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PEPAXTO is the first anticancer peptide‑drug conjugate for adult patients with MM who have received at least 4 prior lines and are triple‑class refractory1,2

PEPAXTO is designed to leverage aminopeptidases, which are overexpressed in MM cells and cause the release of the cytotoxic payload3-6

PEPAXTO® enters the cell due to its lipophilicity. PEPAXTO is released by aminopeptidases. The cytotoxic alkylating payload irreversibly damages tumor DNA and induces apoptosis.PEPAXTO® enters the cell due to its lipophilicity. PEPAXTO is released by aminopeptidases. The cytotoxic alkylating payload irreversibly damages tumor DNA and induces apoptosis.
PEPAXTO enters
the cell due to
its lipophilicity3
PEPAXTO is
released by
aminopeptidases3,6
The cytotoxic alkylating
payload irreversibly
damages tumor DNA
and induces apoptosis4,5
  • PEPAXTO circumvents the development of transporter-associated resistance through its passive uptake into the cell7
  • In vitro studies demonstrated that PEPAXTO leverages the overexpression of aminopeptidases in MM cells to selectively direct its antitumor effect6,8

Defining the triple-class refractory patient

Triple-class refractory means a patient is resistant to at least 1 therapy in each of the 3 standard-of-care classes.
  • This triple-class refractory patient population tends to have more aggressive disease with poorer response rates and survival outcomes9‑11
New strategies are needed for
patients
with triple-class
refractory disease12

IMPORTANT SAFETY INFORMATION

PEPAXTO is contraindicated in patients with a history of serious allergic reaction to melphalan flufenamide or melphalan.

PEPAXTO may cause thrombocytopenia, which may lead to hemorrhage. Monitor platelets at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first 2 months of treatment with PEPAXTO. Do not administer PEPAXTO if the platelet count is less than 50 x 109/L. Withhold PEPAXTO until platelet count is 50 x 109/L or greater and resume at same or reduced dose based on duration of interruption. Adjust dose and/or dose schedule based on signs and symptoms of bleeding.

PEPAXTO may cause neutropenia, which may lead to infection. Monitor neutrophil counts at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first 2 months of treatment with PEPAXTO. Do not administer PEPAXTO if absolute neutrophil count is less than 1 x 109/L. Withhold PEPAXTO until absolute neutrophil count is 1 x 109/L or greater and resume at same or reduced dose based on duration of interruption. Consider leukocyte growth factor as clinically appropriate.

PEPAXTO may cause anemia. Monitor red blood cell counts at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first 2 months of treatment with PEPAXTO. Treat anemia as clinically indicated. Dosage modification and dose delay of PEPAXTO may be required to allow for recovery of red blood cells.

Patients taking PEPAXTO experienced infections, including fatal infections. Consider antimicrobials as clinically appropriate.

Nonclinical safety studies with melphalan flufenamide at dosages exceeding the recommended dose for PEPAXTO were associated with mortality. The safety and efficacy of PEPAXTO has not been established for use as a conditioning regimen in patients receiving transplant.

Secondary malignancies such as myelodysplastic syndromes or acute leukemia have been reported in patients with multiple myeloma who were treated with PEPAXTO. Monitor patients long term for the development of secondary malignancies.

Based on its mechanism of action, PEPAXTO can cause fetal harm when administered to a pregnant woman because it is genotoxic and targets actively dividing cells. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with PEPAXTO and for 6 months after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with PEPAXTO and for 3 months after the last dose.

The most common adverse reactions (20%; Grades 1‑4) were fatigue (55%), nausea (32%), diarrhea (27%), pyrexia (24%), and respiratory tract infection (24%).

Special Considerations
Use in Pregnancy

Based on the mechanism of action, PEPAXTO can cause fetal harm when administered to a pregnant woman. There are no available data on PEPAXTO use in pregnant women to evaluate for a drug-associated risk. PEPAXTO is a genotoxic drug. Advise a woman of childbearing potential of the potential risks to the fetus.

