EvaluatePharma and Vantage recently released their Vantage 2019 Preview which looks at the current year’s biopharma market and makes projections about the upcoming year. Although it was an amazing year for biotech initial public offerings (IPO), the stock market’s volatility at the end of the year and other factors have made 2019 appear “on very shaky footing.”
At least in terms of investing. The report believes venture capital firms will be a little more cautious in 2019, although biotech IPOs will likely still be strong in the upcoming year.
On the product side, the report lists 20 pipeline projects to keep an eye on. Here’s a look:
- Vertex’s VX-659 + Tezacaftor + Ivacaftor. On September 6, Vertex Pharmaceuticalsannounced it had completed enrollment for two Phase III clinical trials for the triple-combination treatment for cystic fibrosis (CF) with one F508del mutation and one minimal function mutation and in people with two F508del mutations. In November, the company announced the trial had met its primary endpoint of improvement in lung function. More data on related trials are expected by mid-2019.
- Celgene’s JCAR017. Celgene’s anti-CD19 CAR-T therapy is in Phase II/III trials for lymphoma and leukemia. The company is expecting data readouts in 2019.
- Novo Nordisk’s semaglutide oral. Semaglutide is Novo Nordisk’s glucagon-like peptide-1 receptor agonist for type 2 diabetes. It has already been approved as a once-weekly injection. In November the company announced that patients on the once-daily tablet version showed a 21 percent decrease in cardiovascular events. The company expects to submit an application to the U.S. Food and Drug Administration (FDA) in the first half of 2019.
- Biogen’s aducanumab. Biogen’s much-watched monoclonal antibody to clear beta-amyloid in Alzheimer’s patients is expecting pivotal data in late 2019 or early 2020.
- Argenx’s ARGX-113. On December 3, Argenx reported data from its Phase II trial of efgartigimod (ARGX-113) in immune thrombocytopenia (ITP). Additional Phase II data and the start of the Phase III trial is expected in 2019.
- Nektar Therapeutics’ NKTR-214. On November 9, Nektar Therapeutics announced clinical and preclinical data from its Phase I/II trial of NKTR-214 plus nivolumab in previously untreated patients with metastatic Stage IV melanoma, as well as preclinical data on the drug with various other therapies. Additional data from the Phase II trial is expected in 2019.
- GENFIT’s elafibranor. Genfit’s elafibranor is being evaluated in several trials for nonalcoholic steatohepatitis (NASH) and primary biliary cholangitis (PBC). On December 6, the company announced positive results from its Phase II trial in PBC. On December 17, a Data Safety Monitoring Board (DSMB) recommended a continuation of RESOLVE-IT, the company’s Phase III trial of the drug in NASH. Topline data is expected by the end of 2019.
- GlaxoSmithKline’s GSK2857916. GlaxoSmithKline is developing the drug for multiple myeloma. It expects Phase II data and the start of a Phase III in 2019.
- Celgene’s ozanimod. Earlier this year, the FDA issued Celgene a Refusal to File letter over its New Drug Application (NDA) for its multiple sclerosis drug ozanimod. For a company of Celgene’s size and experience, it came as quite a shock. After re-evaluation and some mea culpas and finger-pointing, the company expects to resubmit the NDA in 2019.
- Daiichi Sankyo’s DS-8201. On September 25, Daiichi Sankyo released updated Phase I safety and efficacy data for trastuzumab deruxtecan (DS-8201), an investigational HER2 targeting antibody-drug conjugate (ADC) in non-small cell lung cancer (NSCLC). Phase III breast cancer data is expected in 2020.
- BioMarin’s valoctocogene roxaparvovec. At BioMarin’s third-quarter results on October 25, the company noted it had presented two years of data on the drug for severe Hemophilia A from the ongoing Phase I/II study at the World Federation of Hemophilia (WFH) 2018 World Congress in the second quarter of the year. The company has noted the potential accelerated filing based on Phase I/II data in the second half of 2019.
- Solid Biosciences’ SGT-001. Earlier this year, the FDA placed a clinical hold on IGNITE DMD, Solid Biosciences’ Phase I/II clinical trial of its microdystrophin gene transfer, SGT-001, for Duchenne muscular dystrophy. In June, the agency lifted the hold. The company relaunched the trial. At the company’s third-quarter report in September, it indicated there were a total of six patients in the trial and no serious adverse events have been observed. Solid says it plans to announce preliminary data in the first quarter of 2019.
- Global Blood Therapeutics’ voxelotor. On December 4, Global Blood Therapeutics announced the FDA had agreed to its proposal for an accelerated approval pathway for voxelotor for sickle cell disease. It plans to file an NDA under this pathway in 2019, which includes a post-approval confirmatory study to demonstrate stroke risk reduction with transcranial doppler flow velocity as its primary endpoint.
- Pfizer’s PF-05280586. This is Pfizer’s biosimilar for Genentech and Biogen’s Rituxan for follicular lymphoma, diffuse large B-cell lymphoma and chronic lymphocytic leukemia. The company is looking for FDA approval in the third quarter of 2019.
- Celgene and bluebird bio’s bb2121. On November 27, Celgene and bluebird bio announcedthey had completed enrollment for the KarMMa pivotal trial of bb2121, their lead investigational anti-BCMA CAR-T therapy for patients with relapsed and refractory multiple myeloma. Phase III data and filing are expected in 2019. There is an expected target action date expected in 2020.
- Gilead Sciences and Galapagos’ filgotinib. In September, Gilead Sciences and Galapagos’ filgotinib met its primary endpoints in adults with moderately-to-severely active rheumatoid arthritis. Filgotinib is a selective JAK1 inhibitor. Additional Phase III data and regulatory filings are expected in 2019.
- FibroGen’s pamrevlumab. In September, the FDA granted FibroGen Fast Track designation for pamrevlumab, an anti-CTGF antibody for the treatment of idiopathic pulmonary fibrosis (IPF). The company also expects Phase III trials in IPF and pancreatic cancer to start in 2019.
- Celgene’s Luspatercept. On December 1, Celgene and Acceleron Pharma announced results from their Phase III trial of luspatercept for adults with beta-thalassemia-associated anemia who require regular red blood cell transfusions. The drug met the primary endpoint of erythroid response. They companies plan to submit the drug to regulatory authorities in the U.S. and Europe in the first half of 2019.
- Madrigal Pharmaceuticals’ MGL-3196. On November 12, Madrigal Pharmaceuticalsreleased results from its Phase II clinical trial in patients with biopsy-proven NASH for its MGL-3196. The drug showed a highly statistically significant reduction in liver fat, lowering of multiple atherogenic lipids including LDL-C, ApoB, triglycerides, ApoCIII and lipoprotein, and lowering of liver enzymes. The Phase III trial is expected to start in 2019.
- Argenx’s ARGX-110. Argenx presented new data for ARGX-110 in AML at the 60th American Society of Hematology Annual Meeting and Exposition. The results were from an ongoing Phase I dose-escalation part of its Phase III clinical trial in AML and high-risk myelodysplastic syndromes. Additional Phase II date is expected in 2019.
A Product Manager with expertise in pharma marketing and sales operations