Originally Published as The Niche Podcast
This week, look closer – with drops, controlled gene therapy, acquisitions in antibodies, new antifungals, new biosimilars, and several IPOs
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Music by Luke Goodson
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About the Show
Life Science Today is your source for stories, insights, and trends across the life science industry. Expect weekly highlights about new technologies, pharmaceutical mergers and acquisitions, news about the moves of venture capital and private equity, and how the stock market responds to biotech IPOs. Life Science Today also explores trends around clinical research, including the evolving patterns that determine how drugs and therapies are developed and approved. It’s news, with a dash of perspective, focused on the life science industry.
Introduction
Welcome to The Niche Podcast – Your weekly rundown of the biotech, pharma, clinical research, and life science industries. I’m your host, Dr. Noah Goodson. This week, look closer – with drops, controlled gene therapy, acquisitions in antibodies, new antifungals, new biosimilars, and several IPOs.
Disclaimer
The views expressed on The Niche Podcast are those of the host and guests. They do not necessarily reflect the opinions of any organizations or companies with which they are affiliated.
Drop the Farsightedness
One of the single most common experiences for aging humans is the development of Presbyopia, commonly called “farsightedness.” While there are multiple causes for poor close-up vision, a common age-related cause is the stiffening of the lens, making it difficult or impossible to focus on close-up objects. Numerous attempts have been made to generate an easy and affordable ophthalmic solution to solve this problem, but none have yet to succeed.
Now, Allergan and AbbVie believe their phase III trial of AGN-190584 may have done the trick. The therapy is a simple eye-drop that has a rapid onset increasing the ability to focus close and lasts for up to 6 hours. A key ingredient, pilocarpine, was used to treat glaucoma in the past but had mixed results from certain side effects. I imagine this novel formulation attempts to circumvent these side effects, and according to the study with some success. I’m not sure how the FDA will look on this study. Since some of the previous concerns with pilocarpine were based on side effects in long term use, I think it’s possible the FDA will ask the sponsors to extend out the trial’s length, but perhaps long-term use data from the real world will suffice to assuage their concerns. For now, we’ll just have to wait till the end of 2021 to see.
Drug Induced Gene Therapy “dimmer switch”
The future of medicine may get quite complex indeed. Researchers at Children’s Hospital of Philadelphia (CHOP) published a Nature paper last week highlighting a novel gene therapy “dimmer switch.” Basically, it’s a mechanism to drive protein expression in gene therapy from oral drug consumption. The details of how precisely this works are fairly nuanced. The basic idea is that most gene therapies insert, replace, or turn off a given gene. But in the body, gene regulation is quite complex. If you could intentionally modulate the activity of a given gene, there could be new possibilities for certain indications. The research team at CHOP have published a mechanism called Xon that allows them to control protein expression through an oral drug route in gene therapy. Basically, you give a “normal” gene therapy but then utilize an oral drug to control the levels. Key features of Xon include the fact that it’s repeatable – you can take the drug and drive protein expression which fades till you take the drug again. It’s dose dependent – if you need more protein, take more therapy. It can be turned off or down by no longer using the oral drug.
This of course is a long way from clinical applications, but advancements like these in biological engineering will eventually lead to refined and precise applications that stretch far beyond human medicine. As we’ve said on this show before, it is a brave new world out there.
Amgen Acquires TeneoBio for $2.5B
Amgen has announced a $2.5B deal to acquire the privately held TeneoBio. Teneo has a suit of technology around antibodies including some pretty cool bispecific antibody solutions. Their pipeline, at least on paper, is pretty dense with partnerships across big pharma. Now Amgen has decided they want the whole package. To do that they’re starting with $900M in cash payment followed by milestone payments into the billions. It should be noted that Teneo does have core assets in preclinical and early clinical phases, many aligned with partners like GSK, Janssen, and AbbVie. With immunotherapies driving a huge portion of global pharmaceutical value. It’ll be interesting to see if Amgen fully incorporates Teneo into their pipeline or if it’s just another acquisition investment bet.
Antifungal Possibilities
Most of us are well aware of the growing prevalence of antibiotic resistant bacteria. The evolutionary forces are clear. As we use antibiotics more and more we are acting as the driver of antibiotic resistance. Our ability to fight bugs, is what makes super bugs. The same turns out to be true for fungal infections. Over the last couple of decades, rare but complex and deadly fungal infections are on the rise. Despite the increase, no new antifungals have been approved in the United States in 15 years. This is in part because anti-fungal treatments, similar to antibiotics, are not all that profitable. But it’s also because they’re quite hard to discover and test . That’s why the FDA put drug-resistant Candida auris as an urgent threat. A new therapy from Cidara Therapeutics appears to be safe and effective based on two Phase III clinical trials of their novel antifungal rezafungin to treat these rare and complex fungal infections. With other data still rolling in from another trial, don’t expect an FDA submission till 2022. But for now, this is promising news on the fungal front.
Biosimilars – Insulin
Biosimilars are a class of biologic medical product that are critical to providing affordable medical care. Unlike more common “generics” which are small molecules made outside of the patent window, biosimilars tend to be large complex molecules. They are “similar” if their biological structure and production process maintains enough similarity to the original to perform the same biological function as a medication.
Viatris Inc and Biocon Biologics have received FDA approval for Semglee as the first biosimilar to insulin glargine. The 351(k) designation gives the companies 12 months exclusive biosimilar rights in the US market. Long term, it means more competition in the long-acting insulin space. For therapies like insulin that are necessary, ongoing, and lifesaving, producing safe and effective biosimilars is a great way to bring down the prices long term.
Sophia $234M closing + Caribou $304M closing + Artois $134M Series C
A few noteworthy rounds of fund-raising this week. First, SOPHiA GENETICS closed a $234M IPO + they also directly sold $20M in private placement shares to GE Healthcare. At this amount, you’re probably thinking SOPHiA is a gene therapy or oncology company. They are in fact a SAAS (software as a service) platform for AI driven data analytics in healthcare. The future of medicine will certainly have AI driven integrations. SOPHiA is pushing some of this technology forward in genomics, radiomics, and most challenging a multimodal integrative model.
Caribou Biosciences has closed an outsized $304M IPO. They have a diverse technological and clinical pipeline with products in CAR-T, CAR-NK for solid tumors, and a significant technology base in CRISPR-Cas gene editing they call chRDNA. Their CRISPR technology is used to edit the CAR-T cells and drive their therapeutic approach. If their pipeline bares fruit, they could be a valuable acquisition target since they combine those key elements of interesting pipeline + inherently valuable technology.
Artois Pharma has raised a $134M series C round to advance their DNA Damage Repair (DDR) technology pipeline. DDR is critical to certain tumors and disrupting this pathway could serve to directly target tumors or be used in combination therapy. Artois is still an early-stage biotech and does not have a clear clinical pipeline to date. Doubtless this new round of funding will advance them to early clinicals.
Closing Credits
Thanks for joining me on The Niche Podcast; your weekly summary of top news in the biotech, pharma clinical research, and life science industries. You can learn more at thenichepod.com or find us on your favorite podcast app. Like, comment, subscribe, and most of all share with your friends. If you like what you hear, please rate and review, it really helps us. Once again, I’m Dr. Noah Goodson, I’ll see you next week.