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Week Ahead (Jun.14-18): FDA Decision, Data Readouts (RDUS, BPMC, ORPH, IPHA…)

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In the trading week that ended June 11, half a dozen stocks delivered more than 50% gain, with Aethlon Medical Inc. (AEMD) being the star performer.

Aethlon Medical closed out the week with over 190% gain, following encouraging news related to its investigational product Hemopurifier, a clinical-stage immunotherapeutic device designed to combat cancer and life-threatening viral infections.

Novan Inc. (NOVN), Orphazyme A/S (ORPH), Clover Health Investments, Corp. (CLOV), Prothena Corporation plc (PRTA), and Axcella Health Inc. (AXLA) were the other stocks which returned over 50% weekly gain.

Now, let's take a look at some of the stocks that are worth watching in the upcoming week.

1. Takeda Pharmaceutical Company Limited (TAK)

Takeda Pharma awaits the FDA decision on its investigational therapy budesonide oral suspension, TAK-721, which is scheduled for June 15.

TAK-721 is proposed for the treatment of eosinophilic esophagitis (EoE), a chronic inflammatory disease that can cause damage to the esophagus, often resulting in difficulty swallowing. About one in 2,000 people in the U.S. are affected by EoE.

If approved, TAK-721 will be the first FDA-approved treatment for eosinophilic esophagitis, and Takeda plans to use the trade name Eohilia.

Eohilia has the potential to achieve peak sales of $500 million, according to the company.

TAK closed Friday's (May 21, 2021) trading at $17.21, down 0.86%.

2. Radius Health Inc. (RDUS)

Radius Health has a Type C meeting with the FDA in the coming week related to RAD011, a pivotal-trial ready synthetic cannabidiol oral solution for the treatment of Prader-Willi syndrome.

The company plans to initiate a pivotal phase II/III study of RAD011 for patients with Prader-Willi syndrome in the second half of 2021 pending regulatory discussion with the FDA.

In a previous abbreviated phase II trial, RAD011 has been found to be directionally supportive of reducing both hyperphagia and has demonstrated a reduction in weight. The hallmark symptom of Prader-Willi syndrome is hyperphagia or insatiable appetite.

The company is slated to present the previous reported data of RAD011 at the PWSA/USA conference that will be held June 23-25, 2021.

RDUS closed Friday's (Jun.11, 2021) trading at $18.81, up 0.43%.

3. Blueprint Medicines Corporation (BPMC)

The FDA decision on Blueprint Medicines' AYVAKIT for the expanded use in the treatment of systemic mastocytosis is expected on June 16.

Systemic mastocytosis (SM) is a rare condition caused by the accumulation of mast cells in the body's organs, such as the skin, bone marrow, liver, spleen and small intestines. Mast cells are an important type of immune cell that normally play a role in the body helping fight infection and triggering the allergic response. In SM, the body produces too many mast cells, causing symptoms throughout the body.

AYVAKIT received its initial FDA approval in January 2020 for the treatment of gastrointestinal stromal tumors, harboring a PDGFRA exon 18 mutation. The drug generated sales of $21.3 million in full year 2020 in the indication of gastrointestinal stromal tumors.

AYVAKIT, if approved for the expanded use, could achieve peak sales of $737 million in the U.S. alone in advanced systemic mastocytosis, according to SVB Leerink analyst Andrew Berens.

Currently, Novartis' Rydapt is the only FDA-approved treatment for advanced systemic mastocytosis.

BPMC closed Friday's trading at $86.90, up 3.32%.

4. Orphazyme A/S (ORPH)

Orphazyme's MIPLYFFA, proposed for the treatment of Niemann-Pick type C, awaits the FDA decision, which is expected to be announced on June 17.

Niemann-Pick type C is a rare inherited lysosomal storage disorder disease characterized by a lack a protein that the body needs to break down fats and cholesterol within cells. There is no cure for this disease, which affects the brain and multiple other organs.

Johnson & Johnson's Zavesca is the standard of care for Niemann-Pick type C in Europe and is used off-label in the U.S.

MIPLYFFA is also under review in Europe, with a decision expected in the fourth quarter of this year.

If approved, MIPLYFFA would become the first approved therapy in the U.S. for people with Niemann-Pick disease Type C (NPC) and the drug could achieve peak global sales of $340 million, according to analysts.

5. Eton Pharmaceuticals (ETON)

The FDA decision on Eton Pharma's ET-203, a ready-to-use formulation of ephedrine injection, is expected on June 18. ET-203 was licensed from Sintetica SA, a Swiss-based pharmaceutical company.

The NDA for ET-203 was originally submitted to the FDA in July 2019 and Sintetica received a refuse-to-file letter. Addressing the concerns raised by the FDA, Sintetica resubmitted the NDA last year.

Eton expects the market opportunity for ET-203 to be over 10 million RTU units annually.

Nexus Pharma's Emerphed, launched in June 2020, is the first and only FDA-approved, Ready-to-Use Ephedrine Injection.

ETON closed Friday's (May 21, 2021) trading at $8.34, up 1.09%.

6. Innate Pharma S.A. (IPHA)

Innate Pharma will be presenting preliminary mycosis fungoides data from its phase II trial of Lacutamab in patients with advanced T-cell lymphomas, dubbed TELLOMAK, at the virtual 16th International Conference on Malignant Lymphoma, on June 22.

In the TELLOMAK trial, Lacutamab is evaluated as a single agent in approximately 60 patients with Sézary syndrome who have received at least two prior systemic therapies and as a single agent in approximately 90 patients with mycosis fungoides (MF) who have received at least two systemic therapies.

In February of this year, the company announced that Lacutamab showed a positive early signal in the KIR3DL2-expressing mycosis fungoides cohort of the TELLOMAK clinical trial.

IPHA closed Friday's trading at $4.14, up 0.24%.

7. Incyte Corp. (INCY)

An FDA panel is scheduled to review Incyte's Retifanlimab injection on June 24.

Retifanlimab is proposed for the treatment of adult patients with locally advanced or metastatic squamous carcinoma of the anal canal (SCAC) who have progressed on or who are intolerant of platinum-based chemotherapy.

Squamous cell carcinoma of the anal canal, which accounts for nearly 3% of digestive system cancers, is associated with human papillomavirus (HPV) and HIV infections. Patients with advanced SCAC have a low 5-year survival rate, and there are currently no FDA-approved therapies for individuals who have progressed after first-line chemotherapy.

The FDA decision on Retifanlimab is expected on July 25, 2021.

INCY closed Friday's trading at $82.58, down 5.66%.

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