On May 4, 2023 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, reported its financial results for the quarter ended March 31, 2023 and reaffirmed its financial guidance for 2023 (Press release, Ultragenyx Pharmaceutical, MAY 4, 2023, View Source [SID1234631059]).

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"In the first quarter we continued the strong growth in product revenue and advanced our clinical programs including completion of the Phase 2 portion of our pivotal study of UX143 in osteogenesis imperfecta. We are analyzing the data and look forward to sharing the results soon. Once completed, we will initiate enrollment of the Phase 3 portion in mid-2023," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "We are also making rapid progress in our Phase 1/2 study of GTX-102 in Angelman syndrome, dosing in the expansion cohorts and activating sites in new countries."

First Quarter 2023 Selected Financial Data Tables and Financial Results

Revenues (dollars in thousands), (unaudited)
Three Months Ended March 31,
2023 2022
Crysvita
Revenue in profit-share territory $ 49,906 $ 45,164
Product revenue 21,234 9,394
Non-cash royalty revenue in European territory 4,882 4,838
Total Crysvita revenue 76,022 59,396
Dojolvi 14,303 12,429
Mepsevii 8,480 4,861
Evkeeza 212 —
Daiichi Sankyo 1,479 3,249
Total revenues $ 100,496 $ 79,935

Net Revenues
Ultragenyx reported $100.5 million in total revenue for the first quarter of 2023, which represents 26% growth compared to the first quarter 2022. The growth is primarily driven by increased demand for Crysvita in Latin America and steady growth from the other regions and products. The first quarter 2023 also included $1.5 million of revenue from the technology transfer which was completed during the quarter as part of the collaboration and license agreement with Daiichi Sankyo.

Selected Financial Data (dollars in thousands, except per share amounts), (unaudited)
Three Months Ended March 31,
2023 2022
Total revenues $ 100,496 $ 79,935
Operating expense:
Cost of sales 12,257 6,100
Research and development 165,698 143,155
Selling, general and administrative 76,646 67,312
Total operating expense 254,601 216,567
Net loss $ (163,972 ) $ (152,320 )
Net loss per share, basic and diluted $ (2.33 ) $ (2.19 )

Operating Expenses
Total operating expenses for the first quarter of 2023 were $254.6 million, including non-cash stock-based compensation of $31.9 million. In 2023, annual operating expenses are expected to decrease as the company manages headcount and increases operational leverage while executing on high-value programs.

Net Loss
For the first quarter of 2023, Ultragenyx reported net loss of $164.0 million, or $2.33 per share basic and diluted, compared with a net loss for the first quarter of 2022 of $152.3 million, or $2.19 per share, basic and diluted.

Cash, Cash Equivalents and Marketable Debt Securities
Cash, cash equivalents, and marketable debt securities were approximately $714.6 million as of March 31, 2023.

2023 Financial Guidance
For the full year 2023, the company reaffirms:

Total revenue in the range of $425 million to $450 million
Crysvita revenue in the range of $325 million to $340 million. This includes all regions where Ultragenyx will recognize revenue, including the royalties in Europe, which have been ongoing, and the royalties in North America, which began in April 2023.
Dojolvi revenue in the range of $65 million to $75 million
Net Cash Used in Operations is expected to be less than $400 million
Recent Updates and Clinical Milestones

UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): Phase 2 enrollment complete; Phase 2 data and Phase 3 initiation expected in mid-2023
Ultragenyx is continuing to dose patients in the Phase 2/3 Orbit study of UX143 in pediatric and adult patients with OI aged five to <26 years. All patients have completed the Phase 2 portion of the study with data expected in mid-2023. These data are expected to include changes in bone biomarkers response and some data on bone mineral density that will be used to establish the dosing algorithm for the Phase 3 portion of the study. The Phase 3 is expected to initiate in mid-2023.

In addition, in the second quarter of 2023, Ultragenyx intends to initiate Cosmic, a randomized study in children under age five with serious bone disease, which compares bisphosphonates to UX143. Younger pediatric patients with OI often have a much higher fracture rate than other age groups driving clinical urgency for better treatment options. The primary endpoint is expected to be the annualized rate of fractures.

