Laminar Pharma announces IND approval by the FDA to proceed with a PhI paediatric trial assessing 2OHOA in children with advanced brain and other solid tumours

On May 29, 2018 Laminar Pharma, a pioneering clinical stage biopharmaceutical company developing a new generation of products modulating metabolism of membrane lipids based on the groundbreaking MLT platform, reported that the FDA has approved an IND for a paediatric trial entitled "a phase I study of 2-hydroxyoleic acid (2OHOA) in pediatric patients with malignant glioma and other advanced solid tumors (Press release, Laminar Pharma, MAY 29, 2018, View Source [SID1234562094])"

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This will be the first clinical study with 2OHOA in children with advanced malignant cancer and will be conducted in collaboration with two leading US paediatric clinical research institutions: Hackensack University Medical Center, in New Jersey and Dana-Farber Cancer Institute in Boston. The main objectives of this study are to determine the safety and tolerability of 2OHOA in paediatric population (under 18 years), to characterize the pharmacokinetic profiles in this population and to assess the preliminary anti-tumour efficacy of the product. The trial follows a standard 3+3 design in the dose escalation phase, where 9 to 18 patients will be recruited in three cohorts, and that will be followed by an expansion cohort with 10 additional patients.

High-grade gliomas (HGG) are relatively rare forms of paediatric brain tumours, constituting only 8–12% of primary central nervous system (CNS) tumours in children. The management of these tumours involves surgical resection to the extent feasible, as well as adjuvant radiation and chemotherapy. Even with these interventions, the prognosis for patients with these tumours is poor, with most patients succumbing to their disease within 12–18 months. The incidence rate of primary malignant and non-malignant brain and CNS tumours in the US in paediatric and adolescent population (0-19 years) is 5.42 cases per 100,000 for a total count of around 23,000 incident tumours per year, of which over 2.500 cases correspond to HGG.

Laminar Pharma is committed to advance the clinical development of this promising product and is excited about the prospect of providing a potential therapeutic alternative for children and adult patients with brain and other aggressive cancers.

The approval by the FDA of the Investigational New Drug (IND) application for this paediatric trial in the US is a significant regulatory milestone for 2OHOA, (re)validating the extensive preclinical and clinical development that Laminar Pharma is carrying out with this innovative product.

A PIIb trial in adult patients with newly-diagnosed glioblastoma (CLINGLIO) with 2OHOA added to the current chemoradiation standard of care for this type of tumours is also planned to open within this year in leading Hospitals in Europe and Israel, following the award of an important H2020 grant by the European Commission to an international consortium lead by Laminar Pharma.

Arcus Biosciences Announces Participation at Upcoming Investor Conference

On May 29, 2018 Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage biopharmaceutical company focused on creating innovative cancer immunotherapies, reported that Terry Rosen, Ph.D., Chief Executive Officer, will participate in a fireside chat at the Goldman Sachs 39th Annual Global Healthcare Conferenceon Tuesday, June 12, 2018at 8:40 am PT at the Terranea Resort, in Rancho Palos Verdes, CA (Press release, Arcus Biosciences, MAY 29, 2018, View Source [SID1234526921]).

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Individuals may access the live audio webcast by visiting the "Events & Presentations" section of the Company’s website at View Source A replay of the webcast will be available for 30 days following the live event.

Genmab to Present at the Goldman Sachs 39th Annual Global Healthcare Conference

On May 29, 2018 Genmab A/S (Nasdaq Copenhagen: GEN) reported that its CEO, Jan van de Winkel, Ph.D., will present a company update at the Goldman Sachs 39th Annual Global Healthcare Conference in Rancho Palos Verdes, California at 4:00 PM PDT on June 12, 2018 (1:00 AM CEST on June 13, 2018) (Press release, Genmab, MAY 29, 2018, View Source [SID1234527080]). A webcast of the presentation will be available on Genmab’s website at View Source

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FDA Accepts Larotrectinib New Drug Application and Grants Priority Review

On May 29, 2018 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company innovating the development of highly selective medicines for patients with genetically defined cancers, reported that the U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) and granted Priority Review for larotrectinib for the treatment of adult and pediatric patients with locally advanced or metastatic solid tumors harboring an NTRK gene fusion (Press release, Loxo Oncology, MAY 29, 2018, View Source [SID1234526924]). The FDA has set a target action date of November 26, 2018, under the Prescription Drug User Fee Act (PDUFA).

