20 Hottest Pipeline Drugs to Watch in 2019

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:

1. 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.

2. 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.

3. 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.

4. 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.

5. 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.

6. 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.

7. 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.

8. 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.

9. 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.

10. 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.

11. 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.

12. 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.

13. 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.

14. 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.

15. 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.

16. 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.

17. 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.

18. Celgene’s Luspatercept. On December 1, Celgene and Acceleron Pharma announcedresults 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.

19. 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.

20. Argenx’s ARGX-110. Argenx presented new data for ARGX-110 in AML at the 60^th 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.

https://www.biospace.com/article/1-report-the-20-most-valuable-pipeline-projects-in-2019/

Natera transplant data show no edge over CareDx, says Piper Jaffray

After Natera (NTRA) published its full cell-free DNA data for kidney rejection in the Journal of Clinical Medicine, Piper Jaffray analyst William Quirk reiterates an Overweight rating on CareDx (CDNA). The publication Natera does not have any advantage over CareDx largely due to the fact that creatinine, which is a non-specific marker for transplant rejection, showed statistical significance in identifying injury, Quirk tells investors in a research note. The analyst thinks “this important element” in the Natera data will require additional clinical work before the test could be considered competitive with CareDx’s. He continues to believe CareDx’s “first mover advantage is significant.”

https://thefly.com/landingPageNews.php?id=2842716

OrthoPediatrics files motion versus former employee and WishBone Medical

OrthoPediatrics files motion versus former employee and WishBone Medical  issued a statement from Daniel Gerritzen, Vice President and General Counsel, concerning actions it filed against Robert von Seggern, a former employee, and WishBone Medical in an Indiana state court in late December 2018. These actions are part of OrthoPediatrics’ existing lawsuit against von Seggern and WishBone Medical for, in part, violations of Indiana’s and federal trade secrets statutes.The underlying lawsuit alleges that von Seggern stole OrthoPediatrics’ trade secrets and confidential information and then turned those over to WishBone Medical for its use in developing pediatric orthopedic products. The Sanctions Motion requests the Whitley Superior Court to sanction von Seggern and WishBone Medical for their repeated, deliberate efforts to subvert the discovery process by refusing to produce responsive documents and information, hiding or otherwise destroying evidence, and making misstatements to OrthoPediatrics and the Court concerning the existence of responsive documents and its efforts to find and produce such materials. “OrthoPediatrics welcomes legitimate competition because that means more kids with pediatric orthopedic conditions will have greater opportunities to be helped,” stated Gerritzen. “However, we will not tolerate individuals or entities misappropriating our property, in part, to leapfrog years of design and testing work and avoid the significant monetary expense of bringing competing products to market. With this in mind, we will continue to aggressively pursue every legal remedy to protect and safeguard our research and product development efforts to improve the lives of children with pediatric conditions. This includes holding those accountable who attempt to use, without authorization, OrthoPediatrics’ trade secrets and confidential information. The trade secrets and confidential information we have become aware von Seggern and WishBone Medical misappropriated relates to aspects of our current Trauma and Deformity Correction medical devices. While very serious, the misappropriation does not significantly jeopardize our overall research and development pipeline

https://thefly.com/landingPageNews.php?id=2842766

Crispr Therapeutics named a top pick for 2019 at Piper Jaffray

Crispr Therapeutics named a top pick for 2019 at Piper Jaffray. Piper Jaffray analyst Edward names Crispr Therapeutics a top pick for 2019 and reiterates an Overweight rating on the shares with a $75 price target. The stock closed the trading day up $1.30 to $29.87. Crispr is developing “revolutionary” CRISPR/Cas-9 technology as a new therapeutic modality with broad ranging applicability, Tenthoff tells investors in a research note. The Phase I/II studies of CTX-001 in transfusion-dependent beta thalassemia and sickle cell disease will report first-in-man data this year potentially curing patients of disease, adds the analyst.

https://thefly.com/landingPageNews.php?id=2842778

FDA expands indication for Bristol-Myers’ Sprycel tablets

Bristol-Myers Squibb announced the U.S. Food and Drug Administration has expanded the indication for Sprycel tablets to include the treatment of pediatric patients one year of age and older with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia in combination with chemotherapy. Sprycel is the only second-generation tyrosine kinase inhibitor approved for this patient population. The approval, which was granted following priority review by the FDA, is based on data from the Phase 2 study, CA180-372. “We recognize the urgency around developing and delivering therapies for children and young adults living with cancer, and today’s approval is an important example of our commitment to pediatric oncology,” said Jeffrey Jackson, Ph.D., development lead, hematology, Bristol-Myers Squibb. “Building on our previous indication for children with Ph+ chronic myeloid leukemia in chronic phase, we’re pleased to bring Sprycel tablets to a second type of pediatric leukemia. This approval will give physicians another treatment option to offer appropriate pediatric patients with Ph+ ALL.”

https://thefly.com/landingPageNews.php?id=2842790

Waters initiated at Needham

Waters initiated with a Hold at Needham. Needham analyst Stephen Unger started Waters Corp. with a Hold rating and $177 fair value estimate.

https://thefly.com/landingPageNews.php?id=2842793

Humana revises FY19 Medicare Advantage members to 375,000-400,000

Based on final annual enrollment period results, the company is revising its net membership growth estimate for its individual Medicare Advantage products for the year ended December 31, 2019 to 375,000 to 400,000 members, compared to its previous estimate of 350,000 to 400,000 members. The revised membership estimate represents expected net membership gains of 12%-13% above the year ended December 31, 2018 membership level of approximately 3.07M members. The company also reiterates its expectations for group Medicare Advantage net membership gains for the same period, projecting an increase of approximately 30,000 members year over year. The membership estimate represents an expected net membership gain of 6% above the year ended December 31, 2018 membership level of approximately 500,000 members. For PDP, the company now estimates a net membership decline of 700,000 to 750,000 members for the year ended December 31, 2019, compared to its previous estimate of a decline of 750,000 to 800,000 members. The membership estimate represents a decline of 14%-15% below the year ended December 31, 2018 membership level of approximately 5M members. The revised estimate is primarily attributable to improved retention estimates.

https://thefly.com/landingPageNews.php?id=2842798