Fresh off the news discussed in my last post on REPARE, what do you know, mere days later we have more good news in the new wave of biotech business here in the city of Montreal! Local bone disease specialist Clementia has filed with the SEC for an IPO of the order of $115M; more than a little irony that the filing first came to light via the team at Renaissance Capital, given that this move represents another significant piece in the renaissance puzzle of the local life science sector.
The company, which has only been around for seven years or so, was founded by Dr. Clarissa Desjardins, who is also Clementia's CEO, alongside Dr. Jean-Claude Tardif, a local eminent cardiologist. Dr. Desjardins is no newcomer to this business, having founded both Advanced Bioconcept and Caprion, residents both (initially) of BRI here in Montreal. Things have progressed quickly at Clementia, with over $100M USD raised to date, which included an oversubscribed $60M mezzanine round that underlined investor belief in the company and its product.
That product (their only pipeline asset for now) is their lead candidate for a disease known as fibrodysplasia ossificans progressiva (FOP), an ultra-rare condition that severely debilitates due to extra-skeletal bone growth in muscles, tendons and other soft tissue. Such heterotopic ossification essentially imprisons the patient in a second skeleton, if left untreated. There is no effective treatment nor cure for this horrific condition which results from an overactive BMP signaling pathway, apart from steroids and anti-inflammatories, which ease some of the pain, only.
This disease affects less than one person in a million, with under 1,000 patients currently living with it; however, companies such as Clementia believe that this type of prevalence should not deter drug developers from investing in such programs and that is a sentiment shared by the entire rare/ultra-rare disease community. Notwithstanding the whole debate ongoing vis-a-vis soaring drug prices, particularly in rare conditions, and what a certain Mr. Trump is going to do south of the border to rail things in!
Clementia's approach is via palovarotene, an oral RAR-γ agonist (R667) that is proposed to be able to prevent the abnormal bone growth seen in this disease, based on key data obtained in animal modeling of FOP. The compound was not discovered at Clementia, but was in-licensed from Roche as recently as 2014, having been discontinued as a drug candidate for COPD after it's safety had been assessed in several hundred people.
This potential product is now in Phase II clinical trials in adults and children with FOP, examining different doses of it in patients with flare-ups, and further documenting its safety in patients living with FOP. The FDA has granted palovarotene orphan drug designation and fast track status, with the equivalent designation being granted by the EMA. The severity of the disease and paucity of adequate therapies underlie such designations.
In the coming year and with a new boost to the coffers via the intended IPO, Clementia will commence the MOVE Phase III trial of palovarotene in FOP, but additionally, initiate a PhaseII/III trial in multiple osteochondroma. There's a lot of hope in this venture, but it remains a highly risky one, as do almost all biotech financings, but with just one product in the pipeline if something goes awry, I wonder what the back-up plan is that continues to apparently comfort investors.
One aspect of such confidence seems to be that in animal modeling of the disease, there was a lack of new bone formed even when treatment with palovarotene was inititated even six days post injury. This implies that during flare-ups, an appropriate treatment might be able to nullify the response prior to new bone formation, which is exciting. Further, researchers found that once palovarotene was withdrawn, its effects were maintained and continued inhibition of bone formation was observed. If such results can be repeated in human, this could represent a solid home run for Clementia, and the future would indeed be bright for both the company and patients, alike!
Like all biotech and drug development ventures, only time will tell, and it is unfortunately an achingly slow process to get from the bench to the clinic to the bedside - but somebody's gotta do it - and I am certain that all FOP patients are delighted that in this case, Clementia is going to try to do it. All the rest of us can do for now is to wish them all the very best, while enjoying their contribution to the revitalisation of Montreal's life science and biotech ecosystem. Bravo!
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