Uniqure NV shares (NASDAQ:QURE) were trading at $5.87, up $2.09, or 55%, after the firm made public updated interim data including up to 24 months of follow-up data from 29 treated patients enrolled in the ongoing U.S. and European phase I/II trials of AMT-130 for Huntington’s disease. New data show a statistically significant, dose-dependent slowing of the progression of the condition and lowering of neurofilament light protein in the cerebrospinal fluid at 24 months. Amsterdam-based Uniqure expects, in the second half of this year, to hold a type B, multidisciplinary, regenerative medicine advanced therapy meeting with U.S. FDA. Talks will involve the possibility for expedited clinical development pathways and accelerated approval of AMT-130, a gene therapy containing a microRNA that silences the huntingtin gene.

Soligenix lights the way with positive T-cell lymphoma data

Soligenix Inc. investors were extremely enthusiastic about the interim update for the company’s cancer therapy and light device for treating for treating early stage cutaneous T-cell lymphoma. The company’s stock (NASDAQ:SNGX) had tallied a whopping 464% increase at midday, with shares going for $11.56 each. Hybryte’s open-label, investigator-initiated study testing extended treatment of the synthetic hypericin sodium candidate for up to 12 months produced positive data as three of four subjects achieved treatment success, defined as 50% or greater improvement in their cumulative modified Composite Assessment of Index Lesion Severity score compared to baseline. Two achieved success within the first 12 weeks and the third was successful within 18 weeks.

Scirhom targeting iRhom2 in autoimmune disease with €63M series A

Autoimmune disease specialist Scirhom GmbH has raised €63 million (US$68 million) in a series A to take an antibody against iRhom2, an enzyme that regulates TNF-alpha secretion, into the clinic. The company has approval for a phase I trial in healthy volunteers that will start later this year, with plans for proof-of-concept trials in rheumatoid arthritis and inflammatory bowel disease to follow.

No surprises in FTC’s interim report on PBM practices

The redacted interim results released today of an ongoing FTC investigation into pharmacy benefit managers (PBMs) shed little, if any, new insight into PBM practices and how they impact availability and pricing of prescription drugs in the U.S. The key finding of the report is that the PBM market has become highly concentrated with the largest PBMs now vertically integrated with the nation’s largest health insurers and specialty and retail pharmacies – something that’s been known for years. When the commissioners voted today to release the report, Commissioner Melissa Holyoak was the only one who objected. She explained her dissent when testifying before a House FTC budget hearing a few hours later, saying the report lacks economic and empirical evidence, fails to look at how PBM practices affect consumers and doesn’t consider all the players in the prescription drug space.

Biocity’s SC-0062 hits endpoint in chronic kidney disease trial

Biocity Biopharmaceutics Co. Ltd.’s selective endothelin receptor type A antagonist, SC-0062, met the primary endpoint of reducing proteinuria in a phase II chronic kidney disease trial. The candidate showed a clinically meaningful and statistically significant reduction in proteinuria, with a clear dose-response relationship and good safety profile. No fluid retention was observed in the treatment group, and for patients who were on SGLT2 inhibitors, the combination with SC-0062 showed a favorable safety profile.

Discgenics’ cell therapy on phase III track for disc degeneration

Phase I/II data of IDCT (rebonuputemcel), a cell therapy in development for chronic and progressive lumbar degenerative disc disease (DDD) and one that has both regenerative medicine advanced therapy and fast track designations in the U.S., has been published in the International Journal of Spine Surgery for showing statistically significant improvements in pain, disability and quality of life. Next, the Salt Lake City-based developer Discgenics Inc. intends to start a phase III trial in the U.S., said Flagg Flanagan, the company’s CEO and chairman, noting that IDCT has the potential to “change the paradigm of care for patients with DDD.” The condition is currently treated with pain relievers, steroid injections, hot and cold therapy, exercise and other lifestyle changes, and sometimes surgery.

Alteogen’s Altos nabs ₩24.5B in series B to develop new eye drug

Altos Biologics Inc., founded as an eye disease-focused subsidiary of Alteogen Inc. in 2020, raised ₩24.5 billion (US$17.7 million) in a series B financing round to advance its pipeline of therapeutics for eye-related conditions, including age-related macular degeneration (AMD). Daejeon, South Korea-based Alteogen said July 9 that the funds raised will be funneled to develop OP-01, Altos’ candidate therapy for AMD. As a multispecific antibody, OP-01 is designed to work by inhibiting both VEGF- and Tie2-regulating pathways and normalizing leaky blood vessels, according to the company.

First Circuit: Frequency class action brushstrokes no van Gogh

Just because a statement turns out to be false doesn’t mean it was intended to be, the U.S. Court of Appeals for the First Circuit said last week in tossing a stockholder class action against Frequency Therapeutics Inc., now part of Korro Bio Inc. Affirming a lower court decision, the appeals court said the stockholders failed to allege sufficient facts to support a finding that the company’s CEO David Lucchino and its Chief Development Officer Carl LeBel knew of, or recklessly disregarded, the falsity of statements they made about a phase IIa trial of the company’s hearing loss candidate FX-322 before interim results were available.

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