Reata Pharmaceuticals, Inc.

The kidney hasn’t always gotten much love from genetic medicine. The organ is vital to our health, but is often viewed as a liability for drug developers. It can flush out otherwise helpful therapies, and many efforts to deliver medicines to it have been foiled by its intricate and effective plumbing. A new startup says that it’s finally figured out how to shuttle RNA-based medicines directly into specific cells of the kidney. After three years in stealth, the Cambridge, MA-based startup, Judo Bio, launched on Monday with $100 million in a combined seed and Series A financing, co-led by Atlas Venture, The Column Group and Droia Ventures. CSO Alfica Sehgal said the idea for the company came during a brainstorming session about the lack of precise treatments in kidney care. “The kidney is not straightforward. It’s a very complex organ with dozens of specialized cell types,” Sehgal told Endpoints News in an interview. To try and solve the problem, the company borrowed a page from one of genetic medicine’s best-known delivery targets — the liver. Alnylam, which pioneered the first gene-silencing treatments, figured out that attaching a sugar molecule known as GalNAc to a siRNA drug would help the treatment get swept up by certain receptors on liver cells. So Judo’s team began to wonder if a similar pairing could be discovered to shuttle siRNA medicines into kidney cells. The startup focused on a kidney receptor known as megalin, which acts like a hungry scavenger, sucking up all manner of proteins and other biomolecules, Sehgal said. Judo has been working on finding molecules that they can use to get siRNA drugs swept up by megalin receptors in the proximal tubular epithelial cells of the kidney, she added. Judo isn’t disclosing the chemical makeup of its megalin-targeting molecules, but in a poster presentation at an RNA conference in Montreal this week, it revealed that in a mouse study, its approach increased the exposure of siRNA in the kidney up to 30-fold compared with naked RNA molecules. One injection lowered gene expression by 50% to 70%, and the effect lasted four weeks. Now more genetic medicine developers are beginning to look at the organ. In August, Versant Ventures and Novartis launched Borealis Biosciences with $150 million to develop RNA medicines for kidney diseases — it disclosed even fewer details about how it plans to deal with delivery. Alnylam leaders have also pointed to the kidney as a potential area of interest as part of its effort to expand the reach of its siRNA medicines beyond the liver. Judo CEO Rajiv Patni, who was recruited to lead the startup last month, told Endpoints it will initially use its siRNA drugs to shut down the production of solute carrier proteins, which line the tubes of the kidney and control levels of molecules like amino acids and glucose in the blood. A class of diabetes drugs known as SGLT2 inhibitors works by blocking certain solute carrier proteins. The startup Jnana Therapeutics, which Otsuka Pharmaceutical bought in a deal worth at least $800 million in August, was focused on making small molecule inhibitors to some of the hundreds of other proteins in the solute carrier family. Judo aims to make genetic medicines that tamp down on these proteins with longer-lasting effects. Patni, who was most recently the head of R&D at Reata Pharmaceuticals before Biogen acquired the company for $7.3 billion last year, won’t say what diseases Judo will focus on first, but he is interested in both rare metabolic diseases like phenylketonuria and urea cycle disorders, as well as more common conditions including diabetes, gout, heart failure and hypertension. “We’re using kidneys as the access point,” Patni said. The company also plans to target kidney cells known as podocytes to directly treat renal diseases, Patni added. But he declined to say when he expects Judo to have a drug candidate ready for the clinic or how long the company’s new funding will last.

