3 substrate clearance at 6-months and 77% reduction in urine podocyte loss in a treated patient with high baseline plasma globotriaosylsphingosine (lyso-Gb3) levels.
-
Reported a clinically meaningful and statistically significant increase in mean general health scores measured across all patients treated in dose escalation phase, as measured by the
SF-36 General Health Survey . -
As of the
October 20, 2022 cutoff date, naïve and pseudo-naïve patients treated in the dose expansion and escalation phases with baseline lyso-Gb3 levels above 80 ng/mL experienced a 40-65% reduction. For the first time, and at the highest dose, a 54% reduction in lyso-Gb3 levels was observed where baseline levels started below 25 ng/mL. -
As of the
October 20, 2022 cutoff date, isaralgagene civaparvovec remained generally well tolerated with no treatment-related adverse events greater than Grade 2 and no treatment-related serious adverse events. -
Since the
October 20, 2022 cutoff date, four additional patients have been dosed in the expansion phase to achieve a total of 17, and a further two patients have been withdrawn from ERT. - Dosing of remaining patients in the expansion phase of the Phase 1/2 STAAR study is ongoing, with a total of 20 sites active and recruiting. We expect dosing to conclude by the end of 2023.
- Preparations for a potential Phase 3 trial actively progress, with a trial start anticipated by the end of 2023, depending on regulatory interactions. Dosing of the first patient is expected to start as early as the first part of 2024.
Renal Transplant Rejection – Progressed manufacturing and clinical activities ahead of anticipated Q2 dosing; potential dose escalation acceleration scenarios in discussion with regulators.
- Progressed manufacturing and clinical activities in the Phase 1/2 STEADFAST study evaluating TX200, our wholly owned autologous CAR-Treg cell therapy treating patients receiving an HLA-A2 mismatched kidney from a living donor.
- The product candidate continues to be generally well tolerated in both patients dosed to date.
- Completed manufacturing of the dose for the final patient in the first cohort, who recently received a kidney transplant. Dosing is expected early in the second quarter of 2023.
- Progressed clinical and manufacturing activities for the first patient in the second cohort, with dosing expected in the summer of 2023.
- Opportunities to accelerate dose escalation are being explored with regulators.
Sickle cell disease – Presented promising 6-month data for first patient dosed with a product candidate manufactured using improved methods; discontinuing additional material investments in Phase 3 planning in order to prioritize capital allocation; intend to seek a potential collaboration partner.
-
Presented promising data from the Phase 1/2 PRECIZN-1 study of BIVV003, a zinc finger nuclease gene-edited cell therapy candidate for the treatment of sickle cell disease at the 64th
American Society of Hematology (ASH) Annual Meeting. The first patient to receive a product candidate manufactured using improved methods achieved fetal hemoglobin levels of 45% and total hemoglobin of 12.4 g/dL at week 26. - Progressed clinical and manufacturing activities for patient 7.
- Agreed on Phase 3 trial design, Chemistry, Manufacturing and Controls (CMC) package and other requirements with the FDA.
- Progressed additional manufacturing improvements which we believe have the potential to further strengthen clinical outcomes and reduce manufacturing costs in a potential Phase 3 trial.
- Announced strategic decision to halt further material investments in the program beyond completion of Phase 1/2 study, to redeploy resources to Fabry and TX200 programs, and to seek a potential collaboration partner to progress this asset to a potential Phase 3 trial.
Hemophilia A – Dosing in Phase 3 AFFINE trial expected to be complete by end of Q1 2023; pivotal data read-out expected in first half of 2024; BLA submission anticipated in second half of 2024.
- Dosing progressed in the Phase 3 AFFINE trial of giroctocogene fitelparvovec, an investigational gene therapy we are developing with Pfizer for patients with moderately severe to severe hemophilia A.
- Dosing to support primary analysis is expected to be complete by the end of Q1 2023.
- A pivotal readout is expected in the first half of 2024, with Pfizer anticipating a BLA submission in the second half of 2024.
Preclinical Programs – Progressed preclinical programs in CAR-Tregs and epigenetic regulation of the central nervous system (CNS), alongside advancements in the zinc finger platform and AAV delivery capabilities.
- By the end of 2023, we expect to announce, and reveal data from, our next wholly owned CNS epigenetic regulation program.
- By the end of 2024, we anticipate submitting two new IND applications from our second wave CAR-Treg and CNS programs.