GTX-102 antisense oligonucleotide for Angelman syndrome: Phase 1/2 dosing in the expansion cohorts

Outside of the U.S., the dose escalation phase of this study has been completed and the company recently transitioned to dosing patients in the expansion cohorts, which are designed to verify the GTX-102 dose and treatment regimen that will be used in the Phase 3 program. The study has also expanded geographically with multiple new sites activated in Europe and Australia. Approximately 40 patients will be enrolled across Cohort A (ages four to

Patients from the dose escalation cohorts continue to exhibit encouraging dose and time-dependent clinical activity following longer-term treatment and maintenance dosing. No additional treatment-related serious adverse events or lower extremity weakness adverse events have occurred since our prior update in January 2023. As of May 2023, 14 patients have had at least six months of exposure to GTX-102 including nine patients with more than one year of continuous therapy. The next data update is expected in the second half of 2023.

In the U.S., patients are continuing to receive GTX-102 at the lower loading and maintenance dose. Productive discussions with the FDA are ongoing to harmonize the dosing strategy with the ex-U.S. protocol.

DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): Dosing in Phase 3 study complete
Ultragenyx has randomized and dosed the last patient in the Phase 3 study. The 48-week study has fully enrolled patients eight years of age and older, randomized 1:1 to DTX401 or placebo. The primary endpoint is the reduction in oral glucose replacement with cornstarch while maintaining glucose control. Phase 3 data are expected in the first half of 2024.

DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 study dosing patients
Ultragenyx is randomizing and dosing patients in the ongoing Phase 3 study. The pivotal, 64-week study will include approximately 50 patients, randomized 1:1 to DTX301 or placebo. The primary endpoints are response as measured by removal of ammonia-scavenger medications and protein-restricted diet and change in 24-hour ammonia levels.

UX701 AAV gene therapy for Wilson Disease: Stage 1 of pivotal clinical study dosing patients; expect interim Stage 1 enrollment completion in the second half of 2023
Dosing in the first stage of the pivotal study is ongoing under an amended protocol that removes placebo from the dose finding stage and includes five patients per cohort. During this stage of the study, safety and efficacy of up to three dose levels of UX701 will be evaluated and a dose will be selected for further evaluation in Stage 2. Completion of Stage 1 enrollment is expected in the second half of 2023 with data expected in the first half of 2024 that would include safety and potentially initial signs of clinical activity.

UX053 mRNA for glycogen storage disease type III (GSDIII): Phase 1/2 single ascending dose cohort enrolled; data in 1H23
Dosing in the single ascending dose stage (SAD) of the Phase 1/2 study of UX053 for the treatment of GSDIII has been completed with no safety issues observed. The company has decided to not enroll patients in the multiple ascending dose cohorts at this time to allow greater focus on other late-stage and larger indication clinical programs. The data from the SAD cohort are being analyzed and are expected in the second quarter of 2023.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Thursday, May 4, 2023, at 2 p.m. PT/ 5 p.m. ET to discuss the first quarter 2023 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call, please register by clicking on the following link (https://register.vevent.com/register/BI1183803cc36b46baa6367e521e12e605), and you will be provided with dial in details. The replay of the call will be available for one year.

On May 4, 2023 Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, reported financial results for the quarter ended March 31, 2023 and highlighted recent corporate progress (Press release, Tyra Biosciences, MAY 4, 2023, View Source [SID1234631058]).

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"2023 is shaping up to be a great year for TYRA. We believe that our lead precision compound, TYRA-300, is well-positioned to become a best-in-class agent in multiple therapeutic areas where FGFR3 plays a major role. Our SURF301 oncology study is progressing well, and we look forward to providing more detail on our clinical plans for TYRA-300 in achondroplasia," said Todd Harris, CEO of TYRA. "In addition, we continued to advance our broader pipeline, with TYRA-200 expected to enter the clinic in 2H 2023, and additional clinical candidates from our in-house SNÅP discovery engine poised for nomination."

