River Vision Development Corp.

A young biotechnology company plans to move an experimental medicine for a rare eye disease into late-stage testing after results from a small study signaled that it is generally safe and works as intended.On Wednesday, Los Angeles-based Acelyrin said a Phase 2b/3 trial will begin in the back half of this year the first of two studies the company hopes will serve as the foundation for an approval filing with the Food and Drug Administration. Acelyrin has been testing its medicine, known scientifically as lonigutamab, in people with thyroid eye disease, an inflammatory condition that progressively damages tissues around the eye.In that smaller trial, participants were divided into two groups. Cohort 1 was placebo-controlled, with two patients in the control arm and six patients given 40 mg of lonigutamab every three weeks over the course of six weeks. Cohort 2, meanwhile, was open-label and used a dosing schedule of one 50 mg loading dose followed by once-weekly 25 mg injections. Acelyrin said six-week data were available from six patients in this cohort.According to the company, there were clinically meaningful responses across each of the main ways the trial measured the effectiveness of lonigutamab. Among the drug-treated participants in Cohort 1, one quarter improved by at least one point on a double vision scale; one-half improved by at least two points on an eyeball protrusion scale; and all improved by at least two points on a clinical activity score which evaluates symptoms of thyroid eye disease.Neither of the two placebo-treated patients showed such improvements.In Cohort 2, two, four and five of the six participants had responses across those three measurements, respectively.Acelyrin noted that patients tolerated its drug well. So far, researchers have not reported any serious adverse events or cases of hearing loss or high blood sugar. An antibody drug, lonigutamab is designed to block a protein tied to insulin-like growth factor-1, a hormone that affects insulin sensitivity as well as the growth of bones and tissues.An already approved drug for thyroid eye disease, Tepezza, targets the same protein. But unlike Acelyrins medicine, which is given via an under-the-skin injection, its administered as an intravenous infusion over the course of 60 to 90 minutes.Genmab and Roche originally investigated Tepezza as a potential cancer treatment, before a small biotech named River Vision began developing it in eye disease. Horizon Therapeutics bought River Vision in 2017 for an upfront cash payment of $145 million. Then, six years later, Amgen acquired Horizon for nearly $28 billion.Within Horizons portfolio of therapies for rare and immune system-related diseases, Tepezza stood out as especially lucrative. First approved in January 2020, the drug has reached blockbuster status in each of the last three years. In 2022, net sales were nearly $2 billion. Amgen, which completed the Horizon acquisition on Oct. 6, recorded $448 million worth of Tepezza sales between then and Dec. 31.Acelyrins drug, should it ultimately secure approval, would compete with Tepezza and may even hold an advantage since its given as a quick injection.It is important to note that this is preliminary data in a small group, however, the positive results are highly promising, said Shoaib Ugradar, a private practice ophthalmology specialist, in a statement from Acelyrin.Given the growing body of evidence that suggests thyroid eye disease may have long-term sequelae, the convenience of a subcutaneous administered medication with a potentially favorable side effect profile becomes critical, Ugradar added.But Acelyrin isnt alone. Viridian Therapeutics is advancing a similar type of medicine. One intravenous version is already in Phase 3 testing, while another in the form of a subcutaneous injection could follow later this year. Though its tough to compare drugs across trials, overall Viridians looks better at this time, wrote Jefferies analyst Michael Yee.The results suggest ... lonigutamab is an active drug, but with significant caveats around all of the data that we havent seen, added Leerink Partners analyst Thomas Smith, in a note to clients.Acelyrin said further data will be presented at future scientific meetings.Shares of the company were up about 6% by late Tuesday morning, to trade at $8.42 apiece.Acelyrin launched in late 2020 with backing from the venture capital firm Westlake Village Partners. It had raised $550 million before going public in May 2023, which provided another $540 million in funding.The companys most advanced drug, called izokibep, is in late-stage testing for various immune disorders that affect the skin, eyes and joints. Acelyrin shares lost more than half of their value last year, after izokibep failed one of those trialsin September. '

