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Home » Authors » Randy Osborne

Articles by Randy Osborne

US FDA says yes to Lexicon’s Inpefa in HF

May 27, 2023
By Randy Osborne
Eye and financial charts

Annexon sights BCVA benefit in missed-endpoint GA phase II

May 25, 2023
By Randy Osborne
On the strength of upside in best corrected visual acuity (BCVA) in patients with geographic atrophy (GA), Annexon Inc. officials will meet with the U.S. FDA to talk about what’s next for ANX-007, after the C1q inhibitor missed the phase II trial’s primary endpoint: mean rate of change, or slope, in the GA lesion area compared to sham.
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Central nervous system

PTC stays the course after phase III Friedreich’s miss; sky clearer for Skyclarys?

May 24, 2023
By Randy Osborne
“We clearly see an active drug here,” PTC Therapeutics Inc. CEO Matthew Klein said of the 15-lipoxygenase inhibitor vatiquinone for Friedreich’s ataxia (FA), tested in a phase III study called Move-FA that missed the primary endpoint of statistically significant change in modified FA Rating Scale score at 72 weeks. The company will “take one step at a time” decisions about the drug, analyzing the results and then consulting with the U.S. FDA regarding how to proceed, he said. Meanwhile, Wall Street wasn’t happy, and South Plainfield, N.J.-based PTC’s shares (NASDAQ:PTCT) closed May 24 at $46.95, down $11.46, or 19.6%.
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Ayvakit product image

Following the Blueprint for expansion-ISM: FDA says yes to widened label in rare mast-cell disease

May 23, 2023
By Randy Osborne
Blueprint Medicines Corp. scored a broader label from the U.S. FDA for Ayvakit (avapritinib), which became the first approved therapy to treat adults with indolent systemic mastocytosis (ISM).
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Handshake with dollar sign and checkmark

Gattex pragmatics drive Ironwood’s $1B buyout of Vectivbio

May 22, 2023
By Randy Osborne
Once weekly vs. once daily injections plus increased potency vs. Gattex (linaclotide) inspired Ironwood Pharmaceuticals Inc. in its $1 billion takeover of Vectivbio Holding AG, which brings aboard the phase III-stage synthetic glucagon-like peptide-2 analog apraglutide, potentially a best-in-class agent for short bowel syndrome with intestinal failure.
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DILI pickle sours adcom vote on Intercept’s bid with OCA; ‘no read-across’ in NASH space?

May 19, 2023
By Randy Osborne
Trouble presaged by U.S. FDA concerns over potential drug-induced liver injury (DILI) caused by obeticholic acid (OCA) 25 mg came to pass during the Gastrointestinal Drugs Advisory Committee meeting May 19 on Intercept Pharmaceuticals Inc.’s accelerated approval effort with the compound.
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Darzalex specs checked in AL amyloidosis as others busy with would-be challengers

May 18, 2023
By Randy Osborne
Word from Immix Biopharma Inc. of updated data due with NXC-201 brought to the forefront an ongoing push by drug developers to come up with a treatment for AL amyloidosis. Immix has the only CAR T therapy in the works for the disease, and the principal investigator in the Nexicart-1 phase Ib/IIa effort is slated to speak May 19 during the annual meeting of the American Society of Gene & Cell Therapy.
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Coming through in PKU: PTC’s sepiapterin weighed against Kuvan post-phase III

May 17, 2023
By Randy Osborne
With PTC Therapeutics Inc.’s positive results in phase III with sepiapterin for pediatric and adult patients with phenylketonuria (PKU), attention turned to the would-be showdown with Kuvan (sapropterin dihydrochloride), the drug from Biomarin Pharmaceuticals Inc. that was approved in December 2017.
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Sarepta win in DMD fuels speculation about other prospects

May 16, 2023
By Randy Osborne
Sarepta Therapeutics Inc.’s balloting March 12 from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (OTAT) in favor of gene transfer therapy SRP-9001 (delandistrogene moxeparvovec) in Duchenne muscular dystrophy (DMD) had Wall Street mulling the odds for others in the space.
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Eye and DNA illustration

Series A backers see Ray of hope; $100M for retinal diseases

May 16, 2023
By Randy Osborne
Ray Therapeutics Inc.’s upsized and oversubscribed $100 million series A financing will support the firm’s ongoing efforts with optogenetics, an approach that deploys adeno-associated virus (AAV) gene therapy to deliver a light-sensitive, highly bioengineered protein found in nature to retinal cells.
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