Karyopharm and Menarini Group Announce Orphan Medicinal Product Designation from the European Commission for Selinexor for the Treatment of Myelofibrosis

NEWTON, Mass. and FLORENCE, Italy, Nov. 1, 2022 /PRNewswire/ — Karyopharm Therapeutics Inc. (NASDAQ: KPTI), a commercial-stage pharmaceutical firm pioneering novel most cancers therapies, and the Menarini Group (“Menarini”), a privately-held, main worldwide pharmaceutical firm, right now introduced that the European Commission (EC) has granted orphan medicinal product designation for selinexor for the therapy of myelofibrosis (MF). Selinexor was granted orphan drug designation in MF by the U. S. Food and Drug Administration (FDA) in May 2022. Karyopharm is at present evaluating selinexor, a first-in-class XP01 inhibitor, as monotherapy in sufferers with beforehand handled MF, and together with ruxolitinib in treatment-naïve sufferers. In December 2021, Karyopharm and Menarini entered into an unique licensing settlement whereby Menarini is accountable for commercializing all present and future indications of NEXPOVIO® in the European Economic Area, United Kingdom and Switzerland, CIS international locations, Turkey and Latin America. Stemline Therapeutics B.V., a completely owned subsidiary of Menarini, is main all commercialization actions in Europe.

“We are very pleased to receive orphan medicinal product designation from the EC for selinexor for the treatment of myelofibrosis,” stated Reshma Rangwala, MD, PhD, Chief Medical Officer of Karyopharm. “Building on our recent orphan drug designation from the FDA, this recognition continues to reinforce the significant unmet need for a drug with a novel mechanism of action like selinexor for this devastating disease. Our clinical plans remain on track, and we look forward to the continued development of selinexor in MF.”

“Myelofibrosis is a difficult-to-treat and complex disorder of the bone marrow with limited therapeutic options and we are committed to bringing novel treatments to patients through our collaboration with Karyopharm. We are excited about the potential to bring selinexor to myelofibrosis patients in Europe, pending positive study read-outs and regulatory approval,” stated Olivia del Puerto, MD LMS, Head of Medical Affairs Oncology – EMEA of Menarini.

About the EMA Orphan Designation

Orphan medicinal product designation in the European Union (EU) is granted by the European Commission which adopts the optimistic opinion issued by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products. The EMA’s orphan designation is on the market to corporations growing remedies for life-threatening or chronically debilitating circumstances that have an effect on fewer than 5 in 10,000 individuals in the EU. Medicines that meet the EMA’s orphan designation standards qualify for monetary and regulatory incentives that embrace a 10-year interval of advertising exclusivity in the EU after product approval, lowered charges and entry to centralized advertising authorization.

About MF

MF is a uncommon kind of bone marrow most cancers that disrupts the physique’s regular manufacturing of blood cells. It causes intensive scarring of the bone marrow, resulting in extreme anemia that may trigger weak point and fatigue. Bone marrow scarring may also trigger a low quantity of platelets, which will increase the threat of bleeding. MF impacts males and females in equal numbers and can happen at any age, though it often impacts people 50 years outdated or older. According to the National Organization of Rare Diseases (NORD), the incidence is estimated to be 1.5 circumstances per 100,000 individuals in the United States and in Northern European international locations, primarily based on research, the incidence is estimated to be 0.5 circumstances per 100,000 individuals.¹

About NEXPOVIO® (selinexor)

NEXPOVIO®, which is marketed as XPOVIO® in the U.S., has been accredited in the following oncology indications by the European Commission: (i) together with dexamethasone for the therapy of a number of myeloma in grownup sufferers who’ve obtained no less than 4 prior therapies and whose illness is refractory to no less than two proteasome inhibitors, two immunomodulatory brokers and an anti-CD38 monoclonal antibody, and who’ve demonstrated illness development on the final remedy; and (ii) together with bortezomib and dexamethasone for the therapy of adults with a number of myeloma who’ve obtained no less than one prior remedy. The advertising authorization of NEXPOVIO is legitimate in the EU Member States in addition to Iceland, Liechtenstein, Norway, and Northern Ireland. NEXPOVIO has been commercially out there in Germany since October 1, 2022.

NEXPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor. NEXPOVIO capabilities by selectively binding to and inhibiting the nuclear export protein exportin 1 (XPO1, additionally referred to as CRM1). NEXPOVIO blocks the nuclear export of tumor suppressor, development regulatory and anti-inflammatory proteins, resulting in accumulation of these proteins in the nucleus and enhancing their anti-cancer exercise in the cell. The pressured nuclear retention of these proteins can counteract a mess of the oncogenic pathways that, unchecked, enable most cancers cells with extreme DNA harm to proceed to develop and divide in an unrestrained style.

