RNAi biotech Atalanta debuts with $110m plus Biogen and Roche CNS tie-ups

US biotech Atalanta Therapeutics has come out of stealth mode backed with $110m from Biogen and Roche, who have also signed separate partnerships to develop new therapies for neurological diseases using RNA interference (RNAi) technology.

While there are RNAi products on the marketplace, with Alnylam becoming the first company to get a product approved in 2018, it is difficult to get this class of drug distributed through the brain and spinal cord.

But with new technology based on branched silencing RNA licensed from the University of Massachusetts Medical School, Atalanta says it could change this.

Co-founded by Craig Mello, who won the Nobel Prize for Physiology or Medicine in 2006 for his work on RNAi, the company says the technology could be used to treat central nervous system diseases such as Huntington’s, Alzheimer’s and Parkinson’s.

Atalanta has actually been working since 2018 with co-founders Anastasia Khvorova, a UMass biochemist and Neil Aronin, a professor from University of Massachusetts Medical School, providing early leadership.

The company hired Alicia Secor as CEO in summer of 2019, who has previously been CEO of Juniper Pharmaceuticals and serves on the boards of neurology and rare diseases firms GW Pharmaceuticals and Orchard Therapeutics.

Atalanta starts life with two different strategic collaborations with its big pharma backers.

The partnership with Biogen will develop RNAi therapeutics for multiple targets, including HTT for the treatment of Huntington’s disease, as well as additional unnamed CNS targets.

Atalanta will be eligible to receive development and milestone payments as well as undisclosed royalties on any resulting products.

The strategic collaboration with Genentech entails the development of RNAi therapeutics for multiple CNS targets for neurodegenerative diseases, including Parkinson’s disease and Alzheimer’s disease.

This could also result in development and milestone payments and royalties.

Unprecedented and astounding

In an interview with pharmaphorum Secor said that the technology comes from a discovery in Khvorova’s lab, which showed the technology allowed potent silencing of Huntington’s for six months in mice.

In 2019 results were published in Nature Biotechnology of the research findings, which were described by Secor as “unprecedented and astounding”.

The founders formed the company with backing from investor F-Prime Capital and has just launched its Series A round with Roche’s Genentech unit and Biogen the first to invest.

Secor said: “In my 30 years I have never seen this level of interest.”

The technology could be used in several indications, according to chief scientific officer and RNAi expert Aimee Jackson.

“There is distribution to multiple regions and deep brain structures and enhanced potency and duration of action,” she said.

With the backing the company already has, Secor said the plan is to focus on preclinical development without needing to worry about funding for the time being.

Quizzed about the possibility of an IPO or buyout from a big pharma company, Secor said the focus is very much on developing the technology.

She told pharmaphorum: “Our long-term vision is to be discovery research and commercialisation platform and product company for a very long time.

“We have a long cash runway to do some building,” she added.

Female leadership

Secor also noted the strong role that women have played in founding and running the company, noting that the name Atalanta refers to the famous female character from Greek mythology.

It also reflects the contribution from Khorova as the founder of the company.

There’s also a connection with Greek mythology from the name of the protein that is targeted by the RNAi drug in Huntington’s – Argonaute 2.

In many versions of Jason’s quest for the Golden Fleece, Atalanta served as the only woman on the crew of the Argo.

Secor noted the recent appointments of Emma Walmsley and Reshma Kewalramani as CEOs at GlaxoSmithKline and Vertex, respectively.

She told pharmaphorum: “I wanted to keep the name to remind us we were named after a woman.

“I think there is a big movement around diversity and inclusion its an opportunity to break through those glass ceilings women should be feeling empowered to take leadership roles.

“It creates a whole lot of opportunities for women. This is a good time for women and I am excited to see more women step into leadership roles.”

 

 

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Clene goes public with gold-based neurology nanotechnology

Biotech Clene Nanomedicine has gone public with a mission to use nanotherapeutics that will use gold to treat devastating neurological diseases including Parkinson’s disease.

Over the Christmas period Clene closed a reverse merger with Tottenham Acquisition I Limited, allowing shares to be publicly traded on the Nasdaq stock exchange.

