Sangamo Reports Positive Data for Fabry Disease Treatment
Written by Emily J. Thompson, Senior Investment Analyst
Updated: 4d ago
0mins
Should l Buy SGMO?
Source: NASDAQ.COM
- Clinical Trial Results: Sangamo Therapeutics announced positive outcomes from its registrational Phase 1/2 STAAR study for Fabry disease treatment with ST-920, showing a mean annualized eGFR slope of 1.965 mL/min/1.73m² at 52 weeks across all patients, indicating improved kidney function that could enhance patient quality of life.
- Stable Cardiac Function: The study observed stable cardiac function over one year, with consistent cardiac structural stability across clinical and demographic subgroups, further supporting the safety and efficacy of ST-920.
- Durability of Effect: The treatment demonstrated sustained elevated alpha-galactosidase A (a-Gal A) activity for up to 4.5 years in the longest-treated patient, indicating long-lasting therapeutic effects that may provide a competitive edge in the market.
- Market Potential: According to Grandview Research, the global Fabry disease treatment market was valued at $2.54 billion in 2023 and is projected to reach $4.93 billion by 2030, growing at a CAGR of 9.9%, presenting significant commercial opportunities for Sangamo's product.
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Analyst Views on SGMO
Wall Street analysts forecast SGMO stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for SGMO is 3.75 USD with a low forecast of 1.00 USD and a high forecast of 10.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
4 Analyst Rating
1 Buy
3 Hold
0 Sell
Hold
Current: 0.353
Low
1.00
Averages
3.75
High
10.00
Current: 0.353
Low
1.00
Averages
3.75
High
10.00
About SGMO
Sangamo Therapeutics, Inc. is a genomic medicine company. The Company’s zinc finger (ZF) epigenetic regulators are ideally suited to potentially address neurological disorders and its capsid engineering platform has demonstrated the ability to expand delivery beyond available intrathecal delivery capsids, including in the central nervous system (CNS), in preclinical studies. Zinc finger technologies are derived from naturally occurring human proteins. They are composed of a DNA-binding domain, which binds with precision to any sequence of DNA, and various functional domains (such as nucleases and transcription factors) based on therapeutic needs. Its pipeline includes Chronic Neuropathic Pain - ST-503 and Isaralgagene civaparvovec (ST-920). ST-503 is an investigational epigenetic regulator for the treatment of intractable pain due to iSFN, a type of chronic neuropathic pain. ST-920 is a wholly owned gene therapy product candidate for the treatment of Fabry disease.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Sangamo Reports Positive Data for Fabry Disease Treatment
- Clinical Trial Results: Sangamo Therapeutics announced positive outcomes from its registrational Phase 1/2 STAAR study for Fabry disease treatment with ST-920, showing a mean annualized eGFR slope of 1.965 mL/min/1.73m² at 52 weeks across all patients, indicating improved kidney function that could enhance patient quality of life.
- Stable Cardiac Function: The study observed stable cardiac function over one year, with consistent cardiac structural stability across clinical and demographic subgroups, further supporting the safety and efficacy of ST-920.
- Durability of Effect: The treatment demonstrated sustained elevated alpha-galactosidase A (a-Gal A) activity for up to 4.5 years in the longest-treated patient, indicating long-lasting therapeutic effects that may provide a competitive edge in the market.
- Market Potential: According to Grandview Research, the global Fabry disease treatment market was valued at $2.54 billion in 2023 and is projected to reach $4.93 billion by 2030, growing at a CAGR of 9.9%, presenting significant commercial opportunities for Sangamo's product.
See More
Sangamo Therapeutics Shares Plunge Over 31%
- Significant Stock Drop: Sangamo Therapeutics shares fell over 31% in recent trading, indicating market concerns about the company's future prospects, which could impact investor confidence and financing capabilities.
- Strong Market Reaction: The sharp decline in stock price may be linked to recent financial data or disappointing R&D progress, leading investors to question the sustainability of its product pipeline, thereby intensifying selling pressure.
- Investor Sentiment Deteriorates: With the stock's steep decline, market perceptions of Sangamo's overall valuation and future growth potential have become increasingly pessimistic, potentially affecting its competitive position in the biotechnology sector.
- Increased Financing Challenges: The significant drop in stock price may pose higher costs and greater difficulties for Sangamo in future capital raising efforts, thereby limiting its ability to fund R&D and market expansion.
See More
Sangamo Therapeutics Shares Plunge 28% After $25M Stock Offering
- Stock Offering Impact: Sangamo Therapeutics priced an underwritten stock offering at $0.4719 per share for approximately 35.1 million shares and 17.8 million pre-funded warrants, leading to a ~28% drop in stock price on Tuesday, indicating negative market sentiment towards the financing.