Please see NEXPOVIO® Summary of Product Characteristics and European Public Assessment Report at https://ec.europa.eu/health/documents/community-register/html/h1537.htm

Please confer with native prescribing data the place XPOVIO/NEXPOVIO is accredited for full data.

IMPORTANT SAFETY INFORMATION

Contraindications: Hypersensitivity to selinexor.

Special warnings and precautions for use:

Recommended concomitant remedies

Patients must be suggested to take care of ample fluid and caloric consumption all through therapy. Intravenous hydration must be thought-about for sufferers in danger of dehydration.

Prophylactic concomitant therapy with a 5-HT3 antagonist and/or different anti-nausea brokers must be offered previous to and throughout therapy with NEXPOVIO®.

Haematology:

Patients ought to have their full blood counts (CBC) assessed at baseline, throughout therapy, and as clinically indicated. Monitor extra regularly throughout the first two months of therapy.

Thrombocytopenia:

Thrombocytopenic occasions (thrombocytopenia and platelet rely decreased) have been regularly reported in grownup sufferers receiving selinexor, which will be extreme (Grade 3/4). Patients must be monitored for indicators and signs of bleeding and evaluated promptly.

Neutropenia:

Severe neutropenia (Grade 3/4) has been reported with selinexor.

Patients with neutropenia must be monitored for indicators of an infection and evaluated promptly.

Gastrointestinal toxicity:

Nausea, vomiting, diarrhoea, which generally will be extreme and might require the use of anti-emetic and anti-diarrhoeal medicinal merchandise.

Weight loss and anorexia:

Patients ought to have their physique weight, dietary standing and quantity checked at baseline, throughout therapy, and as clinically indicated. Monitoring must be extra frequent throughout the first two months of therapy.

Confusional state and dizziness:

Patients must be instructed to keep away from conditions the place dizziness or confusional state could also be an issue and to not take different medicinal merchandise which will trigger dizziness or confusional state with out ample medical recommendation. Patients must be suggested to not drive or function heavy equipment till signs resolve.

Hyponatraemia:

Patients ought to have their sodium ranges checked at baseline, throughout therapy, and as clinically indicated. Monitoring must be extra frequent throughout the first two months of therapy.

Cataract:

Selinexor could cause new onset or exacerbation of cataract. Ophthalmologic analysis could also be carried out as clinically indicated.  Cataract must be handled as per medical pointers, together with surgical procedure if warranted.

Tumour lysis syndrome (TLS):

TLS has been reported in sufferers receiving remedy with selinexor. Patients at a excessive threat for TLS must be monitored intently. Treat TLS promptly in accordance with institutional pointers.

Fertility, being pregnant and lactation

Women of childbearing potential/contraception in males and females:

Women of childbearing potential and male grownup sufferers of reproductive potential must be suggested to make use of efficient contraceptive measures or abstain from sexual activity whereas being handled with selinexor and for no less than 1 week following the final dose of selinexor.

Pregnancy:

There aren’t any knowledge from the use of selinexor in pregnant girls.  Selinexor just isn’t advisable throughout being pregnant and in girls of childbearing potential not utilizing contraception.

Breast-feeding:

It is unknown whether or not selinexor or its metabolites are excreted in human milk. A threat to breast-fed youngsters can’t be excluded. Breast-feeding must be discontinued throughout therapy with selinexor and for 1 week after the final dose.

Undesirable results

Summary of the security profile

The most frequent antagonistic reactions (≥30%) of selinexor together with dexamethasone have been nausea, thrombocytopenia, fatigue, anaemia, decreased urge for food, decreased weight, diarrhea, vomiting, hyponatraemia, neutropenia and leukopenia.

The mostly reported severe antagonistic reactions (≥3%) have been pneumonia, sepsis, thrombocytopenia, acute kidney damage, and anaemia.

Description of chosen antagonistic reactions

Infections: Infection was the commonest non-haematological toxicity. Upper respiratory tract an infection and pneumonia have been the mostly reported infections with 25% of reported infections being severe and deadly infections occurring in 3% of handled grownup sufferers.

Elderly inhabitants

Patients 75 years and older had a better incidence of discontinuation on account of an antagonistic response, greater incidence of severe antagonistic reactions, and greater incidence of deadly antagonistic reactions.

Reporting of suspected antagonistic reactions

Reporting of suspected antagonistic reactions after Authorisation of the medicinal product is necessary. It permits continued monitoring of the profit/threat stability of the medicinal product. Healthcare professionals are requested to report any suspected antagonistic reactions by way of the nationwide reporting system listed in Appendix V.