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"We are excited the larotrectinib NDA has been accepted by FDA and granted Priority Review status," said Josh Bilenker, M.D., chief executive officer of Loxo Oncology. "Larotrectinib marks an important shift towards treating cancer based on the tumor’s genetics rather than its site of origin in the body."

The FDA grants Priority Review for the applications of medicines that, if approved, would provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. Larotrectinib has also been granted Breakthrough Therapy Designation, Rare Pediatric Disease Designation and Orphan Drug Designation by the FDA.

Loxo Oncology and Bayer are engaged in a collaboration for the development and commercialization of larotrectinib. Bayer plans to submit a Marketing Authorization Application (MAA) in the European Union in 2018.
About Larotrectinib (LOXO-101)
Larotrectinib is an oral and highly selective investigational tropomyosin receptor kinase (TRK) inhibitor in clinical development for the treatment of patients with cancers that harbor a neurotrophic tyrosine receptor kinase (NTRK) gene fusion. Growing research suggests that the NTRK genes, which encode for TRKs, can become abnormally fused to other genes, resulting in growth signals that can lead to cancer in many sites of the body. In clinical trials, larotrectinib demonstrated anti-tumor activity in patients with tumors harboring NTRK gene fusions, regardless of patient age or tumor type. In an analysis of 55 RECIST-evaluable adult and pediatric patients with NTRK gene fusions, larotrectinib demonstrated a 75 percent centrally-assessed confirmed overall response rate (ORR) and an 80 percent investigator-assessed confirmed ORR, across many different types of solid tumors. The majority of all adverse events were grade 1 or 2.

Larotrectinib has been granted Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation and Orphan Drug Designation by the U.S. FDA.

In November 2017, Loxo Oncology and Bayer entered into an exclusive global collaboration for the development and commercialization of larotrectinib and LOXO-195, a next-generation TRK inhibitor. Bayer and Loxo Oncology will jointly develop the two products with Loxo Oncology leading the ongoing clinical studies as well as the filing in the U.S., and Bayer leading ex-U.S. regulatory activities and worldwide commercial activities. In the U.S., Loxo Oncology and Bayer will co-promote the products.

For additional information about the larotrectinib clinical trials, please refer to www.clinicaltrials.gov. Interested patients and physicians can contact the Loxo Oncology Physician and Patient Clinical Trial Hotline at 1-855-NTRK-123 or visit www.loxooncologytrials.com/trk-trials.

About TRK Fusion Cancer
TRK fusion cancer occurs when a neurotrophic tyrosine receptor kinase (NTRK) gene fuses with another unrelated gene, producing an altered tropomyosin receptor kinase (TRK) protein. The altered protein, or TRK fusion protein, is constantly active, triggering a permanent signal cascade. These proteins become the primary driver of the spread and growth of tumors in patients with TRK fusion cancer. TRK fusion cancer is not limited to certain types of cells or tissues and can occur in any part of the body. NTRK gene fusions occur in various adult and pediatric solid tumors with varying prevalence, including appendiceal cancer, breast cancer, cholangiocarcinoma, colorectal cancer, GIST, infantile fibrosarcoma, lung cancer, mammary analogue secretory carcinoma of the salivary gland, melanoma, pancreatic cancer, thyroid cancer, and various sarcomas. It may affect greater than 60 percent of both adult and pediatric patients with certain rare tumor types, such as secretory breast, secretory salivary gland and infantile fibrosarcoma. Only sensitive and specific tests can reliably detect TRK fusion cancer. Next-generation sequencing (NGS) can provide a comprehensive view of genomic alterations across a large number of genes. Fluorescence in situ hybridization (FISH) can also be used to test for TRK fusion cancer, and immunohistochemistry (IHC) can be used to detect the presence of TRK protein

Ipsen Announces Clinical Data to Be Presented at ASCO Demonstrating Its Commitment to Patients with Cancer

On May 29, 2018 Ipsen (Euronext: IPN; ADR: IPSEY) reported that cabozantinib (Cabometyx) and irinotecan liposome injection (Onivyde) are the subject of 8 presentations at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting. The meeting takes place in Chicago, Illinois, June 1-5, 2018 (Press release, Ipsen, MAY 29, 2018, View Source [SID1234526925]).

