• US FDA requested Botanix to change its instructions paper
  • Antisense says ATL1102 could be effective in muscular dystrophy
  • Actinogen changes Phase 2b design to reduce cost and time to initial results


Botanix plunges after receiving FDA feedback

Cannabis focused biotech Botanix Pharma (ASX:BOT) plunged 25% this morning after receiving a Complete Response Letter from the US FDA in relation to its NDA submission for Sofpironium Bromide gel, 15%.

The FDA has requested changes to the product use instructions – a paper inserted in the product carton that tells the patient how to safely and effectively use it.

However, no clinical efficacy, safety, pharmacology, non-clinical or manufacturing issues were raised by the FDA, and no additional clinical studies are required to support approval.

Botanix will now meet with the FDA and undertake the relatively minor activities required to address the product use instructions, and resubmit the NDA by the first quarter of 2024 with a target approval of mid-year.

Based on the letter and interactions with the regulator, the company believes no new review issues are anticipated in the resubmission review.

While disappointed, Botanix executive chairman, Vince Ippolito, says the company is now clear on what is required, and it is a priority to work with the FDA to address their comments.

“No safety, efficacy, or manufacturing issues were raised by FDA and no new clinical trials are required – so this gives us a great confidence in our initial submission, data and future resubmission,” Ippolito said.

Botanix’s Sofpironium Bromide gel, 15%, is used for the treatment of primary axillary hyperhidrosis.


Antisense’s drug is effective in muscular dystrophy

Antisense Therapeutics (ASX:ANP) has shared new clinical data from a research collaboration investigating ATL1102 in the treatment of limb girdle muscular dystrophy R2 (LGMDR2).

Results show that investigational drug ATL1102 can reduce inflammation and fat in Duchenne muscular dystrophy by targeting CD49d, a protein involved in the activity of the immune system.

In a mouse model of LGMDR2, treatment with ATL1102 partially normalised the function and physiology of the calf muscles to those of healthy animals.

LGMDR2, also known as dysferlinopathy, is a rare genetic condition leading to a reduction or absence of the protein dysferlin in muscle fibres. Patients with LGMDR2 experience progressive muscle wasting, as well as inflammation, fibrosis, and fat accumulation in muscle.

ATL1102 is currently the subject of an ongoing international Phase IIb clinical trial, with data anticipated in the second half of 2024.


Actinogen changes trial design to expedite results

Actinogen Medical (ASX:ACW) has announced changes to the design of the XanaMIA Phase 2b trial in patients with mild-to-moderate Alzheimer’s disease – in order to reduce the cost and time to initial results.

Initially, the FDA-approved three-arm trial was designed to explore two dose levels, in what is called ‘dose-ranging’.

Actinogen says the new design will keep the same endpoints, 36-week treatment length and other features, with the exception of dose-ranging.

The company says removal of the 5mg dose group – while deferring the dose-ranging aspect of the Xanamem program to the Phase 3 trial – does not detract from the strong confirmatory value of the trial design.

Changes in the new trial design include: 10mg or placebo daily versus 10mg, 5mg or placebo previously; and 220 patients vs 330 patients previously.


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