Allogene Therapeutics Inc. made known its aim to find a place in first-line treatment of large B-cell lymphoma (LBCL) with CAR T cemacabtagene ansegedleucel (cema-cel, previously known as ALLO-501A). The hope is that cema-cel will leapfrog other CAR Ts and take lead position in LBCL. Pivotal Alpha2 and Expand trials in third-line, relapsed/refractory LBCL have been deprioritized. “Overall, Allogene today reset the CD19 program in liquid tumors by reprioritizing resources to first-line consolidation in LBCL and chronic lymphocytic leukemia,” J.P. Morgan analyst Brian Cheng said a skeptical-sounding report. Shares of South San Francisco-based Allogene (NASDAQ:ALLO) were trading midday at $2.86, down 53 cents, or 15%.

Applied Therapeutics falls on phase III miss in diabetic cardiomyopathy

Shares of Applied Therapeutics Inc. (NASDAQ:APLT) were trading down 36% Jan. 5 on news that its oral aldose reductase inhibitor, AT-001 (caficrestat), failed to reach statistical significance in a phase III study in patients with diabetic cardiomyopathy. The New York-based firm, however, pointed to positive trends in the Arise-HF study that could offer a path forward, albeit via a partner.

Rhythm to acquire LG Chem’s rare genetic obesity drug for $305M

Rhythm Pharmaceuticals Inc. is picking up LG Chem Ltd.’s oral small-molecule drug, LB-54640, for potentially $305 million, effectively growing its MC4R pipeline that houses U.S. FDA-approved Imcivree (setmelanotide) for genetic obesity disorders. Under the terms of the global licensing deal inked on Jan. 4, Boston-based Rhythm agreed to pay up front $40 million in cash and $20 million in Rhythm equity to Seoul, South Korea-based LG Chem, along with an additional $40 million in cash 18 months after closing. For achieving certain regulatory and sales milestones, LG Chem is eligible for up to an additional $205 million, along with royalties.

Newco news: Perpetual aims to be peptide powerhouse

The field of peptides is exploding, Perpetual Medicines Corp. co-founder, chairman and CEO Kerry L. Blanchard recently told BioWorld, “with a projected growth rate far surpassing large and small molecules, and gene therapies. The area is underinvested, too, so this is a good opportunity to focus on peptide therapeutics.”

Newco news: Kynexis takes run at kynurenine pathway for psychiatry’s cognitive problems

Kynexis BV recently launched with a series A of €57 million (US$62 million) and a lead asset, KYN-5356, that targets the kynurenine pathway. The company is preparing for clinical trials that will test the compound for the treatment of cognitive impairment associated with schizophrenia.

Biopharma financings end 2023 up from last year, with $70.97B raised

The biopharma industry concluded 2023 on a positive note regarding financings, with an increase of 17% in value over last year. A total of $70.97 billion was raised from financings in 2023, up from $60.81 billion raised in 2022. Value is down, however, from $118.29 billion in 2021 and $134.53 billion in 2020, but is higher than all previous years tracked by BioWorld from 2011-2019. In December, $4.18 billion was raised through 928 transactions in December, down 27% from the $5.74 billion tracked by BioWorld in November.

Eisai wearable enables early Alzheimer’s disease screening

Eisai Co. Ltd. and Oita University in Oita Prefecture, Japan, developed a first-of-its-kind machine learning (ML) model to predict amyloid beta accumulation in the brain using a wristband sensor. The model, which collects biological and lifestyle data from daily life, is expected to enable screening for brain amyloid beta accumulation to identify those at risk for Alzheimer's disease (AD), particularly because amyloid beta begins to accumulate in the brain about 20 years before the onset of the disease. The approval of new drugs like Eisai/Biogen Inc.’s Leqembi (lecanemab), which targets amyloid beta, is triggering massive efforts to improve screening measures to identify early AD.

In 2023, big projects create ‘satellite maps’ of cell biology

If we unraveled the DNA of the 46 chromosomes of a single human cell, it would barely measure 2 meters. If we did the same with the rest of the body, if we aligned the 3 billion base pairs of its 5 trillion cells, we could travel the distance from the Earth to the Sun more than 100 times. It seems unreachable. However, that is the unit of knowledge of the large sequencing projects achieved in 2023.

BioWorld highlights the top biopharma trends of 2023

As industry gears up for the annual J.P. Morgan Healthcare conference to kick off 2024, take a moment to review the successes and challenges that defined 2023. It was a year when biopharma faced a tough capital market, as it settled into the new post-pandemic normal, and a year when drug pricing debates went global. But the industry continued to innovate, as always, celebrating advances in gene editing and introducing the world’s first two RSV vaccines. And for companies working on GLP-1 drugs or antibody-drug conjugates, 2023 was a very, very good year.

Also in the news

Aditxt, Alimera, Allovir, Apollomics, Astrazeneca, Avenue, Biomx, C4, Catalyst, Compass Pathways, Corteria, Debiopharm, Dyne, Emergex, Evexta, Genascence, Glycomimetics, GPN Vaccines, Immutep, Inflarx, Innate, Insilico, Intellia, Jiangsu Vcare, Kymera, Kynexis, Kyverna, Lumos, Lyndra, Medicus, Medivir, Menarini, Mineralys, Nimbus, Nurexone, Ocuphire, Omniose, Polypid, Praxis, Quanta, Repare, Rhythm, Sail, Sensorion, Sitryx, Soligenix, Sosei, Tango, Telix, Tenacia, Tryp, Ultragenyx, Upstream, Verrica, Vico, Voyager, Werewolf, Xencor