The US-based company says it aims to revolutionise treatment of diseases including multiple sclerosis and amyotrophic lateral sclerosis with a new class of drugs that use gold to catalyse the cellular reactions fundamental to life.

Proceeds from the transaction totalled $31.9 million, combining funds held in Tottenham’s trust account and financing from Clene shareholders.

The current pipeline includes a phase 3 study in ALS and four phase 2 studies in ALS, MS and Parkinson’s.

Lead candidate is CNM-Au8, is an orally administered, bioenergetic gold nanocatalyst designed to enhance critical intracellular bioenergetic reactions necessary for repairing and reversing neuronal damage.

The company says its approach is based on the understanding that energy is the essential building block to life and that bioenergetic failure underlies the makeup of many neurodegenerative diseases.

Clene says its technology is based on active nanocrystals to activate reactions within the body that have shown to enhance cellular repair and regeneration.

Preliminary blinded data from the phase 2 RESCUE-ALS trial announced at the Symposium on ALS/MND show that more than 40% of enrolled patients with completed 12-week data experienced an improvement in motor neuron function as assessed by a standardised score.

Compared to baseline values the average score showed an increase that exceeded the expectations on which the study was based, the company said.

This suggested that CNM-Au8 may have neuro-reparative potential in ALS and expects completed unblinded results from the RESCUE-ALS study in the second half of 2021.

CNM-Au8 was selected as one of the first drug regimens to be evaluated in the phase 3 HEALEY ALS Platform Trial, a placebo-controlled study testing several novel ALS therapies at the same time to cut costs.

It includes substantial financial support from philanthropic donors and foundations and provides access to 54 expert ALS clinical trial sites across the US.

Dosing was initiated in the Clene-specific portion of the platform trial in July 2020 and full enrolment is expected by the end of Q2 2021, with top-line data available in the first half of 2022.

 

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Biogen signs $2.1 billion Parkinson’s disease deal with Denali

Biogen has bought a licence to co-develop and co-market potential Parkinson’s disease drugs with US biotech Denali, in a deal worth more than $2.1 billion.

Denali has been working on small-molecule compounds capable of crossing the blood-brain barrier and Biogen has bought a licence to co-develop and co-market compounds known as LRRK2 inhibitors in Parkinson’s disease.

Biogen and Denali will co-market the lead LRRK2 product in the US and China, and Biogen will commercialise in all other markets.

A molecule codenamed DNL151, currently in phase 1 development, has been selected to progress into late stage clinical studies that are expected to begin in 2021.

Mutations in the LRRK2 gene can cause Parkinson’s disease. LRRK2 regulates lysosomes, which play a vital role in cell function by breaking down excess or worn-out cell parts, and can also destroy invading viruses and bacteria.

Function of lysosomes is impaired in Parkinson’s disease and may contribute to the neurodegeneration that causes the symptoms of the disease.

Inhibition of LRRK2 activity may slow the progression of Parkinson’s disease in patients with and without known genetic risks based on restoration of lysosomal function.

As well as the LRRK2 drug Biogen will also receive an exclusive option to license two preclinical programmes from Denali’s drug transport platform, which aims to improve brain uptake of biotherapeutics.

Denali’s TV platform is a proprietary technology designed to effectively deliver large therapeutic molecules such as antibodies, enzymes, proteins and oligonucleotides across the blood-brain barrier after intravenous administration.

Biogen will have right of first negotiation on two additional transport vehicle (TV) enabled therapeutics, currently at a preclinical stage, should Denali decide to seek a partner.

Biogen will make an upfront payment to Denali of $560 million and make a $465 million equity investment in Denali from the purchase of 13.3 million newly issued shares of Denali common stock at approximately $34.94 per share, representing 11.2% of Denali’s outstanding stock.

Should the LRRK2 program achieve certain development and commercial milestones, Denali will be eligible to receive up to $1.125 billion in potential milestone payments.

People who have Parkinson’s disease experience numerous symptoms, including tremors, slow movement, muscle stiffness and impaired balance.

As these symptoms become progressively worse, patients have difficulty walking, talking or completing other simple tasks.

Parkinson’s disease is the second most common neurodegenerative disease and there are no approved therapies capable of slowing progression.

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