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Alexandre Lebeaut, Executive Vice President, R&D and Chief Scientific Officer, Ipsen stated: "Ipsen’s oncology products, notably Onivyde, Decapeptyl and Cabometyx, have been evaluated by many scientific teams around the world: either directly by investigators, our partners or by Ipsen. Results from some of these investigations will be the subject of oral abstract sessions. We are committed in our efforts against cancer, and through our interactions at ASCO (Free ASCO Whitepaper) 2018 we will continue to advance innovation for patient care in Oncology ."

Cabozantinib (Cabometyx) will be presented in 7 abstracts:

Cabozantinib (Cabometyx) will be featured in one oral poster discussion:

[Abstract 4019] Poster 208 Discussion: Sunday, 3 June, 16:45–18:00, Hall D2
Cabozantinib (C) versus placebo (P) in patients (pts) with advanced hepatocellular carcinoma (HCC) who have received prior sorafenib: results from the randomized phase 3 CELESTIAL trial
Presenting author: Ghassan Abou-Alfa [Sponsor: Exelixis]
Cabozantinib (Cabometyx) will be featured in other presentations:

[Abstract 4556] Poster 382 – Category: Genitourinary (Nonprostate) Cancer; Saturday, 2 June, 8:00–11:30, Hall A
Title: Quality-adjusted time without symptoms or toxicity (Q-TWiST): Analysis of cabozantinib (Cabo) vs sunitinib (Sun) in patients with advanced renal cell carcinoma (aRCC) of intermediate or poor risk (Alliance A031203)
Presenting author: Ronald Chen [Sponsor: Ipsen]
[Abstract 4598] Poster 418a – Category: Genitourinary (Nonprostate) Cancer; Saturday, 2 June, 8:00–11:30, Hall A
A phase 3, randomized, open-label study of nivolumab combined with cabozantinib vs sunitinib in patients with previously untreated advanced or metastatic renal cell carcinoma (RCC; CheckMate 9ER)
Presenting author: Toni Choueiri [Sponsor: Bristol-Myers Squibb, Exelixis & Ipsen]
[Abstract 4601] Poster 419b – Category: Genitourinary (Nonprostate) Cancer; Saturday, 2 June, 8:00–11:30, Hall A
CANTATA: A randomized phase 2 study of CB-839 in combination with cabozantinib vs. placebo with cabozantinib in patients with advanced/metastatic renal cell carcinoma
Presenting author: Nizar Tannir [Sponsor: Calithera Biosciences, Inc]
[Abstract 4088] Poster 277 – Category: Gastrointestinal (Noncolorectal) Cancer; Sunday, 3 June, 8:00–11:30, Hall A
Outcomes based on receipt of sorafenib (S) as the only prior systemic therapy in the phase 3 CELESTIAL trial of cabozantinib (C) versus placebo (P) in advanced hepatocellular carcinoma (HCC)
Presenting author: Robin Kelley [Sponsor: Exelixis]
[Abstract 4090] Poster 279 – Category: Gastrointestinal (Noncolorectal) Cancer; Sunday, 3 June, 8:00–11:30, Hall A
Outcomes based on age in the phase 3 CELESTIAL trial of cabozantinib (C) versus placebo (P) in patients (pts) with advanced hepatocellular carcinoma (HCC)
Presenting author: Lorenza Rimassa [Sponsor: Exelixis]
[Abstract TPS 4157] Poster 333a – Category: Gastrointestinal (Noncolorectal) Cancer; Sunday, 3 June, 8:00–11:30, Hall A
A phase II trial of cabozantinib and erlotinib for patients with EGFR and c-MET co-expressing metastatic pancreatic adenocarcinoma
Presenting author: Olumide Gbolahan [Sponsor: Exelixis]
nal-IRI / liposomal irinotecan (ONIVYDE) will be featured in 1 poster:

