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6 Valuable Pipeline Drugs With Upcoming Catalysts

Shanthi Rexaline

Heading into December, biotech stocks held up fairly well in 2018. Evaluate Pharma, a firm specializing in data-driven news and analysis in biotech, compiled a list of blockbuster drugs in the works with catalytic events expected sometime in 2019. Let's dig in.

VX-659, Tezacaftor, Ivacaftor Triple Combo&

Sponsor Company: Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX)

Indication: Cystic fibrosis patients with one F508del mutation and one minimal function mutation, as well as patients with two F508del mutations.

Net Present Value: $14.4 billion

Current Status: data from two late-stage studies of this triple combo regimen released Nov. 27 showed improvement in lung function.

Catalyst: Potential NDA filing no later than mid-2019.

Vertex is also evaluating another triple combo regimen, namely VX-445, Tezacaftor and Ivacaftor, and said it will use the data readout from this combo due in Q1 and the results for the VX-659, Tezacaftor and Ivacaftor combo to decide which regimen should be submitted for potential regulatory approval.

Related Link: FDA Snubs Mallinckrodt's Abuse-Deterrent Opioid Reformulation

Liso-cell/JCAR017

Sponsor Company: Celgene Corporation (NASDAQ: CELG)

Indication: A CD-19 protein-targeting CAR T-cell therapy developed for B-cell lymphoma and leukemia.

Net Present Value: $8.5 billion.

Current Status: Preliminary results of a Phase 1/2 study dubbed TRANSCEND CLL 004 that evaluated JCAR017 in heavily pretreated CLL/SLL patients were presented at the ASH 2018 meeting in December. The results showed an 81-percent response rate and "undetectable" minimal residual disease in patients with both high-and standard-risk disease who had previously received Imbruvica.

Catalyst: The trial moves to Phase 2 portion in 2019.

Semaglutide Oral

Sponsor Company: Novo Nordisk A/S (NYSE: NVO)

Indication: Oral GLP-1 agonist for type 2 diabetes.

Net Present Value: $7.6 billion

Current Status: Headline results from the last global Phase 3a trial, dubbed PIONEER 6, released Nov. 23 demonstrated cardiovascular safety as well as a significant reduction in both CV and all-cause mortality in people with Type 2 diabetes who are at high cardiovascular risk.

Catalyst: regulatory filing in the U.S. and EU in the first half of 2019.

See also: Attention Biotech Investors: Mark Your Calendar For These December PDUFA Dates

Aducanumab

Sponsor Company: Biogen Inc (NASDAQ: BIIB)

Indication: mild cognitive impairment due to Alzheimer's disease and mild Alzheimer's disease.

Net Present Value: $7.4 billion.

Current Status: the drug is being evaluated in two separate Phase 3 trials dubbed ENGAGE and EMERGE.

Catalyst: Phase 3 data due in late 2019/early 2020.

Aducanumab is an anti-beta-amyloid monoclonal antibody. Thirty six- and 48-month data from the ongoing long-term extension Phase 1b trial presented at a conference in late October showed the results were consistent with previously reported analysis of the study.

Efgartigimod/ARGX-113

Sponsor Company: argenx SE – ADR (NASDAQ: ARGX)

Indication: AGRX-113, or efgartigimod, is a first-in-class antibody fragment used to treat patients with severe autoimmune diseases associated with high level of pathogenic immunoglobin G antibodies. It is being evaluated for several indications such as multiple sclerosis, immune thrombocytopenia, myasthenia gravis, pemphigus vulgaris and chronic inflammatory demyelinating polyneuropathy.

Net Present Value: $6.5 billion.

Current Status: Detailed Phase 2 data for efgartigimod in patients with immune thrombocytopenia presented at the ASH 2018 conference in early December showed clinically meaningful improvements in platelet counts.

Catalyst: The immune thrombocytopenia program is likely to be advanced to a Phase 3 trial in 2019, and efgartigimod with a subcutaneous mode of administration is to be tested in a Phase 2 trial in 2019.

NKTR-214

Sponsor Company: Nektar Therapeutics (NASDAQ: NKTR)

Indication: Along with Bristol-Myers Squibb Co (NYSE: BMY)'s Opdivo or Opdivo/Yervoy, NKTR-214, Nektar's lead candidate, is evaluated in more than 20 indications across nine tumor types.

Net Present Value: $6.2 billion.

Current Status: New data from the PIVOT-02 Phase 1/2 presented at the 2018 Society for Immunotherapy of Cancer Annual Meeting showed NKTR-214 has the potential to lead to tumor clearance and tumor specific immunologic memory when combined with therapies with complementary mechanisms of action.

Catalyst: Further data from the PIVOT-02 study is expected in 2019.

NKTR-214 binds to the CD122 receptor on the surface of cancer-fighting immune cells in order to stimulate their proliferation.

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