Alnylam, Intellia, Third Harmonic, Galvanize

A $900 raise, gene therapy news, a quick IPO, and merge three for $100M

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Story References
Alnylam, Link 2, Link 3
Intellia Therapeutics
Third Harmonic Bio
Galvanize Therapeutics

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. 

Transcript

Introduction

Welcome to Life Science Today, your source for stories, insights, and trends across the life science industry. I’m your host, Dr. Noah Goodson. This week, a $900 raise, gene therapy news, a quick IPO, and merge three for $100M. 

Disclaimer

The views expressed on Life Science Today are those of the host and guests. They do not necessarily reflect the opinions of any organizations with which they are affiliated. 

Alnylam Raises $900M Senior Notes

Alnylam, the RNAi biotechnology company, has announced plans to raise $900M in convertible senior notes. The raise comes on the heals of a series of positive data across their clinical pipeline, including promising data from their Phase 3 study of the therapy Patisiran in patients with ATTR Amyloidosis with Cardiomyopathy (ATTR-CM). The study demonstrated a positive safety profile and improvements in function compared to the placebo control. ATTR-CM primarily impacts the heart where amyloid tissue build’s up resulting in fatigue, swelling, and other heart-related issues. In addition to clinically relevant functional outcomes, the therapy showed statistically significant 87% reductions in blood-serum levels of TRR, the key protein driver. Based on this data it’s likely Patisiran could go to the FDA for expanded approval in the near future. Alnylam also released positive results from their phase II study of Cemdisiran to treat IgA Nephropathy and will progress to phase 3 studies. 

These two pieces of news expand the future outlook for RNA-based biotechnology companies. Unlike gene-editing companies, RNAi infusions provide targeted interference RNA, which work to silence the translation of transcribed genes. In cases of gene-over production, this approach can decrease excess buildup of the targeted proteins, but unlike gene-editing, the effect is not permanent – additional infusions will be required over time to maintain the effectiveness over time. This is both positive in that it is possible to stop the therapy and space dosing’s to therapeutically relevant windows for individual patients, but it also means repeated infusions which are the primary source of adverse events to date. The big news for now, is that Alnylam is on track to demonstrate a portfolio of RNAi therapies can be developed targeting multiple rare genetically driven conditions. The nearly $1B raise will help them see these to market. 

Intellia Provides More Promise for CRISPR

Not to be left in the dust by RNAi therapies, Intellia Therapeutics has reported positive early results from their in vivo therapy targeting ATTR-CM. Yes, they are targeting the same condition, but this time with a gene therapy. Intellia is at a much early stage with a first in human gene therapy, but there data so far show positive safety profiles and remarkably robust reduction in blood serum levels of serum transthyretin. The study will continue, but doubtless Intellia are ramping up plans for a phase II study in 2023 once all data are collected. In similar news, they also announced positive interim data for a separate CRISPR gene therapy treating hereditary angioedema (HAE). So far, the data show the therapy effectively reduces the overactive protein kallikrein that drives the condition as well as decreases in the potentially fatal swelling attacks that occur as a result. The mean reduction in attacks was 75% in the low does and 92% in the high dose. While these studies are early stage it suggests both impact on the underlying causes and measurable clinical differences. 

Taken together with the previous story, these therapies are coming to life, but the path is complex, especially for the organizations forging the path for approval, battling to preserve investor confidence and at times fighting to win on the same rare disease conditions. I’m not sure if Intellia feels this way, but I remember my first-time editing gene in living creatures using CRISPR. It was such battle of careful technique to complete each step of the process, but when I saw the first data demonstrating unequivocally that it worked I was shocked. And I’d like that to be the take-away here, not some speculation  on stocks or company outcomes. We are using a bacterial defense system discovered just a decade ago in humans and permanently stopping a disease’s symptoms with a single dose. I think that is pretty amazing. 

Third Harmonic Upsized $185M IPO

Third Harmonic Bio has announced a $185M upsized IPO. Opening on the Nasdaq Thursday, shares shot up 20% but settled with much of the rest of the biotech market on Friday. Their candidate THB001 is a KIT inhibitor currently in early clinical studies for the treatment of the dermatological condition urticaria, also known as chronic hives. THB001 is being studied to treat both the inducible form, triggered by certain stimuli, and spontaneous urticaria, which occurs for no known reason. The KIT inhibitor specially targets the action of MAST cells, which are critical to immune defense and wound healing, but also play a role in allergic reactions. Third Harmonic has set hives as the first target, but believes multiple conditions may benefit and has preclinical work underway for asthma. The short runway from stealth exit earlier this year to IPO may be related to the single-molecule pipeline but I suspect it’s more driven by approach of their VC backers. Either way, this capital should progress Third Harmonic through early phase studies.

Galvanize Therapeutics Merges with $100M Series B

Galvanize Therapeutics has announced the merger with its sibling companies Gala Therapeutics and Galaxy Medical into one company focused on pulsed electric field energy therapies for a range of conditions. All three organizations originated under Apple Tree Partners. They were developing in parallel but non-identical directions. The merger reads more like a pragmatic refocusing of an R&D department than the merger of three distinct organizations which may reflect the operational reality or specious conjecture on my part. Regardless of the internal and fiscal politics of the move, the announcement is accompanied by the critical raising of $100M series B, with financing lead by Fidelity. The company further announced that they have received a 510(k) for a soft tissue ablation system, and two CE Marked systems for other indications. While they are still pre-commercial, they certainly have some starting material to expand out their clinical efforts with the newly combined and funded organization. 

Closing Credits

Thanks for joining me for Life Science Today, your source for stories, insights, and trends across the life science industry. Learn more at LifeScienceTodayPodcast.com. If you like what you hear, please tell a friend. Once again, I’m Dr. Noah Goodson, I’ll see you next week.