- Warrant Adjustments: The company announced a reduction in the exercise price of certain outstanding warrants from $1.00 to $0.4719 and extended their term to five and a half years, aiming to boost investor confidence and improve liquidity.
- Executive Changes: The termination of Principal Financial Officer Prathyusha Duraibabu on February 3, with Nikunj Jain appointed as Interim CFO, may impact the continuity of the company's financial strategy during a critical period.
- Cash Flow Projections: Sangamo anticipates approximately $20.9 million in cash and cash equivalents by the end of 2025, highlighting the need for careful cash flow management to support ongoing operations post-offering.
See More
Sangamo Therapeutics Announces Pricing of Stock Offering
- Offering Size: Sangamo Therapeutics announced the pricing of 35,190,292 shares of common stock and 17,787,033 pre-funded warrants at $0.4719 per share, with expected gross proceeds of approximately $25 million aimed at enhancing working capital and general corporate purposes.
- Warrant Terms: The offering includes accompanying warrants with an exercise price of $0.4719, exercisable six months post-issuance and expiring in five and a half years, providing investors with flexible investment options that may enhance market appeal.
- Existing Warrant Adjustment: Sangamo agreed to reduce the exercise price of existing warrants held by investors from $1.00 to $0.4719 and extend their term to five and a half years, a move designed to bolster investor confidence and stimulate market activity.
- Underwriter Role: Cantor and Wells Fargo Securities are acting as joint book-running managers for the offering, ensuring a smooth issuance process while providing the company with professional market support and resources, further enhancing Sangamo's image in the capital markets.
See More
Sangamo Initiates BLA Submission for ST-920, Expected in Q2 2026
- Clinical Trial Success: Sangamo's STAAR study demonstrated a positive mean annualized eGFR slope at 52 weeks across all dosed patients, with the FDA agreeing to use this as an endpoint for accelerated approval, indicating ST-920's potential as an effective treatment for Fabry disease patients.
- Favorable Safety Profile: Isaralgagene civaparvovec exhibited a favorable safety and tolerability profile in clinical trials, suggesting its potential as a one-time gene therapy that can provide durable clinical benefits, surpassing current treatment standards for Fabry disease.
- Accelerated Approval Pathway: Sangamo expects to complete its Biological License Application (BLA) submission to the FDA in the second quarter of 2026 using a rolling submission approach, allowing for ongoing review of completed modules, thereby expediting the approval process to meet urgent patient needs.
- Regulatory Support: Isaralgagene civaparvovec has received Orphan Drug, Fast Track, and RMAT designations from the FDA, along with Orphan Medicinal Product designation from the European Medicines Agency, highlighting its potential value and regulatory recognition in global markets, further enhancing Sangamo's competitive position.
See More
Sangamo Initiates BLA Submission for ST-920, Expected Completion in Q2 2026
- Clinical Trial Results: Sangamo's STAAR study demonstrated a positive mean annualized eGFR slope at 52 weeks across all dosed patients, with the FDA agreeing to use this as an endpoint for accelerated approval, indicating ST-920's potential in treating Fabry disease.
- Safety Profile: Isaralgagene civaparvovec has shown a favorable safety and tolerability profile, expected to provide a safe and durable treatment option for Fabry patients, significantly improving multi-organ clinical benefits.
- Submission Timeline: Sangamo plans to complete its Biological License Application (BLA) submission in Q2 2026 using a rolling submission approach, allowing the FDA to review completed modules continuously, thus expediting the approval process.
- Regulatory Support: The therapy has received Orphan Drug, Fast Track, and RMAT designations from the FDA, reflecting regulatory recognition of its potential market value and enhancing Sangamo's competitive position in the gene therapy sector.
See More
Sangamo Reports Positive Data for Fabry Disease Treatment
- Clinical Trial Results: Sangamo Therapeutics announced positive outcomes from its registrational Phase 1/2 STAAR study for Fabry disease treatment with ST-920, showing a mean annualized eGFR slope of 1.965 mL/min/1.73m² at 52 weeks across all patients, indicating improved kidney function that could enhance patient quality of life.
- Stable Cardiac Function: The study observed stable cardiac function over one year, with consistent cardiac structural stability across clinical and demographic subgroups, further supporting the safety and efficacy of ST-920.
- Durability of Effect: The treatment demonstrated sustained elevated alpha-galactosidase A (a-Gal A) activity for up to 4.5 years in the longest-treated patient, indicating long-lasting therapeutic effects that may provide a competitive edge in the market.
- Market Potential: According to Grandview Research, the global Fabry disease treatment market was valued at $2.54 billion in 2023 and is projected to reach $4.93 billion by 2030, growing at a CAGR of 9.9%, presenting significant commercial opportunities for Sangamo's product.
