Weekly Quick Hits (Philly) – Week of August 7, 2023

FDA approves Janssen’s Talvey for r/r Multiple Myeloma, Bristol Myers Squibb buys back $4 billion in shares, and plenty of Q2 reports. Read on for more news.

By Mark Terry

August 11, 2023

Quick Hits is BioBuzz’s weekly round up of all the life science news you don’t want to miss, in your region of interest. Don’t miss a beat; SUBSCRIBE to our weekly Quick Hits newsletter via LinkedIn (BioHealth Capital Region, Greater Philadelphia, Research Triangle Park) or get it delivered to your inbox every week.

Funding, Awards and Collaborations 

Tabula Rasa HealthCare Q2: Revenues Increased 24% to $90.0 Million

Moorestown, NJ-based Tabula Rasa HealthCare reported its second-quarter financials, including revenue of $90.0 million, an increase of 24%. This included medication revenue growth of 25% and technology-enabled solutions revenue growth of 22%.

NRx Licenses Patent to Support NRX-101 for Chronic Pain

NRx Pharmaceuticals (Radnor, Pa.) inked a License Agreement for U.S. Patent 8,653,120 for using D-cycloserine to treat chronic pain in exchange for a commitment to pay milestones and royalties. The patent is based on extensive nonclinical data and early clinical data.

Passage Bio Q2: Reports Several Clinical Milestones for GM1 Gangliosidosis

Philadelphia-based Passage Bio reported its second-quarter financials and business highlights. It reported promising interim data from Cohorts 1-4 in the Imagine-1 trial for PBGM01 for GM1 gangliosidosis, that Dose 2 of the drug hit healthy control levels of CSF Beta-Gal activity and GM1 gangliosides, and treated the first patient at Dose 3 in the trial. The company also reported $151.5 million in cash as of June 30.

Madrigal Q2: Recently Submitted NDA for NDA for NASH

Madrigal Pharmaceuticals (Conshohocken, Pa.) reported its second-quarter financial results and corporate updates, focusing on its recent submission of a New Drug Application (NDA) to the FDA for resmetirom to treat non-alcoholic steatohepatitis (NASH). In addition to the submission, the company is gearing up for a “potential first-to-market launch in the U.S.” The company reported $298.4 million in cash as of June 30.

Neuronetics Q2: Quarterly Revenue Increased 8% to $17.6 Million

Malvern, Pa.-based Neuronetics reported its financial and operating results for the second quarter. Its quarterly revenue increased 8% to $17.6 million, with U.S. treatment session revenue increasing 9%. International revenue increased by 63%, driven largely to an increase in NeuroStar Advanced Therapy system sales and treatment session revenue.

Verrica Q2: FDA Approved Ycanth for Molluscum, Launch Expected in September

Verrica Pharmaceuticals (West Chester, Pa.) reported its second-quarter financials, focusing on the July FDA approval of Ycanth for the treatment of molluscum, a highly contagious viral skin infection. The company also expanded Part 2 enrollment of a Phase II trial of VP-315 in basal cell carcinoma. It has secured a $125 million debt facility to support the September launch of Ycanth.

BMS Inducted into the Billion Dollar Roundtable

Bristol Myers Squibb was inducted into the Billion Dollar Roundtable, joining other Fortune 100 companies that have invested $1 billion into diverse-owned suppliers. BMS created aspirational inclusion and diversity goals and health equity commitments in 2020 to address health disparities by 2025 and spent $1 billion with diverse and minority-owned businesses.

Larimar Q2: Cleared for 50mg Cohort in Phase II Friedreich’s Ataxia Trial

Balal Cynwyd, Pa.-based Larimar Therapeutics reported its second-quarter financials and business update. The company was cleared by the FDA to initiate a 50mg cohort in its Phase II Friedreich’s ataxia dose exploration trial after the agency reviewed the unblinded 25mg cohort from the study. The company reported $104.2 million in cash as of June 30.

Temple Cannabis Researchers Partner with Cresco Labs

Temple University’s Center for Substance Abuse Research (CSAR) partnered with medical cannabis producer Cresco Labs. The university’s CSAR was founded in 1998 and is expanding its research into how cannabis can be used to treat chronic pain, food allergies, and other conditions.

In the Clinic

Helius Announces Neurology Center of New England Joining its Therapeutic Experience Program

Newtown, Pa.-based Helius Medical Technologies announced the addition of Neurology Center of New England to its Therapeutic Experience Program (TEP). The Centers joins four other Centers of Excellence in this company-sponsored open-label observational trial to study subjects’ adherence to Portable Neuromodulation Stimulator (PoNS) therapy in MS patients.

Marinus Q2: Ztalmy Product Revenue Hit $4.2 Million, Increased Guidance for Year

Radnor, Pa.-based Marinus Pharmaceuticals reported business highlights and financial data for the second quarter. Of note, Ztalmy (ganaxolone) net product revenues for the quarter were $4.2 million. This prompted increasing the 2023 net product revenue guidance to between $17 and $18.5 million. The European Commission also approved the drug in CDKL5 deficiency disorder.