First Quarter 2023 and Recent Corporate Highlights

TYRA-300

•
Advanced SURF301 Phase 1/2 Study for Oncology. TYRA continued to advance its SURF301 Phase 1/2 clinical study of TYRA-300, an investigational agent and a once-daily oral FGFR3-selective inhibitor, with an initial focus on patients with metastatic urothelial carcinoma of the bladder and urinary tract. SURF301 (Study in Untreated and Resistant FGFR3+ Advanced Solid Tumors) (NCT05544552) is a multi-center, open label study designed to determine the optimal and maximum tolerated doses and the recommended Phase 2 dose of TYRA-300, as well as to evaluate the preliminary antitumor activity of TYRA-300. During the first quarter of 2023, TYRA continued to enroll and dose patients in SURF301 at multiple clinical sites.
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Expanded Development into Achondroplasia. In March 2023, TYRA announced the expansion of development of TYRA-300 into achondroplasia based on positive preclinical results in a study performed in collaboration with the Imagine Institute in Paris, France. TYRA is on track to submit an Investigational New Drug application (IND) to the U.S. Food and Drug Administration (FDA) to enable a Phase 2 study of TYRA-300 in pediatric achondroplasia in 2024.

TYRA-200

•
IND Cleared by FDA; Phase 1 Study on Track for 2H 2023. In March 2023, TYRA announced that the FDA cleared its IND to proceed with a Phase 1 clinical study of TYRA-200, an FGFR1/2/3 inhibitor with potency against activating FGFR2 gene alterations and resistance mutations. The trial will be focused on intrahepatic cholangiocarcinoma resistant to prior FGFR inhibitors. TYRA expects the first patient will be dosed in this trial in the second half of 2023.

SNÅP Platform and Pipeline

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TYRA continued to advance its in-house precision medicine discovery engine, SNÅP, to develop therapies in targeted oncology and genetically defined conditions including FGF19+/FGR4-driven cancers, and RET (REarranged during Transfection kinase) driven cancers.

First Quarter 2023 Financial Results

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First quarter 2023 net loss was $11.9 million compared to $14.8 million for the same period in 2022.
•
First quarter 2023 research and development expenses were $10.4 million compared to $9.6 million for the same period in 2022.
•
First quarter 2023 general and administrative expenses were $3.9 million compared to $5.2 million for the same period in 2022.
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As of March 31, 2023, TYRA had cash and cash equivalents of $241.7 million that will support TYRA through important clinical and operational milestones over at least the next two years.

About TYRA-300

TYRA-300 is the Company’s lead precision medicine program stemming from its in-house SNÅP platform. TYRA-300 is an investigational, oral, FGFR3-selective inhibitor currently in development for the treatment of cancer and skeletal dysplasias including achondroplasia. TYRA-300 is being evaluated in a multi-center, open label Phase 1/2 clinical study, SURF301 (Study in Untreated and Resistant FGFR3+ Advanced Solid Tumors). SURF301 (NCT05544552) was designed to determine the optimal and maximum tolerated doses (MTD) and the recommended Phase 2 dose (RP2D) of TYRA-300, as well as to evaluate the preliminary antitumor activity of TYRA-300. SURF301 is currently enrolling adults with advanced urothelial carcinoma and other solid tumors with FGFR3 gene alterations. In skeletal dysplasias, TYRA-300 has demonstrated positive preclinical results and the Company expects to submit an IND for the initiation of a Phase 2 clinical study in pediatric achondroplasia in 2024.

On May 4, 2023 TG Therapeutics, Inc. (NASDAQ: TGTX) reported that Michael S. Weiss, the Company’s Chairman and Chief Executive Officer, will participate in the Bank of America Securities 2023 Health Care Conference, being held at the Encore Hotel, in Las Vegas, NV on Tuesday May 9th through Thursday May 11th, 2023 (Press release, TG Therapeutics, MAY 4, 2023, View Source [SID1234631057]). The fireside chat is scheduled to take place on Tuesday, May 9, 2023, at 3:00 PM PT.

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A live webcast of the fireside chat will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source

On May 4, 2023 Surface Oncology (Nasdaq: SURF), a clinical-stage immuno-oncology company developing next-generation immunotherapies that target the tumor microenvironment, reported financial results and corporate highlights for the first quarter 2023, as well as anticipated near-term milestones (Press release, Surface Oncology, MAY 4, 2023, View Source [SID1234631056]).

