Biotech Stocks Facing FDA Decision In December 2023

(RTTNews) - The month of November witnessed a couple of notable firsts, including the approval of the first treatment for congenital thrombotic thrombocytopenic purpura, the first chikungunya vaccine and the first treatment for progressing desmoid tumors.

So far, this year, the FDA has approved 52 novel drugs compared to just 37 for the full year of 2022.

Now, let's take a look at the biotech companies awaiting FDA decision in December.

Vertex Pharmaceuticals Inc. (VRTX)

The final decision of FDA on Vertex Pharma's Exa-cel in the proposed treatment of severe sickle cell disease is due on December 8, 2023.

Exa-cel, formerly known as CTX001, is a CRISPR/Cas9 gene-edited therapy, developed by Vertex and CRISPR Therapeutics.

An FDA advisory committee meeting for Exa-cel in sickle cell disease was completed in October of this year.

Exa-cel received approval in the U.K. on Nov.16, 2023, under the brand name Casgevy. This marks a historic milestone as Casgevy becomes the first CRISPR-based gene therapy globally, specifically designed for addressing sickle cell disease and transfusion-dependent beta thalassemia.

VRTX closed Wednesday's (Nov.29, 2023) trading at $351, up 1.14%.

2seventy bio Inc. (TSVT)

The FDA decision on the expanded use of Abecma, in the proposed treatment of adult patients with relapsed and refractory multiple myeloma who have received an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody, is due on December 16, 2023.

Abecma is already approved for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody.

The drug is being jointly developed and commercialized in the U.S. by Bristol Myers Squibb (BMY) and 2seventy bio (TSVT). Bristol Myers Squibb assumes sole responsibility for Abecma drug product manufacturing and commercialization outside of the U.S.

On Nov.20, 2023, the company was notified that an FDA panel would review the application seeking approval for the expanded use of Abecma and therefore a decision is unlikely to be made on the assigned date of December 16.

Abecma U.S. revenues, as reported by Bristol Myers Squibb, were $69 million in the third quarter of 2023 compared to $75 million in the third quarter of 2022.

TSVT closed Wednesday's trading at $1.92, up 2.13%.

OptiNose Inc. (OPTN)

OptiNose Inc. (OPTN) is seeking FDA approval for the expanded use of its lead drug Xhance in the treatment of chronic rhinosinusitis, and a decision is anticipated on December 16, 2023.

Chronic rhinosinusitis is a serious nasal inflammatory condition characterized by nasal congestion/obstruction, facial pain and pressure, rhinorrhea (drainage and postnasal drip), and loss of sense of smell and taste. As many as 30 million adults in the United States are affected by chronic rhinosinusitis.

There are 2 subgroups namely chronic rhinosinusitis with nasal polyps and chronic rhinosinusitis without nasal polyps.

Xhance received its initial FDA approval for the treatment of nasal polyps and that was in September 2017.

If approved for the expanded use, Xhance would be the first and only drug indicated for the treatment of chronic rhinosinusitis either with or without nasal polyps.

For the nine-month period ended September 30, 2023, Xhance's sales totaled $51.1 million, a decline of 8% compared to $55.4 million during the nine-month period ended September 30, 2022.

OPTN closed Wednesday's trading at $1.25, up 1.63%.

Arcutis Biotherapeutics Inc. (ARQT)

The FDA decision on Arcutis Biotherapeutics' Roflumilast foam, proposed for the treatment of moderate-to-severe seborrheic dermatitis, is due on December 16, 2023.

Seborrheic dermatitis is a common chronic or recurrent skin condition that causes red patches covered with large, greasy, flaking yellow-gray scales, and persistent itch.

Roflumilast cream of 0.3% strength was approved by the FDA in July 2022 for the treatment of plaque psoriasis in adults and adolescents 12 years of age and older. and is marketed under the brand name Zoryve.

ARQT closed Wednesday's trading at $1.90, down 6.86%.

Calliditas Therapeutics AB (CALT)

The FDA decision to convert the accelerated approval of Calliditas Therapeutics' Tarpeyo to full approval is expected on December 20, 2023.

Tarpeyo contains the active ingredient Budesonide, a synthetic corticosteroid. The drug received accelerated approval from the FDA in December 2021 to reduce proteinuria in adults with primary immunoglobulin A nephropathy. It received conditional marketing authorization from the European Commission in July 2022, and is marketed under the trade name Kinpeygo.

For the third quarter ended September 30, 2023, Tarpeyo net sales were SEK 283.6 million compared to SEK 123.4 million in the year-ago quarter.