Dive Brief:SR One, the former venture arm of GSK, has raised its second fund since spinning out of the pharmaceutical company, announcing Monday a new $600 million bankroll to invest in young biotechnology companies.As the biotech sector pulls back on initial public offerings, SR One is eyeing opportunities to get in at more attractive entry points for later-stage investments, though it will still invest in seed and Series A rounds, said CEO Simeon George.In 2020, SR One completed a $500 million fund that infused cash into emerging biotechnology companies such as Nimbus Therapeutics and MiroBio before their blockbuster deals, along with Arcellx before its Nasdaq debut.Dive Insight:SR Ones decision to become a standalone firm after serving as GSKs venture arm for 35 years was what George described as a win-win for both entities.GSK wanted to put more money into its drug pipeline. The investors at SR One, on the other hand, were itching to build their own portfolio.Three years later, that split has paid off. SR One is now free to broaden the scope of its investments, while GSK shares in its successes by contributing to its fund.Some of the firms bets have paid off handsomely, too. SR One supported Nimbus, for example, before the biotech sold an autoimmune drug to Takeda for $4 billion, and MiroBio prior to its acquisition by Gilead Sciences. It also invested in Arcellx, one of a short list of biotechs that have found IPO success over the last two years.It was, frankly, an amazing year for us, George said. It's really focused on those fundamental value creation points show me that you have a drug that's actually treating patients. If you do that, even in the worst year that we've had in a number of years, you create value.SR One has already been putting its second fund to work. One of the beneficiaries was Mineralys Therapeutics, which raised $192 million in 2023s largest IPO to date.Going forward, George and his fellow investors are interested in product-focused and platform biotechs based on broader drugmaking technologies, he said.The company was an early investor in the gene editing biotech CRISPR Therapeutics, and remains excited to do platform investments, he said. But George expressed a preference for more narrowly built platform companies that have a clear value proposition, a sentiment thats become more common during the markets pullback.There does need to be a thesis around where you can exemplify why this is the right modality to be able to derive a clinical benefit, he said of investments in platforms.One investment thats currently in the works has roots in River Vision, a startup acquired by Horizon Therapeutics and a key reason Amgen recently agreed to buy the company for $28 billion. SR One was an investor in River Vision, and is working with the scientists behind the startup to develop two companies focused on kidney disease, he said.The fundraise, meanwhile, adds to whats been a record haul of late for venture capital investors. Venture firms raised about $163 billion in 2022 and put nearly $31 billion into biotech and pharma investments last year, according to the National Venture Capital Association.Editors note: This story has been updated to clarify that SR One invested in River Vision, not Horizon Therapeutics. '

WILMINGTON, Mass.--( BUSINESS WIRE )--Charles River Laboratories International, Inc. (NYSE: CRL) recently opened the first Charles River Accelerator and Development Lab (CRADL®) location in Chicago, expanding contract vivarium space for the biopharmaceutical industry in Illinois. CRADL Chicago is located in Fulton Labs , in the heart of Chicago’s Fulton Market, which is quickly emerging as a hub of the local life sciences community. Fulton Labs also includes premier wet and dry laboratory and office space, offered by Portal Innovations. By offering on-demand facilities, both emerging and established biopharmaceutical companies and research institutions are able to quickly start new projects, accelerating the early stage of research. This allows scientists to focus on research, while leaving the animal husbandry and daily vivarium management to a trusted partner. The Combined Expertise and Reach of Explora BioLabs and CRADL With the acquisition of Explora BioLabs in April 2022, the CRADL-Explora Vivarium Network was established as a premier partner for clients to launch or expand their drug research programs, with the added benefit of access to Charles River’s complete portfolio of integrated drug discovery and non-clinical development resources. The CRADL-Explora Vivarium Network operates 28 vivarium facilities, providing over 380,000 square feet of turnkey rental capacity in key biohubs, including Massachusetts, California, Washington, Shanghai, London, and now Illinois. This network supports the growth of the entire biotech ecosystem in each city, allowing researchers to rapidly engage in their research while maintaining the flexibility to relocate or co-locate, knowing they will have reliable, high-quality facilities nearby. Approved Quotes “Chicago is emerging as a key biohub in the United States, with a growing life sciences community. By providing contract vivarium services, we’re able to support clients in starting projects quickly, without the burden of building infrastructure, which can be costly and time consuming.” –Colin Dunn, Ph.D., Corporate Senior Vice President, Global Research Models & Services, Charles River "Charles River has the largest network of contract vivarium facilities globally, and we're thrilled to have one at our Fulton Market location. Their depth of experience provides an incredible opportunity for the life sciences community in Chicago and other biotech communities. We're glad to have established a relationship with a world-class organization like Charles River, and we look forward to working together in the future.” —John Flavin, Founder and CEO, Portal Innovations About Charles River Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visit www.criver.com.