About Karyopharm Therapeutics

Karyopharm Therapeutics Inc. (NASDAQ: KPTI) is a commercial-stage pharmaceutical firm pioneering novel most cancers therapies. Since its founding, Karyopharm has been the business chief in oral Selective Inhibitor of Nuclear Export (SINE) compound expertise, which was developed to deal with a elementary mechanism of oncogenesis: nuclear export dysregulation. Karyopharm’s lead SINE compound and first-in-class, oral exportin 1 (XPO1) inhibitor, XPOVIO® (selinexor), is accredited in the U.S. and marketed by the Company in three oncology indications and has obtained regulatory approvals in varied indications in a rising quantity of ex-U.S. territories and international locations, together with Europe and the United Kingdom (as NEXPOVIO®), China, Singapore, Canada, Israel, South Korea, and Australia. Karyopharm has a targeted pipeline focusing on a number of excessive unmet want most cancers indications, together with in a number of myeloma, endometrial most cancers, myelodysplastic syndromes and myelofibrosis. For extra details about our individuals, science and pipeline, please go to www.karyopharm.com, and observe us on Twitter at @Karyopharm and LinkedIn.

About Menarini Group

The Menarini Group is a number one worldwide pharmaceutical and diagnostics firm, with a turnover of over $4 billion and over 17,000 workers. Menarini is concentrated on therapeutic areas with excessive unmet wants with merchandise for oncology, cardiology, pneumology, gastroenterology, infectious illnesses, diabetology, irritation, and analgesia. With 18 manufacturing websites and 9 Research and Development facilities, Menarini’s merchandise can be found in 140 international locations worldwide. For additional data, please go to  www.menarini.com and LinkedIn.

Forward-Looking Statements

This press launch accommodates forward-looking statements inside the which means of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements embrace these relating to the means of selinexor to deal with sufferers with a number of myeloma and expectations associated to future medical growth and potential regulatory submissions of selinexor. Such statements are topic to quite a few necessary components, dangers and uncertainties, many of that are past Karyopharm’s management, which will trigger precise occasions or outcomes to vary materially from Karyopharm’s present expectations. For instance, there will be no assure that Karyopharm will efficiently commercialize XPOVIO®; or that any of Karyopharm’s drug candidates, together with selinexor and eltanexor, will efficiently full needed medical growth phases or that growth of any of Karyopharm’s drug candidates will proceed. Further, there will be no assure that any optimistic developments in the growth or commercialization of Karyopharm’s drug candidate portfolio will lead to inventory worth appreciation. Management’s expectations and, subsequently, any forward-looking statements on this press launch may be affected by dangers and uncertainties referring to a quantity of different components, together with the following: the threat that the COVID-19 pandemic may disrupt Karyopharm’s business extra severely than it at present anticipates, together with by negatively impacting gross sales of XPOVIO, interrupting or delaying analysis and growth efforts, impacting the means to acquire adequate provide for the growth and commercialization of selinexor or different product candidates, delaying ongoing or deliberate medical trials, impeding the execution of business plans, deliberate regulatory milestones and timelines, or inconveniencing sufferers; the adoption of XPOVIO in the business market, the timing and prices concerned in commercializing XPOVIO or any of Karyopharm’s drug candidates that obtain regulatory approval; the means to acquire and retain regulatory approval of XPOVIO or any of Karyopharm’s drug candidates that obtain regulatory approval; Karyopharm’s outcomes of medical trials and preclinical research, together with subsequent evaluation of present knowledge and new knowledge obtained from ongoing and future research; the content material and timing of selections made by the U.S. Food and Drug Administration and different regulatory authorities, investigational evaluation boards at medical trial websites and publication evaluation our bodies, together with with respect to the want for further medical research; the means of Karyopharm or its third get together collaborators or successors in curiosity to totally carry out their respective obligations below the relevant settlement and the potential future monetary implications of such settlement; Karyopharm’s means to enroll sufferers in its medical trials; unplanned money necessities and expenditures; growth or regulatory approval of drug candidates by Karyopharm’s opponents for merchandise or product candidates during which Karyopharm is at present commercializing or growing; and Karyopharm’s means to acquire, preserve and implement patent and different mental property safety for any of its merchandise or product candidates. These and different dangers are described below the caption “Risk Factors” in Karyopharm’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2022, which was filed with the Securities and Exchange Commission (SEC) on August 4, 2022, and in different filings that Karyopharm might make with the SEC in the future. Any forward-looking statements contained on this press launch converse solely as of the date hereof, and, besides as required by legislation, Karyopharm expressly disclaims any obligation to replace any forward-looking statements, whether or not consequently of new data, future occasions or in any other case.

XPOVIO® and NEXPOVIO® are registered emblems of Karyopharm Therapeutics Inc. Any different emblems referred to on this launch are the property of their respective homeowners.

¹ NORD, Rare Disease Database, Primary Myelofibrosis, Accessed on 10/4/22, https://rarediseases.org/rare-diseases/primary-myelofibrosis/

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