[Abstract 4111] Poster 300 – Category: Gastrointestinal (Noncolorectal) Cancer; Sunday, 3 June, 8:00–11:30, Hall A
A phase 1/2, open-label dose-escalation study of liposomal irinotecan (nal-IRI) plus 5- fluorouracil/leucovorin (5-FU/LV) and oxaliplatin (OX) in patients with previously untreated metastatic pancreatic cancer (mPAC).
Presenting author: Andrew Dean [Sponsor: Ipsen]
ABOUT ONIVYDE (irinotecan liposome injection)

ONIVYDE is an encapsulated formulation of irinotecan available as a 43 mg/10 mL single dose vial. This long-circulating liposomal form is designed to increase length of tumor exposure to both irinotecan and its active metabolite, SN- 38.

On April 3, 2017, Ipsen completed the acquisition from Merrimack Pharmaceuticals of ONIVYDE and gained exclusive commercialization rights for the current and potential future indications for ONIVYDE in the U.S., as well as the current licensing agreements with Shire for commercialization rights ex-U.S. and PharmaEngine for Taiwan.

ONIVYDE is approved by the U.S. FDA in combination with fluorouracil (5-FU) and leucovorin (LV) for the treatment of patients with metastatic adenocarcinoma of the pancreas after disease progression following gemcitabine-based therapy. Limitation of Use: ONIVYDE is not indicated as a single agent for the treatment of patients with metastatic adenocarcinoma of the pancreas.

IMPORTANT SAFETY INFORMATION – UNITED STATES

BOXED WARNINGS: SEVERE NEUTROPENIA and SEVERE DIARRHEA

Fatal neutropenic sepsis occurred in 0.8% of patients receiving ONIVYDE. Severe or life- threatening neutropenic fever or sepsis occurred in 3% and severe or life-threatening neutropenia occurred in 20% of patients receiving ONIVYDE in combination with 5-FU and LV. Withhold ONIVYDE for absolute neutrophil count below 1500/mm3 or neutropenic fever. Monitor blood cell counts periodically during treatment
Severe diarrhea occurred in 13% of patients receiving ONIVYDE in combination with 5-FU/LV. Do not administer ONIVYDE to patients with bowel obstruction. Withhold ONIVYDE for diarrhea of Grade 2–4 severity. Administer loperamide for late diarrhea of any severity. Administer atropine, if not contraindicated, for early diarrhea of any severity
CONTRAINDICATION

ONIVYDE is contraindicated in patients who have experienced a severe hypersensitivity reaction to ONIVYDE or irinotecan HCl
WARNINGS AND PRECAUTIONS

Severe Neutropenia: See Boxed WARNING. In patients receiving ONIVYDE/5-FU/LV, the incidence of Grade 3/4 neutropenia was higher among Asian (18/33 [55%]) vs White patients (13/73 [18%]). Neutropenic fever/neutropenic sepsis was reported in 6% of Asian vs 1% of White patients
Severe Diarrhea: See Boxed WARNING. Severe and life-threatening late-onset (onset >24 hours after chemotherapy [9%]) and early-onset diarrhea (onset ≤24 hours after chemotherapy [3%], sometimes with other symptoms of cholinergic reaction) were observed
Interstitial Lung Disease (ILD): Irinotecan HCl can cause severe and fatal ILD. Withhold ONIVYDE in patients with new or progressive dyspnea, cough, and fever, pending diagnostic evaluation. Discontinue ONIVYDE in patients with a confirmed diagnosis of ILD
Severe Hypersensitivity Reactions: Irinotecan HCl can cause severe hypersensitivity reactions, including anaphylactic reactions. Permanently discontinue ONIVYDE in patients who experience a severe hypersensitivity reaction
Embryo-Fetal Toxicity: ONIVYDE can cause fetal harm when administered to a pregnant woman. Advise females of reproductive potential to use effective contraception during and for 1 month after ONIVYDE treatment
ADVERSE REACTIONS