Lactation

There is no information regarding the presence of melphalan flufenamide or its metabolites in human breast milk, or the effects on the breastfed child or on milk production. Because of the potential for serious adverse reactions in a breastfed child, advise women not to breastfeed during treatment with PEPAXTO and for 1 week after the last dose.

Females and Males of Reproductive Potential

PEPAXTO can cause fetal harm when administered to a pregnant woman. Advise females of reproductive potential to use effective contraception during treatment with PEPAXTO and for 6 months after the last dose.

PEPAXTO may damage the developing male germ cells, resulting in possible genetic fetal abnormalities. Advise males with a female partner of reproductive potential to use effective contraception during treatment with PEPAXTO and for 3 months after the last dose.

PEPAXTO can cause amenorrhea in premenopausal women and result in infertility. Based on findings of melphalan flufenamide in animals, PEPAXTO may impair male fertility. Alkylating drugs, such as PEPAXTO, can also cause irreversible testicular suppression in patients.

INDICATION

PEPAXTO is indicated in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38‑directed monoclonal antibody. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Limitation of Use

PEPAXTO is not indicated and is not recommended for use as a conditioning regimen for transplant outside of controlled clinical trials.

Please click here for full Prescribing Information.

IMPORTANT SAFETY INFORMATION

PEPAXTO is contraindicated in patients with a history of serious allergic reaction to melphalan flufenamide or melphalan.

PEPAXTO may cause thrombocytopenia, which may lead to hemorrhage. Monitor platelets at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first 2 months of treatment with PEPAXTO. Do not administer PEPAXTO if the platelet count is less than 50 x 109/L. Withhold PEPAXTO until platelet count is 50 x 109/L or greater and resume at same or reduced dose based on duration of interruption. Adjust dose and/or dose schedule based on signs and symptoms of bleeding.

PEPAXTO may cause neutropenia, which may lead to infection. Monitor neutrophil counts at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first 2 months of treatment with PEPAXTO. Do not administer PEPAXTO if absolute neutrophil count is less than 1 x 109/L. Withhold PEPAXTO until absolute neutrophil count is 1 x 109/L or greater and resume at same or reduced dose based on duration of interruption. Consider leukocyte growth factor as clinically appropriate.

PEPAXTO may cause anemia. Monitor red blood cell counts at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first 2 months of treatment with PEPAXTO. Treat anemia as clinically indicated. Dosage modification and dose delay of PEPAXTO may be required to allow for recovery of red blood cells.

Patients taking PEPAXTO experienced infections, including fatal infections. Consider antimicrobials as clinically appropriate.

Nonclinical safety studies with melphalan flufenamide at dosages exceeding the recommended dose for PEPAXTO were associated with mortality. The safety and efficacy of PEPAXTO has not been established for use as a conditioning regimen in patients receiving transplant.

Secondary malignancies such as myelodysplastic syndromes or acute leukemia have been reported in patients with multiple myeloma who were treated with PEPAXTO. Monitor patients long term for the development of secondary malignancies.

Based on its mechanism of action, PEPAXTO can cause fetal harm when administered to a pregnant woman because it is genotoxic and targets actively dividing cells. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with PEPAXTO and for 6 months after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with PEPAXTO and for 3 months after the last dose.

The most common adverse reactions (20%; Grades 1‑4) were fatigue (55%), nausea (32%), diarrhea (27%), pyrexia (24%), and respiratory tract infection (24%).

Special Considerations
Use in Pregnancy

Based on the mechanism of action, PEPAXTO can cause fetal harm when administered to a pregnant woman. There are no available data on PEPAXTO use in pregnant women to evaluate for a drug-associated risk. PEPAXTO is a genotoxic drug. Advise a woman of childbearing potential of the potential risks to the fetus.

Lactation

There is no information regarding the presence of melphalan flufenamide or its metabolites in human breast milk, or the effects on the breastfed child or on milk production. Because of the potential for serious adverse reactions in a breastfed child, advise women not to breastfeed during treatment with PEPAXTO and for 1 week after the last dose.