Fourth Quarter and Full Year 2022 Financial Results
Consolidated net loss for the fourth quarter ended
Revenues
Revenues for the fourth quarter ended
The decrease of
Revenues were
The increase of
GAAP and Non-GAAP operating expenses
Three Months Ended | Year Ended | |||||||||||||||
(In millions) |
2022 |
2021 |
2022 |
2021 |
||||||||||||
Research and development |
$ |
66.2 |
|
$ |
51.8 |
|
$ |
249.9 |
|
$ |
230.8 |
|
||||
General and administrative |
|
16.4 |
|
|
16.1 |
|
|
62.7 |
|
|
63.2 |
|
||||
Total operating expenses |
|
82.6 |
|
|
67.9 |
|
|
312.6 |
|
|
294.0 |
|
||||
Stock-based compensation expense |
|
(8.3 |
) |
|
(8.1 |
) |
|
(31.7 |
) |
|
(33.0 |
) |
||||
Non-GAAP operating expenses |
$ |
74.3 |
|
$ |
59.8 |
|
$ |
280.9 |
|
$ |
261.0 |
|
Total operating expenses on a GAAP basis for the fourth quarter ended
The increase in total operating expenses on a GAAP basis was primarily attributable to increased spending on our internal infrastructure and external services as we progress our clinical trials coupled with higher headcount related personnel costs, and an adjustment in 2021 related to dissolution of the repayment obligation of a grant from
Total operating expenses on a GAAP basis in 2022 were
The increase in total operating expenses in the full year on a GAAP basis was primarily driven by increased spending on our internal infrastructure and external services as we progress our clinical trials coupled with higher headcount related personnel costs and our ongoing collaborations, and an adjustment in 2021 related to dissolution of the repayment obligation of a grant from
Cash, cash equivalents and marketable securities
Cash, cash equivalents and marketable securities as of
Initial Financial Guidance for 2023
On a GAAP basis, we expect total operating expenses in the range of approximately
We expect non-GAAP total operating expenses, excluding estimated non-cash stock-based compensation expense of approximately
Upcoming Events
Sangamo plans to participate in the following events:
Scientific / Medical Conferences
-
19th Annual WORLDSymposium,
February 22-24, 2023 . Platform presentation taking placeFriday February 24 , during a session from8:00-9:00am Eastern Time and poster session3:00-4:00pm Eastern Time (Poster Ref: 169).
Investor Conferences
-
Cowen 43rd Annual
Health Care Conference ,March 6, 2023 -
Barclays Global Healthcare Conference ,March 15, 2023 -
2023
Bank of America Global Healthcare Conference ,May 9-11, 2023 -
2023
RBC Global Healthcare Conference ,May 16-17, 2023 -
Stifel 2023 Tailoring Genes: Genetic Medicines Day,
May 30, 2023
Access links for available webcasts for these investor conferences will be available on the
Conference Call to Discuss Fourth Quarter and Full Year 2022 Results
The Sangamo management team will discuss these results on a conference call today,
Participants should register for, and access, the call using this link. While not required, it is recommended you join 10 minutes prior to the event start. Once registered, participants will be given the option to either dial into the call with the number and unique passcode provided or to use the dial-out option to connect their phone instantly.
An updated corporate presentation is available in the Investors and Media section under Presentations.
The link to access the live webcast can also be found on the
About
Our Annual Report on Form 10-K summarizing the updated preliminary results from the Phase 1/2 STAAR clinical study in more detail will be filed by Sangamo, and this press release is subject to the further detail provided in the form 10-K.