See More
Sangamo Therapeutics Shares Plunge Over 31%
- Significant Stock Drop: Sangamo Therapeutics shares fell over 31% in recent trading, indicating market concerns about the company's future prospects, which could impact investor confidence and financing capabilities.
- Strong Market Reaction: The sharp decline in stock price may be linked to recent financial data or disappointing R&D progress, leading investors to question the sustainability of its product pipeline, thereby intensifying selling pressure.
- Investor Sentiment Deteriorates: With the stock's steep decline, market perceptions of Sangamo's overall valuation and future growth potential have become increasingly pessimistic, potentially affecting its competitive position in the biotechnology sector.
- Increased Financing Challenges: The significant drop in stock price may pose higher costs and greater difficulties for Sangamo in future capital raising efforts, thereby limiting its ability to fund R&D and market expansion.
See More
Sangamo Therapeutics Shares Plunge 28% After $25M Stock Offering
- Stock Offering Impact: Sangamo Therapeutics priced an underwritten stock offering at $0.4719 per share for approximately 35.1 million shares and 17.8 million pre-funded warrants, leading to a ~28% drop in stock price on Tuesday, indicating negative market sentiment towards the financing.
- Warrant Adjustments: The company announced a reduction in the exercise price of certain outstanding warrants from $1.00 to $0.4719 and extended their term to five and a half years, aiming to boost investor confidence and improve liquidity.
- Executive Changes: The termination of Principal Financial Officer Prathyusha Duraibabu on February 3, with Nikunj Jain appointed as Interim CFO, may impact the continuity of the company's financial strategy during a critical period.
- Cash Flow Projections: Sangamo anticipates approximately $20.9 million in cash and cash equivalents by the end of 2025, highlighting the need for careful cash flow management to support ongoing operations post-offering.
See More
Sangamo Therapeutics Announces Pricing of Stock Offering
- Offering Size: Sangamo Therapeutics announced the pricing of 35,190,292 shares of common stock and 17,787,033 pre-funded warrants at $0.4719 per share, with expected gross proceeds of approximately $25 million aimed at enhancing working capital and general corporate purposes.
- Warrant Terms: The offering includes accompanying warrants with an exercise price of $0.4719, exercisable six months post-issuance and expiring in five and a half years, providing investors with flexible investment options that may enhance market appeal.
- Existing Warrant Adjustment: Sangamo agreed to reduce the exercise price of existing warrants held by investors from $1.00 to $0.4719 and extend their term to five and a half years, a move designed to bolster investor confidence and stimulate market activity.
- Underwriter Role: Cantor and Wells Fargo Securities are acting as joint book-running managers for the offering, ensuring a smooth issuance process while providing the company with professional market support and resources, further enhancing Sangamo's image in the capital markets.
See More
Sangamo Initiates BLA Submission for ST-920, Expected in Q2 2026
- Clinical Trial Success: Sangamo's STAAR study demonstrated a positive mean annualized eGFR slope at 52 weeks across all dosed patients, with the FDA agreeing to use this as an endpoint for accelerated approval, indicating ST-920's potential as an effective treatment for Fabry disease patients.
- Favorable Safety Profile: Isaralgagene civaparvovec exhibited a favorable safety and tolerability profile in clinical trials, suggesting its potential as a one-time gene therapy that can provide durable clinical benefits, surpassing current treatment standards for Fabry disease.
- Accelerated Approval Pathway: Sangamo expects to complete its Biological License Application (BLA) submission to the FDA in the second quarter of 2026 using a rolling submission approach, allowing for ongoing review of completed modules, thereby expediting the approval process to meet urgent patient needs.
- Regulatory Support: Isaralgagene civaparvovec has received Orphan Drug, Fast Track, and RMAT designations from the FDA, along with Orphan Medicinal Product designation from the European Medicines Agency, highlighting its potential value and regulatory recognition in global markets, further enhancing Sangamo's competitive position.
See More
Sangamo Initiates BLA Submission for ST-920, Expected Completion in Q2 2026
- Clinical Trial Results: Sangamo's STAAR study demonstrated a positive mean annualized eGFR slope at 52 weeks across all dosed patients, with the FDA agreeing to use this as an endpoint for accelerated approval, indicating ST-920's potential in treating Fabry disease.
- Safety Profile: Isaralgagene civaparvovec has shown a favorable safety and tolerability profile, expected to provide a safe and durable treatment option for Fabry patients, significantly improving multi-organ clinical benefits.
- Submission Timeline: Sangamo plans to complete its Biological License Application (BLA) submission in Q2 2026 using a rolling submission approach, allowing the FDA to review completed modules continuously, thus expediting the approval process.
- Regulatory Support: The therapy has received Orphan Drug, Fast Track, and RMAT designations from the FDA, reflecting regulatory recognition of its potential market value and enhancing Sangamo's competitive position in the gene therapy sector.
See More
