Ann: PTX-100 Trial Update, page-26

Some information about CTCL, its relationship to PTCL and current treatments.

T-cell lymphomas comprise approximately 10-15% of all Non-Hodgkin’s Lymphomas. The main subsets are peripheral T-cell lymphoma (PTCL) and cutaneous T-cell lymphoma (CTCL)….

CTCL originates in the skin, including the main subtypes mycosisfungoides and Sézary syndrome… .

Agents have been approved by the FDA for use in the relapsed and refractory PTCL setting, belinostat, romidepsin, pralatrexate, have overall relapse rate (ORR) ranges from 23-33%.

Therapeutic options for CTCL are mainly palliative focusing on relief of symptoms. Whereas early stage patients are well served, in the case of persistent or recurrent disease, therapeutic options remain unacceptable with FDA approved HDAC inhibitors, vorinostat and romidepsin. On Aug. 8, 2018, the Food and Drug Administration approved mogamulizumab-kpkc (Poteligeo, Kyowa Kirin, Inc.) for adult patients with relapsed or refractory mycosis fungoides (MF) or Sézary syndrome (SS) after at least one prior systemic therapy. *

Of the two FDA-approved HDAC inhibitors, the first, romidepsin, was withdrawn from the market last year by BMS after Phase 3 results failed to justify its accelerated approval.

The second, vorinostat, comes with a list of “more common” side effects requiring consultation with a doctor that almost defies belief

Anxiety; black, tarry stools; bleeding gums; bloating or swelling of the face, arms, hands, lower legs, or feet; blood in the urine or stools; body aches or pain; chest pain; chills; confusion; cough; decreased urine; diarrhea; dizziness or lightheadedness; dry mouth; ear congestion; fainting; fast or irregular heartbeat; fever; headache; increased thirst; large, hive-like swelling on the face, eyelids, lips, tongue, throat, hands, legs, feet, or genitals; loss of appetite; loss of voice; lower back or side pain; mood changes; muscle pain or cramps; nasal congestion; nausea; no blood pressure; no breathing; no pulse; numbness or tingling in the face, lips, arms, hands, feet, or legs; pain, redness, or swelling in the arm or leg; painful or difficult urination; pale skin; pinpoint red spots on the skin; rapid weight gain; runny nose; seizures; sore throat; sores, ulcers, or white spots on the lips or in the mouth; swollen glands; trouble speaking, thinking, or walking; troubled breathing; troubled breathing with exertion; unusual bleeding or bruising; unusual drowsiness, dullness, tiredness, weakness, or feeling of sluggishness; vomiting.

As for mogamulizumab (Poteligeo), the package insert carries a warning that the drug can cause serious or life-threatening side effects.

With respect to efficacy, independent review committee assessment of data from mogamulizumab’s registrational trial in patients who had received at least one prior systemic therapy, found that the estimated median PFS was 6.7 months (95% CI, 5.6 to 9.4) in the POTELIGEO arm and 3.8 months (95% CI, 3.0 to 4.7) in the vorinostat arm (hazard ratio 0.64; 95% CI: 0.49, 0.84).

* https://www.lls.org/article/t-cell-lymphomas-tcl

https://news.bms.com/news/details/2...-T-cell-Lymphoma-U.S.-Indication/default.aspx

https://www.drugs.com/sfx/vorinostat-side-effects.html