BMS Enters into $4 Billion Accelerated Share Repurchase Agreements

Bristol Myers Squibb (Princeton, NJ) entered into accelerated share repurchase (ASR) deals under agreements with each of Bank of America, N.A., Citibank, N.A., JPMorgan Chase Bank, N.A. and Morgan Stanley & Co., LLC to repurchase, in aggregate, $4 billion of BMS common stock. After the deal, the company will have about $2 billion of remaining share repurchase authorization.

Soligenix Opens Enrollment in Cutaneous T-Cell Lymphoma Trial

Princeton, NJ-based Soligenix opened patient enrollment for the investigator-initiated trial at the University of Pennsylvania, supported by a $2.6 million Orphan Products Development grant from the FDA. The study will evaluate the expanded treatment, including up to 12 months of treatment, with HyBryte (synthetic hypericin) in patients with early-stage cutaneous T-cell lymphoma.

Carisma Q2: Dosed 1^st Patient in Phase I HER2-Overexpressing Solid Tumor Trial

Carisma Therapeutics (Philadelphia) reported its second-quarter financial results and business highlights. At the top, the company dosed the first patient in the Phase I trial of CT-0508 in combination with Merck’s Keytruda (pembrolizumab) in patients with HER2-overexpressing solid tumors. It also added an additional oncology target as part of its collaboration with Moderna to develop in-vivo targeted CAR-M therapies. The company has $117.1 million in cash, enough to fund itself through 2024.

Palatin Launches Clinical Program for Erectile Dysfunction Patients

Cranbury, NJ-based Palatin Technologies initiated a clinical development program studying bremelanotide, a MCR4 agonist, co-formulated with a PD35 inhibitor for the treatment of erectile dysfunction in patients that don’t respond to PDE5i monotherapy. The trial is expected to launch in the fourth quarter.

Agile Q2: Twirla Revenue Increases 44% to $5.5 Million

Agile Therapeutics (Princeton, NJ) reported its second-quarter revenue, including Twirla net revenue of $5.5 million, an increase of 44% from the first quarter of this year. The company’s gross margin grew 58% for the quarter from 47% in the first quarter.

PMV Q2: Progress in Ongoing Phase I/II PYNNACLE Solid Tumor Study

Princeton, NJ-based PMV Pharmaceuticals reported its second-quarter financial and corporate highlights. The company reported continued progress in its ongoing Phase I/II PYNNACLE trial of PC14586, a first-in-class precision oncology therapy in advanced solid tumors with a p53 Y22OC mutation. The company indicated $218.8 million in cash as of June 30, 2022.

Research Roundup

Penn Research: Influenza Shows No Seasonality in Tropics

Public health officials have assumed that influenza is seasonal in tropical climates, the way it is in places like North America and Europe. However, new research out of Penn State evaluating influenza in Vietnam found very little repeatable patterns related to the seasons.

Imvax Published Data Supporting IGV-001 in Glioblastoma

Philadelphia-based Imvax published preclinical research with IGV-001 in the Journal for ImmunoTherapy of Cancer. The data supported the use of the drug in glioblastoma. It is currently being studied in a Phase IIb trial in combination with standard of care for newly diagnosed glioblastoma.

Penn Research: Brain Mechanisms That Helps Focus Despite Distractions

Neuroscientists in the Perelman School of Medicine at the University of Pennsylvania published research that identified “visual-movement” neurons in the front of the brain that help people stay focused despite distractions. They discovered a pattern of coordinated activity called “beta bursts” in a set of neurons in the lateral prefrontal cortex (LPFC).

CHOP Research: COVID-19 Causes Mitochondrial Dysfunction in Heart and Other Organs

A multi-institutional consortium of research led by a team at Children’s Hospital of Philadelphia (CHOP) and the COVID-19 International Research Team (COV-IRT) found that mitochondrial genes can be negatively impacted by SARS-CoV-2, the virus that causes COVID-19. This can lead to dysfunction in multiple organs beyond the lung.

Penn Research: Catching Up on Sleep May Not Improve CV Cost of Sleep Loss

Researchers at the University of Pennsylvania found that cardiovascular health measures, including heart rate and blood pressure, get worse over the course of the week when people get five hours or less sleep per night. And worse, attempting to catch up on sleep during the weekend isn’t enough to normalize those CV risks.

On the Hill – Regulatory and Advocacy

FDA Approves Janssen’s Talvey for Multiple Myeloma

The FDA granted The Janssen Pharmaceutical Companies of Johnson & Johnson’s Talvey (talquetamab-tgvs) accelerated approval for adults with relapsed or refractory multiple myeloma who have received at least four previous lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody. The drug is a first-in-class bispecific antibody.

People on the Move

The Wistar Institute Brings on Virologist Alexander Price

The Wistar Institute (Philadelphia) recruited Alexander Price, Ph.D., as an assistant professor in the institute’s Gene Expression and Regulation Program of the Ellen and Ronald Caplan Cancer Center. Price’s research targets how viral genomes are controlled during infection, and how viruses regulate and exploit RNA transcription and processing.