The Week Ahead In Biotech (Aug. 24-29): Lipocine Binary Event, Liver Congress In The Spotlight

Editor's note: This story has been updated with the correct PDUFA date for Lipocine Inc. 

After seeing some strength earlier on, biotech stocks came under pressure in the week ended Aug. 21. With the earnings season tapering and not much of noise on the coronavirus front, the sector reacted to some clinical readouts, two M&A announcements and a couple of adverse rulings handed down by the FDA.

Three biotech issues IPOed this week, collecting a cumulative $404.1 in gross proceeds. This apart, Israeli medical imaging startup Nano-X Imaging Ltd NNOX raised $165.2 million.

Here're the key catalysts for the unfolding week:


  • Virtual World Orphan Drug Congress USA 2020: Aug. 24-27
  • European Association for the Study of the Liver, or EASL, Digital International Liver Congress 2020: Aug. 27-29
  • European Society of Cardiology, ESC, Congress 2020: Aug. 29 – Sept. 1
  • 46th Annual Meeting of the European Society for Blood and Marrow Transplantation: Aug. 29 – Sept. 1


Lipocine Inc LPCN is knocking at FDA's doors for the fourth time with respect to its Tlando, an oral prodrug of testosterone containing testosterone undecanoate that is designed to help restore normal testosterone levels in hypogonadal men. The FDA has set a PDUFA action date of Aug. 28 (Friday).

Clinical Readouts

Odonate Therapeutics Inc ODT announced it will hold a conference call on Monday at 8:30 a.m. ET. Although the company has not given details, it is scheduled to report top-line results from the CONTESSA Phase 3 study investigating tesetaxel as a potential treatment for patients with metastatic breast cancer in the third quarter.

See also: Why This 9 Meters Biopharma Analyst Sees 800% Upside Ahead

EASL Presentations

Assembly Biosciences Inc ASMB: additional interim analyses from Study 211, a Phase 2 open-label extension study of ABI-H0731 in combination with standard of care therapy for hepatitis B virus, and data from multiple dose cohorts in the Phase 1b clinical of ABI-H2158-containing regimens for chronic hepatic B virus infection

Mirum Pharmaceuticals Inc MIRM: Phase 2 open-label data on five-year transplant-free survival for pediatric patients with progressive familial intrahepatic cholestasis type 2

CymaBay Therapeutics Inc CBAY: final results on durability of treatment response after 1 year of therapy from an international phase 2 study of seladelpar in patients with primary biliary cholangitis, or PBC

Adaptimmune Therapeutics PLC – ADR ADAP: Updated data from the third dose cohort of an ongoing Phase 1 study of ADP A2AFP SPEAR T-cells in hepatocellular carcinoma

Eiger Biopharmaceuticals Inc EIGR: Phase 2 LIFT study end-of-treatment data for peginterferon Lambda in hepatitis delta virus

Arrowhead Pharmaceuticals Inc ARWR: an oral presentation showing short-term treatment with RNA interference therapy, JNJ-3989 (Friday)

Viking Therapeutics Inc VKTX: new data from the Phase 2 study of VK2809 in patients with non-alcoholic fatty liver disease, or NAFLD, and elevated LDL-cholesterol

Axcella Health Inc AXLA: key data from AXA1125-003, assessing the impact of AXA1125 and AXA1957 in NAFLD

Intercept Pharmaceuticals Inc ICPT: new interim analysis of data from the Phase 3 REGENERATE study of obeticholic acid in non-alcoholic steatohepatitis, or NASH, and additional long-term data of OCA through six years of open-label treatment in PBC

Alnylam Pharmaceuticals, Inc. ALNY and Vir Biotechnology Inc VIR: results from an ongoing Phase 2 study of VIR-2218 in patients with chronic hepatitis B virus

NGM Biopharmaceuticals Inc NGM: Positive topline results from a 24-week, Phase 2 paired liver biopsy study of the FGF19 analogue aldafermin, codenamed NGM282, in patients with NASH

Enanta Pharmaceuticals Inc ENTA: oral presentation of data from the Phase 2a ARGON-1 study of DP-305 in NASH (Friday), and final results of a Phase 1 study of EDP-514 in hepatitis B virus infection (Saturday)

ESC Presentations

Akcea Therapeutics Inc AKCA and Ionis Pharmaceuticals Inc IONS will present data from the Phase 2 study of AKCEA-APOCIII-LRx in a late-breaking clinical trial session. AKCEA-APOCIII-LRx is an antisense drug developed using Ionis' proprietary ligand conjugated antisense technology platform and is designed to reduce the production of apolipoprotein C-III, a protein produced in the liver that plays a central role in the regulation of serum triglycerides.

Myokardia Inc MYOK will present 38-week data from its pivotal Phase 3 EXPLORER-HCM study that is evaluating the efficacy and safety of mavacamten in adults with symptomatic obstructive hypertrophic cardiomyopathyclinical during a late-breaker session.

Arrowhead Pharma is scheduled to make oral presentations on RNA Interference Targeting Apolipoprotein C-3 with ARO-APOC3 in inherited apolipoprotein C-3 deficiency and RNAi Inhibition of Angiopoietin-like Protein 3 with ARO-ANG3 in familial combined hypolipidemia.

EBMT Presentations

bluebird bio Inc BLUE is scheduled to do multiple presentations at the conference; new Phase 3 study of its investigational elivaldogene autotemcel - eli-cel, Lenti-D gene therapy - in patients with cerebral adrenoleukodystrophy and encore presentations from its cerebral adrenoleukodystrophy, sickle cell disease, transfusion-dependent β-thalassemia and multiple myeloma programs


  • Medtronic PLC MDT (Tuesday, after the close)
  • Edap Tms SA EDAP (Wednesday, after the close)
  • Ascendis Pharma A/S ASND (Thursday, after the close)
  • IPO Quiet Period Expiry

Allovir Inc ALVR

Related Link: Novavax's Coronavirus Vaccine Candidate Protects Against Viral Replication In Animal Studies

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