Total net revenue was $17.4 million in the second quarter of 2023, a 40% increase from $12.4 million in the first quarter of 2023
Positive topline results from two Phase 3 clinical trials evaluating DFD-29 demonstrated achievement of co-primary and all secondary endpoints versus placebo and Oracea® (doxycycline) with no significant safety issues
Fortress is advancing several late-stage clinical assets with two NDA submissions anticipated in the second half of 2023 for DFD-29 and CUTX-101
Cosibelimab longer-term results demonstrated substantial increases in complete response rates in advanced cutaneous squamous cell carcinoma
PDUFA goal date of January 3, 2024, set by FDA for cosibelimab to treat metastatic or locally advanced cutaneous squamous cell carcinoma
MIAMI, Aug. 14, 2023 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”), an innovative biopharmaceutical company focused on efficiently acquiring, developing and commercializing or monetizing promising therapeutic products and product candidates, today announced financial results and recent corporate highlights for the second quarter ended June 30, 2023.
Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, “Fortress, along with our partner companies and subsidiaries, demonstrated meaningful progress in the second quarter of 2023 and subsequent months, including:
- Our revenue numbers remain strong for the second quarter, totaling $17.4 million, which represents a 40% growth rate over the first quarter of 2023.
- Additionally, we are excited by all of the positive data milestones recently achieved, notably:
- Positive topline results from our two Phase 3 DFD-29 clinical trials for papulopustular rosacea;
- Positive data from our Phase 1/2 single center clinical trial of our CAR T cell therapy, MB-106, to treat a wide range of hematologic malignancies;
- Positive data from the Phase 1 dotinurad clinical trial in healthy volunteers in the U.S.;
- Excellent longer-term positive Phase 3 cosibelimab data demonstrating substantial increases in complete response rates in advanced cutaneous squamous cell carcinoma (“cSCC”);
- New pharmacokinetic (“PK”) modeling data for cosibelimab supporting the extension to an every-three-week dosing regimen; and
- Positive preclinical data in the BAER-101 trial supporting a Phase 2 study in epilepsy.
- On the regulatory front:
- We reached agreement with the U.S. Food and Drug Administration (“FDA”) on key elements of the Phase 3 safety study, including the primary endpoint and statistical analysis approach, for intravenous (“IV”) tramadol, which is in development for the treatment of acute post-operative pain in a medically supervised setting. We believe that a positive study outcome could result in the FDA approval of IV tramadol.
- By the end of 2023, we expect to have two additional New Drug Applications (“NDA”) on file with the FDA for CUTX-101 and DFD-29.
- There is a Prescription Drug User Fee Act (“PDUFA”) goal date of January 3, 2024, for cosibelimab to treat metastatic or locally advanced cSCC.”
Dr. Rosenwald continued, “With an expanding portfolio of marketed dermatology products, more than 25 drug candidates across our partner companies, and the potential for multiple FDA approvals over the next two years, we believe that our business is well-positioned for continued growth. Our strategy is focused on targeting exciting clinical-stage medicines with proof-of-concept data in areas of unmet need. Fortress and our partner companies are poised to achieve our collective objective of providing new treatment options to patients in need, while creating significant long-term value for our shareholders through our equity interests and royalties.”
Recent Corporate Highlights1:
Marketed Dermatology Products and Product Candidates
- Journey Medical Corporation (Nasdaq: DERM) (“Journey Medical”), our partner company, primarily focuses on selling and marketing of prescription dermatology products.
- Journey Medical’s total product net revenues were $17.0 million for the second quarter of 2023, compared to second quarter 2022 total product net revenues of $18.2 million, and showed sequential growth compared to $12.2 million total product net revenues in the first quarter of 2023.
