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Thursday, January 3, 2019

Deciphera Pharmaceuticals Initiates a Phase 1b/2 Cancer Trial

Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH), a clinical-stage biopharmaceutical company focused on addressing key mechanisms of tumor drug resistance, today announced that it has initiated an open-label, multicenter, Phase 1b/2 study of rebastinib, the Company’s investigational small molecule switch control inhibitor of TIE2 kinase, in combination with carboplatin in patients with advanced or metastatic solid tumors.
“We are extremely pleased with the recent progress made on our rebastinib program, including today’s announcement that we have initiated a second Phase 1b/2 clinical study of rebastinib in combination with chemotherapy,” said Michael D. Taylor, Ph.D., President and Chief Executive Officer of Deciphera. “This second clinical study will evaluate rebastinib in combination with carboplatin, a treatment regimen that, based on preclinical observations, we believe has the potential to be an important new approach to treating patients with advanced solid tumors.”
https://www.businesswire.com/news/home/20190103005177/en/Deciphera-Pharmaceuticals-Initiates-Phase-1b2-Clinical-Trial
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Pacira Reports Preliminary 2018 Net EXPAREL® Sales of $331 M

Pacira Pharmaceuticals, Inc. (NASDAQ:PCRX) today reported preliminary unaudited EXPAREL® (bupivacaine liposome injectable suspension) net product sales of $94 million for the fourth quarter of 2018 and $331 million for the full-year, as compared to $79 million and $283 million reported for the fourth quarter and full-year 2017, respectively. The company previously guided to full-year EXPAREL net product sales of $325 to $330 million.
“We are very pleased with our 2018 results, with fourth quarter EXPAREL sales exceeding our expectations and marking the highest quarterly revenues ever reported by Pacira,” said Dave Stack, chairman and chief executive officer of Pacira. “With an expanding number of payers, providers and patients recognizing the value of EXPAREL as a critical component within opioid-sparing pain management strategies, we are entering 2019 with strong momentum. Our robust partnership network also remains a core driver as we continue to focus on maximizing the value of EXPAREL, including its role in transitioning certain inpatient procedures to the ambulatory setting. We are particularly excited by new reimbursement codes, which went into effect on January 1, 2019, that we believe will meaningfully expand patient access through a product-specific C-code for ambulatory surgery centers and a procedure-based D-code for the oral surgery setting.
Looking ahead, we have put in place a number of growth initiatives that include building a pipeline of innovative non-opioid pain management and regenerative health solutions. Our goal is that these, coupled with our unique experience and position in the marketplace as a leader in non-opioid postsurgical pain management, will make a key contribution in confronting the current U.S. pain and opioid crises.”
The financial information included in this press release is preliminary, unaudited and subject to adjustment. It does not present all information necessary for an understanding of the company’s fourth quarter and full-year financial results for 2018. Pacira expects to report its complete financial results for 2018, along with financial guidance for 2019, during the company’s fourth quarter earnings call, which will take place in the first quarter of 2019.
https://globenewswire.com/news-release/2019/01/03/1680035/0/en/Pacira-Reports-Preliminary-2018-Net-EXPAREL-Sales-of-331-Million.html
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First Patient Enrolled in Biogen’s Phase 3b MS Study

The first patient has been enrolled in a global Phase 3b study evaluating the efficacy and safety of extended interval dosing (EID; every six weeks) for natalizumab compared to standard interval dosing (SID) in patients with relapsing multiple sclerosis (MS), Biogen Inc. (Nasdaq: BIIB) announced. Currently commercialized under the brand name TYSABRI®, natalizumab 300 mg dosed every four weeks is the only approved dosing regimen.
The new study, NOVA (NCT03689972), is a two-year, prospective, randomized, interventional, controlled, open-label, rater-blinded, international Phase 3b study that will assess the efficacy, safety and tolerability of six-week natalizumab dosing intervals in people with relapsing-remitting MS. Patients who switch to EID after one year of treatment with natalizumab SID will be evaluated in relation to patients receiving continued SID treatment. The study will enroll approximately 480 patients worldwide. The primary endpoint is the number of new or newly enlarging T2 hyperintense lesions at week 48.
“For more than a decade, natalizumab has been considered a highly effective treatment option for patients with relapsing forms of MS,” said Alfred Sandrock, Jr., M.D., Ph.D., executive vice president and chief medical officer at Biogen. “The NOVA study may generate valuable data that we hope will answer questions for the scientific community about the efficacy of natalizumab when its dosing schedule is extended to every six weeks, and in conjunction with prior safety analyses, may inform on the drug’s benefit-risk profile.”
NOVA was initiated following analyses that showed that EID was associated with a significant reduction in the risk of progressive multifocal leukoencephalopathy (PML), a rare but serious brain infection. The pre-specified, retrospective analysis of the U.S. TOUCH (TYSABRI®Outreach: United Commitment to Health) REMS program examined the impact of EID as compared to SID on the risk of PML, and the NOVA study aims to assess the efficacy of EID natalizumab to further evaluate the drug’s benefit-risk profile.
https://globenewswire.com/news-release/2019/01/03/1680036/0/en/First-Patient-Enrolled-in-Biogen-s-Phase-3b-Study-to-Evaluate-Extended-Interval-Dosing-EID-with-Natalizumab-in-Multiple-Sclerosis.html
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Ascletis’ IND Filing for Its Third HCV Drug ASC21 Accepted by NMPA


Ascletis Pharma Inc.(1672.HK), a commercial-stage biotechnology company addressing unmet medical needs in therapeutic areas including anti-viral, cancer and fatty liver diseases, announces today that the investigational new drug application (IND) for its third HCV drug ASC21 was accepted by the National Medical Products Administration (NMPA).
ASC21 is an NS5B nucleotide polymerase inhibitor that has been shown in in vitro studies to have potent, pan-genotypic anti-viral activity and a high genetic barrier to resistance. ASC21, in combination with Ravidasvir, forms a regimen which has the potential to have pan-genotypic activity and to be effective in treating patients with difficult-to-cure genotypes, cirrhosis and HCV/HIV co-infection.
In June 2018, Ganovo®, Ascletis’ first HCV drug got New Drug Application (NDA) approval and launched successfully. On August 1, 2018, Ascletis’ NDA for its second HCV drug Ravidasvir was accepted and designated as priority review in October by NMPA. With three innovative HCV drugs having differentiation in mechanism of actions, Ascletis is committed to treating hepatitis C in greater China with its multiple leading antiviral combinations.
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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.
  1. 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.
  1. 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.
  1. 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.
  1. 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.
  1. 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.
  1. 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.
  1. 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.
  1. 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.
  1. 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.
  1. 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.
  1. 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.
  1. 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.
  1. 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.
  1. 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.
  1. 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.
  1. 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.
  1. 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.
  1. 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.
  1. 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.
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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.”
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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
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