The most common adverse reactions (≥20%) were diarrhea (59%), fatigue/asthenia (56%), vomiting (52%), nausea (51%), decreased appetite (44%), stomatitis (32%), and pyrexia (23%)
The most common Grade 3/4 adverse reactions (≥10%) were diarrhea (13%), fatigue/asthenia (21%), and vomiting (11%)
Adverse reactions led to permanent discontinuation of ONIVYDE in 11% of patients receiving ONIVYDE/5-FU/LV; The most frequent adverse reactions resulting in discontinuation of ONIVYDE were diarrhea, vomiting, and sepsis
Dose reductions of ONIVYDE for adverse reactions occurred in 33% of patients receiving ONIVYDE/5-FU/LV; the most frequent adverse reactions requiring dose reductions were neutropenia, diarrhea, nausea, and anemia
ONIVYDE was withheld or delayed for adverse reactions in 62% of patients receiving ONIVYDE/5-FU/LV; the most frequent adverse reactions requiring interruption or delays were neutropenia, diarrhea, fatigue, vomiting, and thrombocytopenia
The most common laboratory abnormalities (≥20%) were anemia (97%), lymphopenia (81%), neutropenia (52%), increased ALT (51%), hypoalbuminemia (43%), thrombocytopenia (41%), hypomagnesemia (35%), hypokalemia (32%), hypocalcemia (32%), hypophosphatemia (29%), and hyponatremia (27%)
DRUG INTERACTIONS

Avoid the use of strong CYP3A4 inducers, if possible, and substitute non-enzyme inducing therapies ≥2 weeks prior to initiation of ONIVYDE
Avoid the use of strong CYP3A4 or UGT1A1 inhibitors, if possible, and discontinue strong CYP3A4 inhibitors ≥1 week prior to starting therapy
USE IN SPECIFIC POPULATIONS

Pregnancy and Reproductive Potential: See WARNINGS & PRECAUTIONS. Advise males with female partners of reproductive potential to use condoms during and for 4 months after ONIVYDE treatment
Lactation: Advise nursing women not to breastfeed during and for 1 month after ONIVYDE treatment
Please see full U.S. Prescribing Information for ONIVYDE.

ABOUT CABOMETYX (UK)

CABOMETYX 20mg, 40mg and 60mg film-coated unscored tablets

Active ingredient: Cabozantinib (S)-malate 20mg, 40mg and 60mg

Other components: Lactose

Indications: CABOMETYX is indicated for the treatment of advanced renal cell carcinoma (RCC) in treatment-naïve adults with intermediate or poor risk or in adults following prior vascular endothelial growth factor (VEGF)-targeted therapy

Dosage and Administration: The recommended dose of CABOMETYX is 60 mg once daily. Treatment should continue until the patient is no longer clinically benefiting from therapy or until unacceptable toxicity occurs. Management of suspected adverse drug reactions may require temporary interruption and/or dose reduction of CABOMETYX therapy. For dose modification, please refer to full SmPC. CABOMETYX is for oral use. The tablets should be swallowed whole and not crushed. Patients should be instructed to not eat anything for at least 2 hours before through 1 hour after taking CABOMETYX.

Contraindications: Hypersensitivity to the active substance or to any of the excipients listed in the SmPC.

Special warnings and precautions for use:

Monitor closely for toxicity during first 8 weeks of therapy. Events that generally have early onset include hypocalcaemia, hypokalaemia, thrombocytopenia, hypertension, palmar-plantar erythrodysaesthesia syndrome (PPES), proteinuria, and gastrointestinal (GI) events.

Perforations and fistulas: serious gastrointestinal perforations and fistulas, sometimes fatal, have been observed with cabozantinib. Patients with inflammatory bowel disease, GI tumour infiltration or complications from prior GI surgery should be evaluated prior to therapy and monitored; if perforation and unmanageable fistula occur, discontinue cabozantinib.

Thromboembolic events: use with caution in patients with a history of or risk factors for thromboembolism; discontinue if acute myocardial infarction (MI) or other significant arterial thromboembolic complication occurs.

Haemorrhage: not recommended for patients that have or are at risk of severe haemorrhage.

Wound complications: treatment should be stopped at least 28 days prior to scheduled surgery (including dental).

Hypertension: monitor blood pressure (BP); reduce with persistent hypertension and discontinue should uncontrolled hypertension or hypertensive crisis occur.

Palmar-plantar erythrodysaesthesia (PPES): interrupt treatment if severe PPES occurs.