- In July 2023, Journey Medical announced positive topline data from the two DFD-29 (Minocycline Hydrochloride Modified Release Capsules, 40 mg) Phase 3 clinical trials for the treatment of rosacea and achieved the co-primary and all secondary endpoints with subjects completing the 16-week treatment with no significant safety issues. DFD-29 demonstrated statistical superiority compared to both Oracea capsules and placebo for Investigator’s Global Assessment treatment success and the reduction in the total inflammatory lesion count in both studies. Journey Medical plans to file its NDA for DFD-29 in the second half of 2023 and expects potential approval from the FDA in the second half of 2024.
- In June 2023, Journey Medical announced positive topline data from the Phase 1 clinical trial assessing the impact of DFD-29 on the microbial flora of healthy adults and also evaluated the safety and tolerability of DFD-29. The study achieved all primary objectives and no significant safety issues were noted during the study. The results indicate that DFD-29 can be safely used for up to 16 weeks with no significant risk of microbiota suppression or development of resistance.
Cosibelimab (Anti PD-L1 antibody)
- Our partner company, Checkpoint Therapeutics, Inc. (Nasdaq: CKPT) (“Checkpoint”), submitted a Biologics License Application (“BLA”) to the FDA for cosibelimab, its investigational anti-PD-L1 antibody, as a treatment for patients with metastatic or locally advanced cSCC who are not candidates for curative surgery or radiation, in January 2023. In March 2023, the FDA accepted the BLA filing for cosibelimab and set a PDUFA goal date of January 3, 2024. In its BLA filing acceptance letter, the FDA indicated that no potential filing review issues have been identified, and that an advisory committee meeting to discuss the application is not currently planned. According to U.S. prescription claims data, in 2021, approximately 11,000 patients with cSCC were treated with systemic therapies. As checkpoint inhibitors comprised less than half of U.S. patient prescriptions, cSCC remains a disease with a need for more effective and tolerable treatment options, particularly for the significant number of cSCC patients with immunosuppressive conditions or autoimmune diseases. With its unique mechanism of action and compelling safety profile, we believe cosibelimab, if approved, would be uniquely positioned to provide an important new treatment option for patients with cSCC who are currently underserved by available therapies.
- In July 2023, Checkpoint announced new, longer-term data for cosibelimab from its pivotal studies in locally advanced and metastatic cutaneous squamous cell carcinoma (“cSCC”). These results demonstrate a deepening of response over time, resulting in substantially higher complete response rates than previously reported (55% objective response rate; 23% complete response rate in locally advanced cSCC and 50% objective response rate; 13% complete response rate in metastatic cSCC). Furthermore, responses continue to remain durable over time with the median duration of response not yet reached in either group.
- In June 2023, Checkpoint announced that new PK modeling data on cosibelimab supporting the extension to an every-three-week dosing regimen were presented at the Population Approach Group Europe 2023 annual meeting. Results support comparability of cosibelimab 800 mg every-two-week and 1200 mg every-three-week dosing regimens.
- Cosibelimab was sourced by Fortress and is currently in development at Checkpoint.
Dotinurad (Urate Transporter (URAT1) Inhibitor)
- Dotinurad is in development for the treatment of gout. Data announced in June 2023 from the Phase 1 clinical trial in healthy volunteers showed comparable pharmacokinetic, pharmacodynamic and safety profile between U.S. and Japanese healthy subjects. We plan to initiate a Phase 1b clinical trial in gout patients in the U.S. in the third quarter of 2023 to confirm the comparability of Japanese and U.S. subjects’ response to dotinurad and we expect to begin pivotal clinical trials in early 2024.
- Dotinurad (URECE® tablet) was approved in Japan in 2020 as a once-daily oral therapy for gout and hyperuricemia. Dotinurad was efficacious and well-tolerated in more than 500 Japanese patients treated for up to 58 weeks in Phase 3 clinical trials. The clinical program supporting approval included over 1,000 patients.
- Dotinurad was sourced by Fortress and is currently in development at Urica.
