ADX-038

ADARx has raised more than $300 million in less than two years. Who says crossover rounds are dying? ADARx has raked in a $200 million series C, almost twice as much as the previous two joint series B rounds combined. The question now is, how quickly can the biotech's RNA pipeline be expanded?  The new financing shared Wednesday will help ADARx advance two lead assets—ADX-324 and ADX-038—into the clinic, though more precise timing wasn’t disclosed. Since September 2021, the biotech had raised $121 from a series B and series B-1 round. The latter was announced in January, adding $46 million to ADARx’s bank. So far so good for the San Diego biotech which said at the time of its unveiling that the first $75 million from the series B would help launch into a first phase 1 trial for lead program ADX-324. That study was launched in January, testing the safety and tolerability of the med in both healthy patients and those with hereditary angioedema. The study’s sole trial site is in Australia with an enrollment goal of 50 patients.  The new proceeds will be used to advance the ongoing trial and push ADX-038 into its first human study, which is in development for multiple complement-mediated diseases, the company says. ADARx added that the money will advance a growing pipeline of mRNA-based therapies, which currently includes at least nine active programs, though a full list has yet to be publicized.  ADARx is trying to take space in a crowded room of RNA contenders, which has only grown in recent years thanks to the success of companies like Moderna and Alnylam. The selling point for the newly-financed company is its multi-faceted siRNA platform that includes both RNA editing and degradation or inhibition capabilities, which can produce therapies that either fix gene mutations or regulate expression.  “With this financing, we are well-positioned to achieve our clinical milestones for multiple clinical programs and to build a leading company in RNA therapeutics,” CEO Zhen Li said in a release.  The potential has evidently caught the eye of a fresh new batch of investors, particularly institutional bigwigs like Blackrock, T. Rowe Price Associates and Venrock. Previous investors Ascenta Capital, Lilly Asia Ventures, OrbiMed and SR One Capital Management also contributed to the round. 

Two companies working on advancing RNA medicines announced Wednesday that they have raised a combined $309 million in separate funding rounds. ADARx Pharmaceuticals, which took in $200 million in Series C investments, is looking to develop two next-generation RNA programmes, while Flagship-backed Alltrna said it raised $109 million in a Series B round to put toward a machine learning-driven platform based on transfer RNA (tRNA) biology.Ricky Sun, a partner at Bain Capital Life Sciences, which co-led the round for ADARx with TCGX, said the company has developed "a unique siRNA platform that we believe has the potential to generate differentiated clinical data from several programmes over the near- and long-term."ADARx says it will use the new funds to advance its ADX-324 programme for hereditary angioedema, as well as its ADX-038 asset under development for multiple complement-mediated diseases. It added that the fundraise, which also drew support from Lilly Asia Ventures, OrbiMed and SR One Capital Management as well as a string of new backers, will help finance a pipeline of mRNA silencing or editing candidates to treat a range of diseases.The company raised $46 million in a Series B-1 round earlier this year, coinciding with the entry of ADX-324 into Phase I. According to CEO Zhen Li, early clinical data "strongly support the potential of ADX-324 to be the best-in-class treatment for patients suffering from hereditary angioedema," and also validate the start-up's targeted delivery PLR platform and SPE technology.Meanwhile, Alltrna said its fundraising haul will help unlock the "combinatorial power of tRNA sequences and modifications," and move its first drug candidates towards the clinic for so-called stop codon diseases, an umbrella term that encompass thousands of genetic illnesses caused by a premature termination codon (PTC) mutation. Aside from Flagship, Alltrna said other participants in the round included "a number of top-tier investors.""Since launch, we've gained unparalleled insights into tRNA biology and shown that we can systematically design and deliver tRNAs with universal readthrough of PTCs and restore full-length functional protein production in vivo," remarked CEO Michelle Werner. She noted that the company's platform "optimises tRNA nucleotide sequence and modifications to turn tRNA's sophisticated biology into programmable medicines with powerful potential therapeutic properties."FirstWord recently caught up with Werner at the American Society of Gene and Cell Therapy (ASGCT) meeting where Alltrna presented some early data showing that its platform could deliver engineered, modified tRNA oligonucleotides with "significantly increased potency." You can read that interview here: ViewPoints: How Alltrna is leveraging tRNA biology to address genetic diseases.For an overview of the RNA landscape, check out Spotlight On: The growing universe of RNA editing technologies and Spotlight On: Startups advancing tRNA therapeutics.