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"We remain highly encouraged by the progress of our two clinical programs, SRF388 a potential first-in-class IL-27 inhibitor, and SRF114, a potentially best-in-class anti-CCR8 antibody, both of which have been enrolling well," said Rob Ross, M.D., chief executive officer of Surface. "At the recent AACR (Free AACR Whitepaper) Annual Meeting, we presented compelling new SRF114 preclinical data which indicate that therapeutic depletion of CCR8 positive tumor infiltrating Tregs results in robust anti-tumorigenic activity across multiple tumor models. With respect to SRF388 and our ongoing Phase 2 trials in liver and lung cancer, we look forward to providing a clinical data update in both indications later in the second quarter."

First Quarter and Subsequent Corporate Highlights

In April 2023, Surface presented new preclinical data on SRF114, a fully human anti-CCR8 antibody, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2023 in Orlando. A poster presentation highlighted new in vivo data showing SRF114 promotes a pro-inflammatory tumor microenvironment resulting in robust antitumor activity in checkpoint inhibition-resistant and checkpoint inhibition-susceptible tumor models. The poster also demonstrated that anti-CCR8 therapy resulted in the depletion of Treg cells selectively in tumor tissue and anti-CCR8 and anti-PD-1 combination therapy increased tumor immune cell infiltration, cytokine production and improved overall survival in a checkpoint inhibitor-resistant melanoma model.

In January 2023, Surface announced the first patient had been dosed in a Phase 1/2 study evaluating SRF114 as a monotherapy in patients with advanced solid tumors. The dose escalation portion of the study is ongoing and will enroll up to 30 patients evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of SRF114.
Selected Anticipated Near-term Corporate Milestones

In the second quarter, Surface expects to share updated clinical data from the ongoing Phase 2 studies investigating SRF388 as a monotherapy in lung cancer and in combination therapy in liver cancer.
Initial safety and efficacy data from the ongoing SRF114 Phase 1/2 clinical study are anticipated in 2024.
Financial Results

As of March 31, 2023, cash, cash equivalents and marketable securities were $102.1 million, compared to $124.8 million on December 31, 2022.

General and administrative (G&A) expenses were $5.9 million for the first quarter ended March 31, 2023, compared to $6.5 million for the same period in 2022. The decrease primarily relates to personnel-related costs from reduced headcount and a reduction in professional fees. G&A expenses included $1.0 million in stock-based compensation expense for the first quarter ended March 31, 2023.

Research and development (R&D) expenses were $13.8 million for the first quarter ended March 31, 2023, compared to $16.6 million for the same period in 2022. This decrease was primarily driven by a reduction in manufacturing costs for our SRF388 program and the strategic decision to pause the SRF617 program as part of our corporate restructuring in November 2022. R&D expenses included $0.6 million in stock-based compensation expense for the first quarter ended March 31, 2023.

For the first quarter ended March 31, 2023, net loss was $19.7 million, or basic and diluted net loss per share of $0.33. Net income was $6.2 million for the same period in 2022, or basic net income per share of $0.13 and diluted net income per share of $0.13.

Surface Oncology continues to project that current cash and cash equivalents are sufficient to fund the company into the third quarter of 2024.

About SRF388

SRF388 is a fully human anti-IL-27 antibody designed to inhibit the activity of this immunosuppressive cytokine. Surface Oncology has identified particular tumor types, including liver and lung cancer, where IL-27 appears to play an important role in the immunosuppressive tumor microenvironment and may contribute to resistance to treatment with checkpoint inhibitors. SRF388 targets the rate-limiting p28 subunit of IL-27, and preclinical studies have shown that treatment with SRF388 blocks the immunosuppressive biologic effects of IL-27, resulting in immune cell activation in combination with other cancer therapies including anti-PD-1 therapy, as well as potent anti-tumor effects as a monotherapy. Furthermore, Surface Oncology has identified a potential biomarker associated with IL-27 that may be useful in helping to identify patients most likely to respond to SRF388. In November 2020, Surface announced that SRF388 was granted Orphan Drug designation and Fast Track designation for the treatment of refractory hepatocellular carcinoma from the FDA.