Forward-Looking Statements
This press release contains forward-looking statements regarding our current expectations. These forward-looking statements include, without limitation, statements relating to: the therapeutic and commercial potential of our product candidates, the anticipated plans and timelines of Sangamo and our collaborators for screening, enrolling and dosing patients in and conducting our ongoing and potential future clinical trials and presenting clinical data from our clinical trials, including expectations regarding the conclusion of dosing in our Phase 1/2 STAAR study, preparations and plans for patient dosing in the STEADFAST study and the potential for acceleration of the study timeline, updates regarding the PRECIZN-1 study, including the dosing of additional patients, the use of improved manufacturing methods and the potential impacts thereof, and plans to halt further investment in the BIVV003 and seek a potential partner for the program, the anticipated advancement of our product candidates to late-stage development, including potential future Phase 3 trials of isaralgagene civaparvovec and BIVV003, plans and timing regarding the completion of dosing in the phase 3 AFFINE trial, the availability and presentation of data from such trial, and plans for a BLA submission for giroctocogene fitelparvovec, expectations regarding advancement of our preclinical programs, including announcement of, and data from, such programs, as well as anticipated IND submissions related to such programs, our 2023 financial guidance related to GAAP and non-GAAP total operating expenses and stock-based compensation, our plans to participate in industry and investor conferences, and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to the effects of the COVID-19 pandemic and the impacts of the pandemic and other macroeconomic factors, including as a result of the ongoing conflict between
There can be no assurance that we and our collaborators will be able to develop commercially viable products. Actual results may differ materially from those projected in these forward-looking statements due to the risks and uncertainties described above and other risks and uncertainties that exist in the operations and business environments of Sangamo and our collaborators. These risks and uncertainties are described more fully in our
Non-GAAP Financial Measure
To supplement our financial results and guidance presented in accordance with GAAP, we present non-GAAP total operating expenses, which exclude stock-based compensation expense from GAAP total operating expenses. We believe that this non-GAAP financial measure, when considered together with our financial information prepared in accordance with GAAP, can enhance investors’ and analysts’ ability to meaningfully compare our results from period to period and to our forward-looking guidance, and to identify operating trends in our business. We have excluded stock-based compensation expense because it is a non-cash expense that may vary significantly from period to period as a result of changes not directly or immediately related to the operational performance for the periods presented. This non-GAAP financial measure is in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP. We encourage investors to carefully consider our results under GAAP, as well as our supplemental non-GAAP financial information, to more fully understand our business.
SELECTED CONSOLIDATED FINANCIAL DATA |
|||||||||||||||
(unaudited; in thousands, except per share data) |
|||||||||||||||
Statement of Operations Data: | |||||||||||||||
Three months ended | Year ended | ||||||||||||||
2022 |
2021 |
2022 |
2021 |
||||||||||||
Revenues |
$ |
27,230 |
|
$ |
27,986 |
|
$ |
111,299 |
|
$ |
110,701 |
|
|||
Operating expenses: |
|
- |
|
|
- |
|
|
- |
|
|
- |
|
|||
Research and development |
|
66,179 |
|
|
51,801 |
|
|
249,898 |
|
|
230,819 |
|
|||
General and administrative |
|
16,443 |
|
|
16,084 |
|
|
62,682 |
|
|
63,219 |
|
|||
Total operating expenses |
|
82,622 |
|
|
67,885 |
|
|
312,580 |
|
|
294,038 |
|
|||
Loss from operations |
|
(55,392 |
) |
|
(39,899 |
) |
|
(201,281 |
) |
|
(183,337 |
) |
|||
Interest and other income, net |
|
3,678 |
|
|
2,336 |
|
|
9,432 |
|
|
5,346 |
|
|||
Loss before income taxes |
|
(51,714 |
) |
|
(37,563 |
) |
|
(191,849 |
) |
|
(177,991 |
) |
|||
Income tax expense (benefit) |
|
259 |
|
|
(67 |
) |
|
429 |
|
|
306 |
|
|||
Net loss |
$ |
(51,973 |
) |
$ |
(37,496 |
) |
$ |
(192,278 |
) |
$ |
(178,297 |
) |
|||
Net loss attributable to non-controlling interest |
|
- |
|
|
- |
|
|
- |
|
|
(11 |
) |
|||
Net loss attributable to |
$ |
(51,973 |
) |
$ |
(37,496 |
) |
$ |
(192,278 |
) |
$ |
(178,286 |
) |
|||
Basic and diluted net loss per share attributable to |
$ |
(0.32 |
) |
$ |
(0.26 |
) |
$ |
(1.25 |
) |
$ |
(1.23 |
) |
|||
Shares used in computing basic and diluted net loss per share attributable to |
|
164,717 |
|
|
145,740 |
|
|
154,345 |
|
|
144,568 |
|
|||
Selected Balance Sheet Data: | |||||||||||||||
Cash, cash equivalents, and marketable securities |
$ |
307,477 |
|
$ |
464,717 |
|
|||||||||
Total assets |
$ |
562,509 |
|
$ |
721,923 |
|
|||||||||
Total stockholders' equity |
$ |
294,958 |
|
$ |
375,343 |
|
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Investor Relations & Media Inquiries
ir@sangamo.com
media@sangamo.com
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