Proteinuria: discontinue in patients with nephrotic syndrome.

Reversible posterior leukoencephalopathy syndrome (RPLS): discontinue in patients with RPLS.

QT interval prolongation: use with caution in patients with a history of QT prolongation, those on antiarrythmics or with pre-existing cardiac disease.

Excipients: do not use in patients with hereditary problems of galactose intolerance, Lapp lactase deficiency or glucose-galactose malabsorption.

Interactions: Cabozantinib is a CYP3A4 substrate. Potent CYP3A4 inhibitors may result in an increase in cabozantinib plasma exposure (e.g. ritonavir, itraconazole, erythromycin, clarithromycin, grapefruit juice). Co-administration with CYP3A4 inducers may result in decreased cabozantinib plasma exposure (e.g. rifampicin, phenytoin, carbamazepine, phenobarbital, St John’s Wort). Cabozantinib may increase the plasma concentration of P-glycoprotein substrates (e.g. fexofenadine, aliskiren, ambrisentan, dabigatran etexilate, digoxin, colchicine, maraviroc, posaconazole, ranolazine, saxagliptin, sitagliptin, talinolol, tolvaptan). MRP2 inhibitors may increase cabozantinib plasma concentrations (e.g. cyclosporine, efavirenz, emtricitabine). Bile salt sequestering agents may impact absorption or reabsorption resulting in potentially decreased cabozantinib exposure. No dose adjustment when co-administered with gastric pH modifying agents. A plasma protein displacement interaction may be possible with warfarin. INR values should be monitored in such a combination. Women of childbearing potential/contraception in males and females: Ensure effective measures of contraception (oral contraceptive plus a barrier method) in male and female patients and their partners during therapy and for at least 4 months after treatment.

Pregnancy and lactation: CABOMETYX should not be used during pregnancy unless the clinical condition of the woman requires treatment. Lactation – discontinue breast-feeding during and for at least 4 months after completing treatment. Drive and use machines: Caution is recommended

Adverse reactions:The most common serious adverse reactions are hypertension, diarrhoea, PPES, pulmonary embolism, fatigue and hypomagnesaemia. Very common (≥1/10): anaemia, lymphopenia, neutropenia, thrombocytopenia, hypothyroidism, dehydration, decreased appetite, hyperglycaemia, hypoglycaemia, hypophosphataemia, hypoalbuminaemia, hypomagnesaemia, hyponatraemia, hypokalaemia, hyperkalaemia, hypocalcaemia, hyperbilirubinaemia, peripheral sensory neuropathy, dysgeusia, headache, dizziness, hypertension, dysphonia, dyspnoea, cough, diarrhoea, nausea, vomiting, stomatitis, constipation, abdominal pain, dyspepsia, oral pain, dry mouth, PPES, dermatitis acneiform, rash, rash maculopapular, dry skin, alopecia, hair colour change, pain in extremity, muscle spasms, arthralgia, proteinuria, fatigue, mucosal inflammation, asthenia, weight decreased, serum ALT, AST, and ALP increased, blood bilirubin increased, creatinine increased, triglycerides increased, white blood cell decreased, GGT increased, amylase increased, blood cholesterol increased, lipase increased. Common (≥1/100 to <1/10): abscess, tinnitus, pulmonary embolism, pancreatitis, abdominal pain upper, gastro-oesophageal reflux disease, haemorrhoids, pruritus, peripheral oedema, wound complications. Uncommon (≥1/1000 to <1/100): convulsion, anal fistula, hepatitis cholestatic, osteonecrosis of the jaw. Selected adverse events: GI perforation, fistulas, haemorrhage, RPLS. Prescribers should consult the SPC in relation to other adverse reactions.

For more information, see the regularly updated registered product information on the European Medicine Agency www.ema.europa.eu

ONIVYDE is a registered trademark of Ipsen Biopharm Limited.

CABOMETYX (cabozantinib), XERMELO (telotristat ethyl) and DECAPEPTYL (triptorelin) are not marketed by Ipsen in the United States. The approved indications may vary by country.

CABOMETYX is marketed by Exelixis, Inc. in the United States. Ipsen has exclusive rights for the commercialization and further clinical development of CABOMETYX outside of the United States and Japan.