MB-106 (CD20-targeted CAR T Cell Therapy)
- Mustang Bio, Inc.’s (Nasdaq: MBIO) (“Mustang Bio”) lead clinical candidate is MB-106, a CD20-targeted, autologous CAR T cell therapy to treat a wide range of hematologic malignancies, including Waldenstrom macroglobulinemia (“WM”) and follicular lymphoma (“FL”). MB-106 continues to demonstrate a favorable safety and efficacy profile in both the Fred Hutch single institution and Mustang Bio multicenter Phase 1/2 clinical trials.
- Phase 1/2 data from the WM cohort in the Fred Hutch clinical trial for MB-106 were presented in a poster session at the European Hematology Association Hybrid Congress. All six patients in the WM cohort in the study were previously treated with Bruton's tyrosine kinase inhibitors (“BTKi”), and their disease continued to progress while on BTKis. 83% (5/6) of the WM cohort patients treated with MB-106 responded to treatment, including 2 complete responses (“CR”), 1 very good partial response (“VGPR”), 1 partial response (“PR”), and 1 minor response, with the remaining patient experiencing stable disease. One of the patients who achieved a CR has remained in remission for 22 months. From a safety perspective, cytokine release syndrome (“CRS”) occurred in five patients with no grade 3 or 4 CRS observed, and one patient experienced grade 1 immune effector cell-associated neurotoxicity syndrome (“ICANS”) with no grade 2, 3 or 4 ICANS observed.
- Fred Hutch also presented MB-106 data from the FL cohort of their clinical trial in an oral presentation at the International Conference on Malignant Lymphoma. A total of 20 patients with relapsed FL with confirmed CD20 expression participated in the study and had day 28 assessment. Median age was 63 years (range: 44 – 81), and median prior lines of treatment was 4 (range: 1 – 12). High-risk features included patients with progression of disease within 24 months of first-line chemoimmunotherapy (POD24) (n=15, 75%), history of histologic transformation (n=4, 20%), prior treatment with a CD19 target CAR T (n=1, 5%). Overall response rate (“ORR”) was 95% (19/20), and CR rate was 80% (16/20). Patients who received higher dose levels had an ORR of 100% and a CR rate of 91%. Ten patients are in remission over one year, seven of whom are in remission over two years. One patient, previously treated with a CD19-targeted CAR T-cell therapy, achieved a CR and remains in remission after 18 months. From a safety profile perspective, all CRS events were grade 1 (n=5, 25%) or grade 2 (n=1, 5%), with no grade ≥ 3 CRS events and there was no occurrence of ICANS of any grade.
- In parallel, Mustang Bio’s multicenter, open-label, non-randomized Phase 1/2 clinical trial evaluating the safety and efficacy of MB-106 continues to accrue, and Mustang Bio anticipates escalation to the final dose level in the Phase 1 indolent lymphoma arm in the third quarter of this year. The FDA granted Orphan Drug Designation to MB-106 for the treatment of WM, and results from this arm are expected to support an accelerated Phase 2 registration strategy for WM, with the first pivotal Phase 2 patient with WM to be treated potentially in the first quarter of 2024. Mustang Bio plans to report initial safety and efficacy data from the multicenter trial shortly, with additional safety and efficacy data from the trial expected in the fourth quarter. Finally, Mustang Bio expects to initiate a pivotal phase 2 trial in at least one additional B-cell malignancy later in 2024.
- MB-106 was sourced by Fortress and is currently in development at Mustang Bio.
CUTX-101 (Copper Histidinate for Menkes disease)
- Our subsidiary, Cyprium Therapeutics, Inc. (“Cyprium”), has completed two pivotal studies in patients with Menkes disease treated with CUTX-101, copper histidinate (CuHis). In a pre-specified analysis of the studies, a 79% reduction in the risk of death was observed in patients treated within four weeks of birth, compared with a historical control cohort of untreated patients, and median overall survival (OS) was 177.1 months for CUTX-101 compared to 16.1 months for the historical control, with a hazard ratio (“HR”) of (95% CI) = 0.208 (0.094, 0.463) p