CALT closed Wednesday's trading at $18.20, down 2.93%.

bluebird bio Inc. (BLUE)

bluebird bio's investigational gene therapy Lovotibeglogene autotemcel, a one-time treatment proposed for the treatment of sickle cell disease, awaits the FDA decision on December 20, 2023.

In sickle cell disease, high concentrations of sickle hemoglobin (HbS) in red blood cells (RBCs) cause RBCs to become sickled, sticky, and rigid with a shorter life span, which manifests acutely as hemolytic anemia, vasculopathy, and vaso-occlusion.

Lovotibeglogene autotemcel, also known as Lovo-cel, is designed to introduce functional copies of a modified version of the ß-globin gene (ßA-T87Q-globin gene) into the hematopoietic (blood) stem cells (HSCs) of the patient. Once the patients acquire the ßA-T87Q-globin gene, their red blood cells (RBCs) can produce anti-sickling hemoglobin (HbAT87Q), which lowers the proportion of sickle hemoglobin. The aim is to decrease the occurrence of sickled RBCs, hemolysis, and other associated complications.

Vertex Pharma and CRISPR Therapeutics' Casgevy received approval in the U.K earlier this month, becoming the world-first gene therapy for sickle-cell disease.

BLUE closed Wednesday's trading at $3.86, up 0.52%.

Ionis Pharmaceuticals Inc. (IONS)

On December 22, 2023, the FDA will decide whether or not to approve Ionis Pharma's Eplontersen.

Eplontersen is an investigational antisense medicine for the treatment of people living with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). It is being jointly developed and commercialized by Ionis and AstraZeneca in the U.S. and will be developed and commercialized in the rest of the world by AstraZeneca, with the exception of Latin America.

Patients with ATTRv-PN suffer ongoing nerve damage throughout their body, causing a gradual loss of motor function. The accumulation of protein Transthyretin in vital organs further impairs their function, leading to death within five to fifteen years of the disease onset.

If approved, Eplontersen would have to compete with Alnylam's Onpattro and Amvuttra.

IONS closed Wednesday's trading at $49.41, up 2.21%.

Glaukos Corp. (GKOS)

Glaukos' iDose TR, a micro-invasive intraocular implant designed to lower intraocular pressure in patients with open-angle glaucoma or ocular hypertension, is at the FDA altar, with a decision anticipated on December 22, 2023.

iDose TR is designed to continuously release therapeutic levels of a proprietary formulation of Travoprost from within the eye for extended duration. Its design allows for removal and replacement with a new iDose TR, potentially providing a drop-free alternative for long-term treatment compared to daily eye drop regimens, according to the company.

Travoprost solution is an approved medicine for the reduction of elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension.

GKOS closed Wednesday's trading at $62, up 3.77%.

Amgen Inc. (AMGN)

The FDA decision to convert the accelerated approval of Amgen's Lumakras to a traditional approval is expected on December 24, 2023.

Lumakras was granted accelerated approval by the FDA in May 2021, for the treatment of adult patients with KRAS G12C mutated locally advanced or metastatic non-small cell lung cancer.

The drug generated sales of $52 million for the third quarter of 2023, representing a decline of 31% over the year-ago period.

AMGN closed Wednesday's trading at $266.60, up 0.41%.

Merck & Co. Inc. (MRK)

Merck's Gefapixant, proposed for the treatment of refractory chronic cough or unexplained chronic cough in adults, which was voted down by an FDA panel on Nov.17, 2023, awaits the regulatory agency's final decision on December 27, 2023.

The FDA had declined to approve Gefapixant in January 2022, citing the need for more information related to measurement of efficacy.

Gefapixant, which is a non-narcotic, orally administered selective P2X3 receptor antagonist, is approved in Japan and Switzerland. A decision from the European Commission is expected later this year.

MRK closed Wednesday's trading at $101.13, up 0.95%.

Zealand Pharma A/S (ZLPDF.OB)

The FDA will decide whether or not to approve Zealand Pharma's Dasiglucagon on December 30, 2023.

Dasiglucagon is proposed for the prevention and treatment of hypoglycemia in pediatric patients 7 days of age and older with congenital hyperinsulinism for up to three weeks of dosing.

Congenital hyperinsulinism (CHI), a rare disease affecting mainly newborns and toddlers, is caused by a defect in pancreatic cells, leading to overproduction of insulin. High levels of insulin result in persistently and often severely low blood sugar levels (hypoglycemia).

CHI develops in one out of 50,000 (or fewer) children, which corresponds to 180-300 children diagnosed in the U.S. and Europe every year.

ZLPDF.OB closed Wednesday's trading at $$46.92, up 0.71%.