The Daily Biotech Pulse: FDA Nod For Viela, 3 IPOs And Hematology Congress Presentations

Here's a roundup of top developments in the biotech space over the last 24 hours.

Scaling The Peaks

(Biotech Stocks Hitting 52-week Highs June 11)

• ADC Therapeutics SA ADCT
• Applied Molecular Transport Inc AMTI (IPOed June 5)
• Celldex Therapeutics, Inc. CLDX
• Novo Nordisk A/S NVO(announced a deal to buy privately held Corvidia Therapeutics that is developing therapies for cardio-renal diseases for up to $2.1 billion)
• Regeneron Pharmaceuticals Inc REGN(announced commencement of first human trial of REGN-COV2, its investigational dual antibody cocktail for the prevention and treatment of COVID-19)
• Sarepta Therapeutics Inc SRPT
• Zynex Inc. ZYXI (announced addition to the S&P SmallCap 600 Index)

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows June 11)

• Ayala Pharmaceuticals Inc AYLA
• CALLIDITAS THER/S ADR CALT(IPOed June 5)
• Evofem Biosciences Inc EVFM
• Hoth Therapeutics Inc HOTH
• Vaccinex Inc VCNX

Stocks In Focus

Leap Therapeutics's Cancer Drug Gets Orphan Drug Designation

Leap Therapeutics Inc LPTX said the FDA U.S. Food and granted orphan drug designation for its DKN-01 for the treatment of gastric and gastroesophageal junction cancer. The company said DKN-01 is currently being evaluated in Phase 1/2 and Phase 2 clinical trials for gastroesophageal, gynecologic, hepatobiliary, and prostate cancers.

The stock climbed 28.44% to $2.40 in after-hours trading.

Virtual European Hematology Association Congress Presentations

Agios Pharmaceuticals Inc AGIO reported interim data from its ongoing Phase 2 study evaluating single agent mitapivat in non-transfusion-dependent α- and β-thalassemia, which showed it induced hemoglobin increase of 1 g/dl or more in 12 of 13 evaluable patients, including 4 of 4 α-thalassemia patients during Weeks 4-12. The company said 7 of the 8 evaluable patients achieved sustained hemoglobin response during Weeks 12-24.

The company noted that one Grade 3 adverse events of renal dysfunction was reported related to the treatment post cut-off date but was resolved upon treatment discontinuation.

The stock was sliding 9.79% to $45.71 in pre-market trading.

Vertex Pharmaceuticals Incorporated VRTX and Crispr Therapeutics AG CRSP presented at the EHA meeting new clinical data for CTX001, an investigational CRISPR/Cas9 gene-editing therapy, from the CLIMB-111 and CLIMB-121 Phase 1/2 trials in transfusion-dependent beta thalassemia, or TDT, and severe sickle cell disease, or SCD.

Long-term follow-up data from a TDT patient showed at 15 months after CTX001 infusion, the patient was transfusion independent and had appreciable hemoglobin levels. The second patient was transfusion independent at five months.

New data from one SCD patient showed nine months after CTX001 infusion, the patient was free of vaso-occlusive crises, was transfusion independent and had an increase in hemoglobin levels.

Vertex shares were edging up 0.42% to $265 and Crispr was advancing 2.18% to $61 in pre-market trading.

Editas Medicine Inc EDIT announced results from a pre-clinical, proof-of-concept study of EDIT-301 in SCD, with both in-vitro and in-vivo studies revealing several desirable properties.

The stock was rising 4.40% to $26.11 in pre-market trading.

Viela Receives FDA Approval For Neurological Autoimmune Disorder Drug

Viela Bio Inc VIE said the FDA approved its Uplizna for the treatment of adult patients with neuromyelitis optica spectrum disorder, or NMOSD, who are anti-AQP4 antibody positive as a twice-a-year maintenance regimen following initial doses. Approximately 80% of all patients with NMOSD test positive for anti-AQP4 antibodies. The company is expects to launch the drug commercially in June.

In pre-market trading, the stock was gaining 6.55% to $54.82.

Quidel Gets $635,000 BARDA Funding For 4-Virus Point-Of-Care Test

Quidel Corporation QDEL said it has received funding from the Biomedical Advanced Research and Development Authority, or BARDA, to support the development of a point-of-care diagnostic assay that potentially tests for four respiratory viruses: SARS-CoV-2, Influenza A, Influenza B, and Respiratory Syncytial Virus. The respiratory virus panel would be developed to run on Quidel's Sofia 2 flagship instrument.

The company said funding began May 29, 2020, and will run through April 2021, totaling approximately $635,000. The goal of the funding, according to the company, is to achieve an Emergency Use Authorization for the test within the funding period.

FDA Approves Mylan-Biocon's Diabetes Drug

Mylan NV MYL along with its Indian partner Biocon said the FDA approved their Semglee - insulin glargine injection - in vial and pre-filled pen presentations, to control high blood sugar in adults with type 2 diabetes and adult and pediatric patients with type 1 diabetes. Semglee has an identical amino acid sequence to Sanofi SA's SNY Lantus and is approved for the same indications.

Mylan shares were adding 3.37% to $16.85 in pre-market trading.

On The Radar

Clinical Readouts/Presentations

Principia Biopharma is due to present at the American Academy of Dermatology Virtual Meeting Experience, full data set presentation from the Phase 2 Part B pemphigus trial with its investigational drug rilzabrutinib.

Oramed Pharmaceuticals, Inc. ORMP will present at the American Diabetes Association 80th Scientific Sessions posters on oral insulin ORMD-0801's effects on glucose parameters in uncontrolled Type 2 diabetes, oral insulin-induced reduction in liver fat content in Type 2 diabetic patients with non-alcoholic steatohepatitis and evening oral insulin glycemic effects in uncontrolled Type 2 diabetes patients

IPO

Vaxcyte, Inc., a company developing vaccines to prevent or treat the most common and deadly infectious diseases, priced its upsized initial public offering, or IPO, of 15.625 million shares of its common stock at $16 per share. The shares will begin trading on the Nasdaq under the ticker symbol PCVX.

La Jolla, California-based Avidity Biosciences, Inc. priced its upsized IPO of 14.4 million shares at $18 per share compared to the estimated price range of $17-$18. The shares of the biopharma, which develops a new class of oligonucleotide-based therapies called Antibody Oligonucleotide Conjugates to treat a wide range of serious diseases, will be listed on the Nasdaq under the ticker symbol RNA.

Cambridge, Massachusetts-based gene therapy company Generation Bio Co priced its IPO of 10.53 million shares at $19 per share compared to the estimated price range of $18-$19. The company's shares will begin trading on the Nasdaq under the ticker symbol GBIO.