More than 30 years after entering the scene as a first-generation monoclonal antibody pioneer, Morphosys AG is to be acquired by Novartis AG for €2.7 billion (US$2.9 billion). The all-cash deal, announced after Nasdaq closed on Feb. 5, will see Novartis paying €68 per share, a premium of 94% to the average daily price in the month leading up Jan. 25, when rumors of a takeover started swirling. The key asset joining the Novartis portfolio is pelabresib, a small molecule that completed a pivotal myelofibrosis phase III trial in November 2023 and which Morphosys is preparing to file with U.S. FDA and EMA in the first half of this year.

Vertex feels no pain in new, head-to-head CF data

Vertex Pharmaceuticals Inc. upped its own ante with pivotal phase III data in its own strong suit, cystic fibrosis. Taking on Trikafta, the company’s bestseller, Vertex’s once-daily vanzacaftor/tezacaftor/deutivacaftor (vanza) combo hit all primary and key secondary endpoints in two randomized controlled studies of those ages 12 and older. In the Skyline 102 and 103 studies, the cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen was shown to be, respectively, noninferior to Trikafta (p<0.0001 and p<0.0001). In the second and third key secondary endpoints, taken from the two Skyline studies, vanza was superior in the proportion of patients below 60 mmol/L, CF’s diagnostic threshold, and below 30 mmol/L, the carrier level, compared to Trikafta. Approved by the U.S. FDA in 2019, Trikafta (elexacaftor/ivacaftor/tezacaftor) was the first triple combo therapy for treating cystic fibrosis patients 12 and older who have at least one F508del mutation in the CFTR gene. The strong program results quickly followed Jan. 30’s mostly positive phase III data of its non-opioid selective NaV1.8 inhibitor for treating moderate to severe acute pain.

Ultragenyx’s gene therapy UX-111 significant for Sanfilippo

A disease-modifying gene therapy for Sanfilippo syndrome type A has demonstrated reductions in heparin sulfate within cerebrospinal fluid, as well as increases in the cognitive function of young patients, arming Ultragenyx Pharmaceutical Inc. with data needed to support an accelerated BLA with the U.S. FDA. UX-111, which Novato, Calif.-based Ultragenyx licensed from Abeona Therapeutics Inc. in 2022, is a one-time intravenous infusion that uses the AAV9 vector to deliver a functional copy of the SGSH gene to central nervous system and peripheral organ cells in order to break down accumulated heparin sulfate that at high levels becomes toxic, leading to developmental decline in children. “Seventy percent of them will not survive beyond age 18, so this is a fatal neurodegenerative disorder in childhood. Our families will often call it a childhood dementia,” Heather Lau, Ultragenyx’s executive director of global clinical development and an expert in the field, told BioWorld. “There’s no treatment right now. It’s only symptomatic support.”

Curacle’s top-line phase IIa data positive for diabetic macular edema drug

Korean bioventure Curacle Co. Ltd. reported positive top-line findings from its U.S.-based phase IIa study of CU06-1004, an oral drug for diabetic macular edema (DME), spurring plans for a bigger phase IIb study in the second half of 2024. CU06-1004 is touted as the potential world’s first oral endothelial dysfunction blocker for DME, developed with Curacle’s Solvadys platform technology to inhibit damage, hyperpermeability and inflammation of endothelial cells. CU06-1004 could expand into indications including wet age-related macular degeneration and diabetic retinopathy, according to Curacle.

Immunogen leads as BCI’s top performer in 2023 as index ends year up 76%

The BioWorld Cancer Index (BCI) had a stellar performance in 2023, finishing the year with an increase of 76.26%. The rise is a notable spike from the end of the third quarter, when it was up 22.96%. BCI’s performance surpassed both the Nasdaq Biotechnology Index and the Dow Jones Industrial Average, and stands in contrast to 2022, when the BCI concluded the year with a decline of 41.98% and 2021, when the index saw a decrease of 36.34% over the year.

Sangamo’s early phase I/II Fabry Staar data shine but funds needed for late-stage advancement

Sangamo Therapeutics Inc.’s gene therapy for Fabry disease, isaralgagene civaparvovec (ST-920), is continuing to show promising efficacy and safety in the phase I/II study, Staar, but further ahead, the company needs to either attract a partner or secure financing to move to a registrational trial.

US FDA foreign drug inspections a casualty of COVID-19

When the House Energy and Commerce’s Subcommittee on Oversight and Investigations held its last hearing, in December 2019, on the U.S. FDA’s foreign drug inspection program, there were “reasons for cautious optimism,” subcommittee Chair Morgan Griffith (R-Va.) said in opening today’s hearing on that issue. But in the wake of the pandemic, the optimism is gone. Although a large portion of U.S. drugs and active pharmaceutical ingredients come from China and India, the FDA currently has only one investigator based in China and five in India. Since the FDA launched an unannounced inspection pilot program in the two countries in March 2022, it has conducted 35 unannounced and short-term inspections in India. “China, on the other hand, is a completely different story,” Griffith said, as the agency has been unable to conduct even one unannounced inspection in that country. And several times when it’s tried, its inspectors have been refused entry.

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