Frazier Life Sciences has added more than $630 million to its evergreen public fund, the firm said Wednesday. The long-only fund, which is based in Palo Alto, CA, has been marketed to investors as a “hybrid structure to navigate volatility and liquidity in SMID-cap biotech,” according to materials posted by the New Mexico State Investment Council, which is committing up to $50 million to the public fund. “We are seeking to strategically take advantage of market volatility,” the firm said in June in its investor presentation. It now has $1.7 billion in total commitments after debuting with $830 million in 2021 and raising $243 million a year later. The biotech landscape is far different than when Frazier unwrapped its public fund in the fall of 2021. In September of that year, the number of life sciences companies with a negative enterprise value was 21. As of Sept. 20 of this year, that tally stood at 133, according to a recent Stifel report . “The number of early-stage companies going public has just exploded over the course of the last five-plus years,” general partner Jamie Brush told Endpoints News in 2021. “So the opportunity set for early-stage companies where Frazier wants to be investing has really increased on the public side.” The biotech IPO market cratered soon after that unveiling. Since then, initial public offerings have been few and far between, though there’s been a string of debuts this fall and more interest-rate cuts could be a positive signal for the market going forward. Follow-on offerings, meanwhile, have had a “ very strong year ,” according to Stifel. Frazier’s strategy Frazier has five main strategies for the fund. The first is “fallen angels,” which Frazier has described as companies that have “fallen out of favor but have promising assets,” like BridgeBio and Immunovant. Bain Capital Life Sciences, which just raised a $3 billion fourth fund, employs a similar strategy . Another bucket is “proprietary information,” in which Frazier gets data that are not publicly available, as it has done in the case of Rocket Pharmaceuticals and Mirum Pharmaceuticals. The third is “structured terms” or “creative” deals. The firm also invests in companies that are newly public like CG Oncology, which had the largest biotech IPO of the year at $380 million in January . And the final strategy is “off-the-beaten path,” or companies that are “lesser known” such as Egetis Therapeutics and Chinook Therapeutics. Novartis bought Chinook in a deal worth up to $3.5 billion last year. Frazier has said its public fund portfolio companies have secured a dozen FDA approvals, including Acadia’s Daybue, bluebird’s three gene therapies, Iovance’s Amtagvi and Phathom’s Voquezna. Some companies have been acquired, including Iveric Bio, which sold to Astellas for $5.9 billion ; Reata’s $7.3 billion exit to Biogen; Alpine Immune Sciences’ $4.9 billion sale to Vertex; and Sierra Oncology’s $1.9 billion exit to GSK. On the venture side, Frazier last disclosed a $987 million life sciences fund in March 2022.

Ernest Mario, Ph.D. steps down as Chair for health reasons; Matthew Pauls, J.D., M.B.A. assumes role of Lead Independent Director Dawn Carter Bir, seasoned biotech executive with significant commercial and strategic expertise, joins the Board of Directors REDWOOD CITY, Calif., Aug. 15, 2024 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced updates to its Board of Directors. Dawn Carter Bir, a seasoned biotechnology executive with over 30 years of industry executive leadership and strategic experience, has been appointed to Soleno’s Board of Directors. In addition, Board Chairman Ernest Mario, Ph.D. is stepping down from the Board due to health reasons. Current Board member Matthew Pauls, J.D., M.B.A. is assuming the role of Lead Independent Director. “We wish Dr. Mario well as he steps down from the Board. We extend our sincere gratitude to him for his invaluable contributions as the Chairman of our Board,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics. “His extensive pharmaceutical industry experience, spanning over four decades, has been instrumental in guiding Soleno’s growth towards becoming a commercial stage company.” “We are also pleased to welcome Ms. Bir to Soleno’s Board of Directors. She has a wealth of experience in commercialization, strategic business development and global operational leadership spanning more than 30 years. Her extensive experience will be instrumental as we prepare for a potential launch of DCCR.” Ms. Bir said, “I am delighted to be joining Soleno’s Board and work towards advancing the company as DCCR approaches potential approval. I look forward to collaborating with the talented Soleno team to help support its growth as they prepare to potentially commercialize their first product.” Ms. Bir recently served as Executive Vice President, member of the core executive team, and Chief Commercial Officer of Reata Pharmaceuticals for over seven years through the company’s first product approval and launch and $7.3B acquisition by Biogen in September 2023. Prior to Reata, Ms. Bir served as Vice President Sales and member of the Pharmacyclics executive leadership team through the launch of the company’s first product, a novel, first-in-class and blockbuster hematology therapeutic. Pharmacyclics was acquired by AbbVie in 2015 for $21B. Ms. Bir currently serves on the Geron Corporation board of directors, a public, commercial-stage oncology company with a first-in-class telomerase inhibitor. About Soleno Therapeutics, Inc.Soleno is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. The company recently submitted an NDA to the FDA, supported by its Phase 3 development program, for its lead candidate, DCCR (diazoxide choline) extended-release tablets, a once-daily oral tablet for the treatment of Prader-Willi syndrome (PWS). For more information, please visit www.soleno.life. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical facts contained in this press release are forward-looking statements, including statements regarding the timing of any regulatory process and ultimate approvals for DCCR for the treatment of PWS. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "anticipate," "could," "intend," "target," "project," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negative of these terms or other similar expressions. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, including the risks and uncertainties associated with the FDA’s filing and / or review of our NDA, market conditions, as well as risks and uncertainties inherent in Soleno’s business, including those described in the company's prior press releases and in the periodic reports it files with the SEC. The events and circumstances reflected in the company's forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Except as required by applicable law, the company does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Corporate Contact:Brian RitchieLifeSci Advisors, LLC212-915-2578