Ionis, AstraZeneca going to FDA with PhIII data in hand; Glaukos touts double PhIII win in eye implant
Ionis and AstraZeneca are hoping its latest results net an FDA approval.
The two companies presented their latest Phase III results from a 35-week analysis of the Phase III trial, dubbed NEURO-TTRansform, of AstraZeneca’s eplontersen in patients with the rare disease known as hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
According to Ionis on Wednesday, Phase III had a positive outlook as eplontersen achieved an 81.2% reduction of serum transthyretin (TTR) concentration in patients compared to baseline, demonstrating reduced TTR protein production. This hit the co-primary endpoint of the trial and earned a p-value of p=<0.0001.
Eplontersen also showed a treatment effect on the other primary endpoint, netting a modified Neuropathy Impairment Score of +7 (mNIS+7). This is used to determine neuropathic disease progression. This co-primary endpoint also received a p-value of p=<0.0001.
According to Ionis, the study also hit the secondary endpoint, which was a change from baseline in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy.
“The promising results from NEURO-TTRansform show that eplontersen had a positive impact on disease progression and improved quality of life in a substantial number of patients,” Ionis chief clinical development officer Eugene Schneider said in a statement.
As a result of the data, Ionis and AstraZeneca will look to get regulatory approval for eplontersen to treat ATTRv-PN and are planning to file an NDA later this year.
The drug is being jointly developed and commercialized by both companies in the US and will be developed and commercialized in the rest of the world by AstraZeneca.
That agreement, which kicked off last year, saw Ionis get a $200 million upfront payment, along with receiving up to $485 million in development and approval milestones, as well as up to $2.9 billion in sales-related milestone payments. — Tyler Patchen
Glaukos churns out positive Phase III data in eye trials
Eye disease biotech Glaukos is touting new Phase III data — and with two trials meeting primary endpoints, the biotech is wanting to go get the FDA to sign off on its product.
The company revealed Phase III data Wednesday morning from two pivotal trials, testing iDose TR, an implant with a formulation of travoprost, a medication used to treat ocular pressure. The data, in short, showed that multiple versions of iDose TR reached the primary endpoint of non-inferiority to the comparator arm at three months in.
In terms of reductions in intraocular pressure (also called IOP), the reduction from baseline over the first three months was 6.6-8.5 mmHg (millimeters of mercury, a type of pressure measurement) in the slow-release arm in one trial, versus 6.6-7.7 mmHg in the control arm. In another trial, IOP reductions from baseline over the first three months were 6.7-8.4 mmHg in the slow-release arm, versus 6.8-7.2 mmHg in control.
Shares of $GKOS shot up 15% in early morning trading.
With 98% of patients continuing at 12 months, plus no adverse events of corneal endothelial cell loss, no serious corneal adverse events and no adverse events of periorbital fat atrophy, the biotech plans to submit an NDA — and expects an FDA review and decision by the end of 2023. — Paul Schloesser
Carmell says it’s filed for an IPO
Pittsburgh-based Carmell Therapeutics said it confidentially filed its draft papers with the SEC en route to a public listing.
The SEC first has to complete the review process before a listing can take place.
The biotech, which claims to be on the regenerative medicine track, is working on so-called growth factors in “allogeneic platelet-enriched plasma” to heal bone, skin and other tissues. The goal is to ramp up the regrowth of hair and spark collagen production after an injury, disease or the much-talked-about aging.
Carmell eyes Phase II studies in tibia fracture healing, for which it has fast track designation from the FDA, in the second half of 2023, at which time the biotech also hopes to begin a mid-stage trial in foot/ankle fusion. Preclinical work is being conducted in spinal fusion, dental bone graft substitute, androgenetic alopecia and chronic wound healing, according to Carmell’s pipeline page. The company’s first clinical trial — HEAL I — was conducted in South Africa.
Founded in 2008 by scientists at Carnegie Mellon University, with the help of Pittsburgh Life Sciences Greenhouse, Carmell is led by former medical device leaders from Johnson & Johnson and its subsidiaries. — Kyle LaHucik