Females and Males of Reproductive Potential

PEPAXTO can cause fetal harm when administered to a pregnant woman. Advise females of reproductive potential to use effective contraception during treatment with PEPAXTO and for 6 months after the last dose.

PEPAXTO may damage the developing male germ cells, resulting in possible genetic fetal abnormalities. Advise males with a female partner of reproductive potential to use effective contraception during treatment with PEPAXTO and for 3 months after the last dose.

PEPAXTO can cause amenorrhea in premenopausal women and result in infertility. Based on findings of melphalan flufenamide in animals, PEPAXTO may impair male fertility. Alkylating drugs, such as PEPAXTO, can also cause irreversible testicular suppression in patients.

INDICATION

PEPAXTO is indicated in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38‑directed monoclonal antibody. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Limitation of Use

PEPAXTO is not indicated and is not recommended for use as a conditioning regimen for transplant outside of controlled clinical trials.

Please click here for full Prescribing Information.

  1. REFERENCES:
  2. 1. PEPAXTO. Prescribing information. Oncopeptides, Inc.; 2021.
  3. 2. Oriol A, Abril L, Ibarra G, et al. Limited treatment options in refractory multiple myeloma: promising therapeutic developments. Expert Rev Anticancer Ther. 2020;20(1):31-44. doi: 10.1080/14737140.2020.1708721
  4. 3. Wickström M, Nygren P, Larsson R, et al. Melflufen - a peptidase-potentiated alkylating agent in clinical trials. Oncotarget. 2017;8(39):66641-66655. doi: 10.18632/oncotarget.18420
  5. 4. Chauhan D, Ray A, Viktorsson K, et al. In vitro and in vivo antitumor activity of a novel alkylating agent, melphalan-flufenamide, against multiple myeloma cells. Clin Cancer Res. 2013;19(11):3019-3031. doi: 10.1158/1078-0432.CCR-12-3752
  6. 5. Ray A, Ravillah D, Das DS, et al. A novel alkylating agent Melflufen induces irreversible DNA damage and cytotoxicity in multiple myeloma cells. Br J Haematol. 2016;174(3):397-409. doi: 10.1111/bjh.14065
  7. 6. Wickström M, Viktorsson K, Lundholm L, et al. The alkylating prodrug J1 can be activated by aminopeptidase N, leading to a possible target directed release of melphalan. Biochem Pharmacol. 2010;79(9):1281-1290. doi: 10.1016/j.bcp.2009.12.022
  8. 7. Richardson PG, Oriol A, Larocca A, et al. Melflufen and dexamethasone in heavily pretreated relapsed and refractory multiple myeloma. J Clin Oncol. 2020;JCO2002259. doi: 10.1200/JCO.20.02259
  9. 8. Miettinen JJ, Kumari R, Traustadottir GA, et al. Aminopeptidase expression in multiple myeloma associates with disease progression and sensitivity to melflufen. Poster presented at: 25th European Hematology Association Annual Conference; June 11-21, 2020; virtual.
  10. 9. Mikhael J. Treatment options for triple-class refractory multiple myeloma. Clin Lymphoma Myeloma Leuk. 2020;20(1):1-7. doi: 10.1016/j.clml.2019.09.621
  11. 10. Sonneveld P. Management of multiple myeloma in the relapsed/refractory patient. Hematology Am Soc Hematol Educ Program. 2017;2017(1):508-517. doi: 10.1182/asheducation-2017.1.508
  12. 11. Nijhof IS, van de Donk NWCJ, Zweegman S, Lokhorst HM. Current and new therapeutic strategies for relapsed and refractory multiple myeloma: an update. Drugs. 2018;78(1):19-37. doi: 10.1007/s40265-017-0841-y
  13. 12. Yong K, Delforge M, Driessen C, et al. Multiple myeloma: patient outcomes in real-world practice. Br J Haematol. 2016;175(2):252-264. doi: 10.1111/bjh.14213