About SRF114

SRF114 is a fully human, afucosylated anti-CCR8 antibody designed to preferentially deplete CCR8+ Treg cells within the tumor microenvironment. In preclinical studies, Surface Oncology has shown that SRF114 induces antibody-dependent cellular cytotoxicity (ADCC) and/or antibody-dependent cellular phagocytosis (ADCP) pathways to deplete intratumoral Treg cells. In addition, SRF114 reduced tumor growth in murine models. These findings support the advancement of SRF114 as a therapeutic candidate that holds the potential to drive anti-tumor immunity in patients.

On May 4, 2023 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported positive clinical results from a compatibility study evaluating HyBryte (synthetic hypericin sodium) in the treatment of cutaneous T-cell lymphoma (CTCL) using the commercially ready Daavlin Series 7 visible light device, which recently received 510(k) clearance from U.S. Food and Drug Administration (FDA) (Press release, Soligenix, MAY 4, 2023, View Source [SID1234631055]). The open-label study (protocol HPN-CTCL-02) enrolled 9 patients to receive 8 weeks of HyBryte treatment of their cancerous lesions, with an assessment of treatment response conducted at week 10 using the Composite Assessment of Index Lesion Severity (CAILS) score. All subjects were enrolled by Brian Poligone, MD, PhD, at the Rochester Skin Lymphoma Medical Group.

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The purpose of the study was to establish that any light device capable of producing visible light of an appropriate and consistent wavelength (500 to 650 nm) was suitable for use with HyBryte and extend the pharmacokinetic profile using a recently developed, more sensitive hypericin assay. In addition to meeting these objectives, the efficacy demonstrated strongly substantiates the results of the Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) study. The treatment response data of 22% following 8 weeks of twice weekly HyBryte therapy recapitulates the results of the FLASH trial, despite the fact that patients in the current study were specifically selected to have more extensive disease consistent with its potential commercial use. Additionally, in this study all patients had improvements in their cumulative CAILS score (average improvement of 36.4%, range 8 to 100%). Results in individual lesions showed that 7 of the 27 index lesions (25.9%) had at least a 50% improvement in their CAILS score and 4 of the 27 index lesions (14.8%) were completely resolved after as little as 8 weeks of treatment. Other key evaluations included measurements of systemic exposure and cardiac output, which yielded extremely low and limited levels of systemic hypericin (plateau concentration of approximately 0.00013 μg/mL) detected in the blood and no observable impact to normal sinus rhythm, reinforcing the safety of HyBryte.

"We were excited for the opportunity to work with Soligenix and make HyBryte available to our patients," stated Brian Poligone, MD, PhD, Director of the Rochester Skin Lymphoma Medical Group, Fairport, NY, and Principal Investigator for the compatibility study and Leading Enrolling Investigator in the FLASH study. "Since the completion of the Phase 3 FLASH study, I have had a number of patients asking about possible access to this promising therapy. Fortunately, we were chosen to conduct this study and the patients’ enthusiasm for the product was evident by their willingness to participate in the trial, allowing for its rapid completion. I look forward to continuing to work with Soligenix to further advance the HyBryte program so my patients can have this much needed treatment option available to them."

"These results reinforce the positive HyBryte data from the FLASH study. Important corporate objectives for the study were to replicate results previously observed in the FLASH study, while using finished drug product manufactured by our proposed commercial contract manufacturer and activated using a commercially viable light device," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "We look forward to continuing to work with Dr. Poligone and all of our committed clinical investigators to make HyBryte available to this underserved orphan patient population."

About HyBryte

HyBryte (research name SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. The active ingredient in HyBryte is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions that is taken up by the malignant T-cells, and then activated by visible light approximately 24 hours later. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker plaques and lesions. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging drugs and other phototherapy that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p=0.04) improvement with topical hypericin treatment whereas the placebo was ineffective. HyBryte has received orphan drug and fast track designations from the FDA, as well as orphan designation from the European Medicines Agency (EMA).

The recently published Phase 3 FLASH trial enrolled a total of 169 patients (166 evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of three treatment cycles. Treatments were administered twice weekly for the first 6 weeks and treatment response was determined at the end of the 8th week of each cycle. In the first double-blind treatment cycle, 116 patients received HyBryte treatment (0.25% synthetic hypericin) and 50 received placebo treatment of their index lesions. A total of 16% of the patients receiving HyBryte achieved at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) compared to only 4% of patients in the placebo group at 8 weeks (p=0.04) during the first treatment cycle (primary endpoint). HyBryte treatment in the first cycle was safe and well tolerated.

