A New Dawn for CRISPR, Intellia Edits Cells Inside the Body

Intellia Therapeutics, along with its partner Regeneron Pharmaceuticals, recently announced its successful attempt in editing the cells inside the body leveraging the CRISPR technique. The world’s first-ever trial that has their DNA edited through an in vivo CRISPR/Cas9 therapy brought good news for everyone as the trials managed to present improvements in a rare inherited liver disorder.

The robust efficacy data was coupled with an encouraging safety profile with no serious adverse events as observed in the first six patients as of day 28. The treatment aims to repair a gene responsible for transthyretin amyloidosis, or ATTR. As per DelveInsight’s ATTR epidemiological analysis, In 2017, the total diagnosed prevalent population in the 7MM was 19,344, which is further expected to rise.

Although, there exists approved medications and drugs such as Onpattro (Patisiran), Tegsedi (Inotersen), and symptomatic therapies such as analgesics for the treatment of transthyretin amyloidosis, and it is expected to act as a hindrance for the firm while vying for the market share. However, gene-editing provides a one-time treatment option meant to offer permanent solutions like halting, or reversing the disease’s downward course, which will surely provide Innovent a leg-up over other pharma players in the market.

Innovent, Synaffix Signs Up an ADC-Tech License Deal

Innovent Biologics has entered into a non-exclusive, target-specific license agreement with Synaffix to leverage its Antibody-drug conjugates (ADCs) technology.

Going by the agreement, Synaffix is eligible for upfront payment, potential milestones payments, along with royalties on the basis of potential future commercial sales of the ADC product in return for all the necessary proprietary ADC technologies including GlycoConnect, HydraSpace, and one of its toxSYN linker-payloads. 

While Innovent will receive the rights to leverage the ADC technology for one therapeutic molecule and will be solely responsible for R&D, development and manufacturing of the product as well as its commercialization, Synaffix will help Innovent with the procurement of the parts and products specific to its ADC technology. 

Eli Lilly’s Tirzepatide Shows Blockbuster Potential in Diabetes 

Eli Lilly’s Tirzepatide continues to steal the attention with its potential in treating Diabetes, outwinning several drugs in clinical trials. 

Lilly’s investigation drug, Tirzepatide, is a dual glucose-dependent insulinotropic polypeptide (GIP) and glucagon-like peptide-1 (GLP-1) receptor agonist, which managed to reduce hemoglobin A1C (HbA1c) levels in people with type 2 Diabetes while supporting weight loss.

It also led to improvements in the change in fasting serum glucose from baseline and improvements in the change in two-hour post-meal glucose values from baseline (SURPASS-1). Based on positive results, the company plans to file for regulatory approval by the end of this year. 

The company is also investigating the drug in non-alcoholic steatohepatitis (NASH) and heart failure with preserved ejection fraction (HFpEF).

Polyphor’s Drug Flops in Breast Cancer, Puts the Firm in Tight Spot 

Swiss pharmaceutical company, Polyphor, saw its share price crashing after its therapy intended to cure Breast cancer failed. 

The announcement follows the results from a global phase III trial (FORTRESS) that evaluated balixafortide (POL6326) and eribulin to treat patients who have HER2 negative, locally recurrent, or metastatic breast cancer. The cocktail therapy failed to produce any such significant difference in the objective response rate (ORR) compared to using just eribulin, even after six months of observations.

The unexpected failure has very much jeopardized the existence of the company, which was solely relying on its cancer therapy for potential future revenue. Although the firm owns a novel class antibiotic that targets lung infections, the sales projections have been poor. Polyphor now plans to further discuss with experts the due course of the drug after the failure of FORTRESS.