It is “financial reporting season” for many publicly-traded companies, and the Philadelphia region is no different. But in addition to several quarterly financial reports and business updates, there has been plenty of research, clinical trial and people-on-the-move stories for the week.
Funding, Awards and Collaborations
Soligenix Inks Deal with Silk Road for Pentoxifylline
Princeton, NJ-based Soligenix inked an exclusive option deal with Silk Road Therapeutics for a novel topical formulation of pentoxifylline (PTX) for treatment of mucocutaneous ulcers in patients with Behcet’s Disease. The drug is a non-biological anti-TNF-alpha inhibitor. BD is a rare multisystem inflammatory vasculitis with no cure, marked by recurrent ulcers in the oral mucosa, skin/genitalia, and eye compartment.
Lannett Enters into Restructuring Support Agreement
Lannett Company, based in Trevose, Pa., announced, that along with some of its subsidiaries, it has entered into a Restructuring Support Agreement with holders of more than 80% of its 7.750% senior secured notes due 2026 and 100% of the lenders party to the Company’s second lien credit and guaranty agreement. The proposed financial restructuring transaction will decrease the company’s outstanding secured indebtedness by about $511 million.
Arbutus Q1: Progress on Hepatitis B and Coronavirus Assets
Arbutus Biopharma, headquartered in Warminster, Pa., reported its first-quarter financials and corporate update. During the quarter the company reported data from AB-729, a Hepatitis B virus (HBV)-focused RNAi therapeutic. It also dosed the first patient in a Phase I trial of AB-161, its oral RNA destabilizer. It also expects to launch a Phase I study AB-343 against COVID-19 later this year.
Jefferson Health and Labcorp Ink Strategic Laboratory Relationship
Burlington, NC-based Labcorp and Philadelphia’s Jefferson Health inked a strategic laboratory relationship, which will expand the scope and productivity of specialty lab testing retained by Jefferson. It will also deepen the academic and research efforts of Thomas Jefferson University. They will partner to establish the Innovation Driving Excellence in Academics (IDEA) initiative
Astellas to Buy Iveric Bio
Tokyo-based Astellas Pharma is buying Parsippany, NJ-based Iveric Bio for an equity value of $5.9 million. Iveric focuses on therapeutics for eye diseases.
Incyte Q1: Year-Over-Year Double-Digit Revenue Growth
Wilmington, Del.-based Incyte reported its first quarter 2023 financial results. Total net product revenue for the quarter was 14% Y/Y at $5693 million. The quarter marked the approval of Opzelura (ruxolitinib) cream in Europe for repigmentation of nonsegmental vitiligo.
Harmony Biosciences Q1: Wakiz Net Revenue Increased ~40% Year-Over-Year
Harmony Biosciences, based in Plymouth Meeting, Pa., reported strong first quarter 2023 financials. The company’s Wakix (pitolisant) net revenue increased ~40% Y/Y to $119.1 million in the first quarter, with the average number of patients on the drug increasing ~1,200 Y/Y to ~5,100 for the first quarter. The drug is approved for treatment of excessive daytime sleepiness or cataplexy in adults with narcolepsy.
AmerisourceBergen Q2: Revenue Increased 9.9% Year-Over-Year to $63.5 Billion
AmerisourceBergen (Conshohocken, Pa.) reported its Q2 2023 financials ending March 31, with $63.5 billion in revenue, an increase of 9.9% year-over-year. The company also updated its outlook for fiscal year 2023, raising its adjusted diluted EPS guidance from $11.50 to $11.75 to a new range of $11.70 to $11.90.
West Pharma’s Ready Pack with Corning Valor RTU Vials Receives Best Technologies Award
West Pharmaceutical Services announced its West Ready Pack™ with Corning® Valor® Vials utilizing Stevanato Group’s EZ-fill® won the Best Technologies Award at INTERPHEX. This award recognizes cutting-edge technologies, either new tech or a novel implementation of existing tech, that have the potential to change the way biopharma companies operate.
CHOP-Based Consortium Awards 5 Medical Device Companies
The Pennsylvania Pediatric Medical Device Consortium (PPDC), funded by the FDA and based at Children’s Hospital of Philadelphia (CHOP), awarded seed grants of up to $50,000 each to five medical device companies developing devices for children. The companies are: Disati Medical (Boston, MA), Little Sparrows Technologies (Woburn, MA), Dr. Omer Inan and Dr. Samer Mabrouk of Georgia Institute of Technology, VoluMetrix (Nashville, TN), and William Fox, MD, attending neonatologist in the Division of Neonatology at CHOP.
Lannett Company Subsidiaries File Prepackaged Chapter 11 Cases
Trevose, Pa.-based Lannett Company announced that it and certain of its subsidiaries have filed prepackaged Chapter 11 cases in the U.S. Bankruptcy Court for the District of Delaware. These filings are to assist transactions being considered by the Restructuring Support Agreement the company announced on May 1, 2023.
Ocugen Q1: Focus on Phase I/II Trial for Ophthalmology Diseases
Malvern, Pa.-based Ocugen reported its first-quarter 2023 financials, focusing on preliminary positive efficacy and safety results from its Phase I/II trial of OCU400. The data supports the drug as a first-in-class therapeutic approach to be a “viable gene-agnostic treatment” for retinitis pigmentosa and Leber Congenital Amaurosis.
Avertix to Become Publicly Traded Via Merger with BIOS Acquisition Corp.