In the second open-label treatment cycle (Cycle 2), all patients received HyBryte treatment of their index lesions. Evaluation of 155 patients in this cycle (110 receiving 12 weeks of HyBryte treatment and 45 receiving 6 weeks of placebo treatment followed by 6 weeks of HyBryte treatment), demonstrated that the response rate among the 12-week treatment group was 40% (p<0.0001 vs the placebo treatment rate in Cycle 1). Comparison of the 12-week and 6-week treatment groups also revealed a statistically significant improvement (p<0.0001) between the two groups, indicating that continued treatment results in better outcomes. HyBryte continued to be safe and well tolerated. Additional analyses also indicated that HyBryte is equally effective in treating both plaque (response 42%, p<0.0001 relative to placebo treatment in Cycle 1) and patch (response 37%, p=0.0009 relative to placebo treatment in Cycle 1) lesions of CTCL, a particularly relevant finding given the historical difficulty in treating plaque lesions in particular.

The third (optional) treatment cycle (Cycle 3) was focused on safety and all patients could elect to receive HyBryte treatment of all their lesions. Of note, 66% of patients elected to continue with this optional compassionate use / safety cycle of the study. Of the subset of patients that received HyBryte throughout all 3 cycles of treatment, 49% of them demonstrated a positive treatment response (p<0.0001 vs patients receiving placebo in Cycle 1). Moreover, in a subset of patients evaluated in this cycle, it was demonstrated that HyBryte is not systemically available, consistent with the general safety of this topical product observed to date. At the end of Cycle 3, HyBryte continued to be well tolerated despite extended and increased use of the product to treat multiple lesions.

Overall safety of HyBryte is a critical attribute of this treatment and was monitored throughout the three treatment cycles (Cycles 1, 2 and 3) and the 6-month follow-up period. HyBryte’s mechanism of action is not associated with DNA damage, making it a safer alternative than currently available therapies, all of which are associated with significant and sometimes fatal, side effects. Predominantly these include the risk of melanoma and other malignancies, as well as the risk of significant skin damage and premature skin aging. Currently available treatments are only approved in the context of previous treatment failure with other modalities and there is no approved front-line therapy available. Within this landscape, treatment of CTCL is strongly motivated by the safety risk of each product. HyBryte potentially represents the safest available efficacious treatment for CTCL. With very limited systemic absorption, a compound that is not mutagenic and a light source that is not carcinogenic, there is no evidence to date of any potential safety issues.

The Phase 3 CTCL clinical study was partially funded by the National Cancer Institute via a Phase II SBIR grant (#1R44CA210848-01A1) awarded to Soligenix, Inc. In addition, the FDA awarded an Orphan Products Development grant to support the evaluation of HyBryte for expanded treatment in patients with early-stage CTCL, including in the home use setting. The grant, totaling $2.6 million over 4 years, was awarded to a prestigious academic institution that was a leading enroller in the Phase 3 FLASH study.

About Cutaneous T-Cell Lymphoma (CTCL)

CTCL is a class of non-Hodgkin’s lymphoma (NHL), a type of cancer of the white blood cells that are an integral part of the immune system. Unlike most NHLs which generally involve B-cell lymphocytes (involved in producing antibodies), CTCL is caused by an expansion of malignant T-cell lymphocytes (involved in cell-mediated immunity) normally programmed to migrate to the skin. These malignant cells migrate to the skin where they form various lesions, typically beginning as patches and may progress to raised plaques and tumors. Mortality is related to the stage of CTCL, with median survival generally ranging from about 12 years in the early stages to only 2.5 years when the disease has advanced. There is currently no cure for CTCL. Typically, CTCL lesions are treated and regress but usually return either in the same part of the body or in new areas.

CTCL constitutes a rare group of NHLs, occurring in about 4% of the approximate 700,000 individuals living with the disease. It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of CTCL that it affects over 25,000 individuals in the U.S., with approximately 3,000 new cases seen annually.