Avertis Medical, based in Eatontown, NJ, maker of the first and only FDA-approved implantable heart attack warning system, is being acquired by BIOS Acquisition Corporation, a special purpose acquisition company (SPAC). This will result in Avertix becoming a publicly traded on Nasdaq and trade under the ticker symbol AVRT.
In the Clinic
BMS’s TRANSCEND Trials of Breyanzi Hit Primary Endpoint
Bristol Myers Squibb, with U.S. offices in Princeton, NJ, announced positive topline results from the TRANSCEND FL Phase II trial and the TRANSCEND NHL 001 Phase I trial. TRANSCEND FL is evaluating Breyanzi (lisocabtagene maraleucel) in relapsed or refractory follicular lymphoma. TRANSCEND NHL 001 is evaluating the drug in relapsed or refractory B-cell non-Hodgkin lymphoma, including mantle cell lymphoma (MCL). Both trials hit the primary endpoint of overall response rate (ORR).
Janssen Reports First Results from Phase II Bladder Cancer Study
Janssen Pharmaceutical Companies of Johnson & Johnson announced the first results from the open-label Phase II SunRISe-1 trial of TAR-200 monotherapy and cetrelimab monotherapy in patients with Bacillus Calmette-Guerin (BCG)-unresponsive high-risk non-muscle-invasive bladder cancer, who are ineligible for, or decline, radical cystectomy. The study demonstrated 72.7% of patients receiving TAR-200 alone and 38.1% with cetrelimab alone achieved the primary endpoint of a complete response. Janssen has offices in Raritan, NJ.
New Products
Teleflex Launches New Arrow VPS Rhythm DLX Device and NaviCurve Stylet
Teleflex (Wayne, Pa.) launched two new devices to enhance peripherally inserted central catheter (PICC) insertion procedures and decrease the chance of complications in the U.S. The new Arrow® VPS Rhythm DLX Device and NaviCurve™ Stylet are designed to work together to give Vascular Access Specialists more efficient and predictable PICC placement.
Research Roundup
Penn Research: A New Approach to Gene Editing
Researchers at the Perelman School of Medicine at the University of Pennsylvania published research in which protein fragments used by some viruses to help them get into cells could also be used to get CRISPR-Cas gene editing molecules into cells with greater efficiency and lower cellular toxicity. The research was published in Nature Biotechnology.
Temple Research: Dual Gene-Editing Approach Suggest Possibility of Eliminating HIV
Researchers at Temple University’s Lewis Katz School of Medicine and at the University of Nebraska Medical Center suggest that a gene-editing therapy targeting HIV-1 and CCR5, a co-receptor that helps the virus enter cells, can effectively eliminate HIV-1 infection. The therapy has proven effective in mice and the researchers hope to test it soon in non-human primates.
Wistar Research: Mechanism of Innate Tumor Suppression
Researchers at The Wistar Institute in Philadelphia led by Maureen E. Murphy, PhD, Deputy Director of the Ellen and Ronald Caplan Cancer Center and Ira Brind Professor and program leader of the Molecular & Cellular Oncogenesis Program, published research identifying a key mechanism on how the p53 protein suppresses tumors. The research focused on an African-specific variant of p53 called Y107H.
Regulatory and Advocacy
FDA Accepts Priority Review sBLA for BMS’s Reblozyl
Bristol Myers Squibb, with U.S. offices in Princeton, NJ, announced the FDA had accepted its supplemental BLA and the European Medicines Agency (EMA) had validated the Type II Variation Application for Reblozyl (luspatercept) to expand its current indication to include anemia without previous use of erythropoiesis-stimulating agents in adults with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require RBC transfusions. In the U.S. it was accepted under Priority Review with a PDUFA date of August 28, 2023.
Cabaletta Bio’s Lupus Drug Granted Fast Track Designation
Philadelphia-based Cabaletta Bio received Fast Track Designation from the FDA for CABVA-201, a 4-1BB-containing fully human CD19-CAR T-cell therapy for patients with systemic lupus erythematosus (SLE) and lupus nephritis (LN). The company has been cleared to launch a Phase I/II trial of the drug for six SLE patients with active LN, and a separate parallel cohort in 6 patients with active SLE without renal involvement.
BMS Receives B-Cell Lymphoma Approval for Breyanzi in Europe
Bristol Myers Squibb, with offices in Princeton, NJ, announced the European Commission (EC) approved Breyanzi (lisocabtagene maraleucel) for adults with diffuse large B-cell lymphoma (CLBCL), high grade B-cell lymphoma (HGBCL), primary mediastinal large B-cell lymphoma (PMBCL) and follicular lymphoma grade 3B (FL3B), who relapsed within 12 months from completion of, or are refractory to, first-line chemotherapy. The drug is a CD19-directed CAR T-cell therapy.
People on the Move
Envision Pharma Appoints Tino Quintero at GM/VP of Market Access & Customer Insights
Envision Pharma (Philadelphia, PA) appointed Tino Quintero to the role of General Manager/VP of Market Access & Customer Insights. Quintero’s latest position was as founder of The Q Group. Prior to that, he was Chief Commercial Officer at Locus Biosciences.
Envision Pharma Group Appoints Jennifer Costello as Chief Medical Officer
Philadelphia’s Envision Pharma Group appointed Jennifer Costello, PharmD, as Chief Medical Officer. Her most recent role was leading cardiovascular patient advocacy for Bristol Myers Squibb across its CV portfolio.
Soligenix Appoints Timothy Coté to its Board
Princeton, NJ-based Soligenix appointed Timothy R. Coté, MD, to its Board of Directors. Coté is a leading national regulatory expert in orphan drug development, with 23 years of Federal service at the FDA, NIH and CDC.
