Adults with generalized myasthenia gravis (gMG) have yet another therapeutic option with the U.S. FDA’s approval of orphan drug Rystiggo (rozanolixizumab-noli), a humanized IgG4 monoclonal antibody that binds to the neonatal Fc receptor (FcRN). The priority review clearance, which is for patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive, comes on the heels of a Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc, Argenx SE and Halozyme Therapeutics Inc.) nod on June 21. That approval, however, is for anti-AChR-positive patients, making Rystiggo the only treatment approved in the U.S. for both anti-AChR- and anti-MuSK-positive patients. It is expected to be available in the third quarter of 2023 and is under review in Europe and Japan. Like Vyvgart Hytrulo, Rystiggo is an injectable subcutaneous formulation that could take market share from Argenx’s Vyvgart, which was approved in the intravenous form as the first FcRN antagonist for gMG in December 2021.

No longer in the rough: Black Diamond posts strong phase I cancer data

The dose-escalation portion of Black Diamond Therapeutics Inc.’s phase I study of BDTX-1535 for treating non-small-cell lung cancer (NSCLC) produced strong data and moved the market. The company claims this the first EGFR tyrosine kinase inhibitor to produce radiographic responses in NSCLC patients whose cancers are driven by diverse mutation families including acquired resistance mutations after osimertinib therapy and in those with cancers driven by classical and intrinsic driver mutations. The company’s stock (NASDAQ:BDTX) had doubled twice at midday, with shares up 220% to about $6 each.

IL-17 stays hot post-Lilly takeover as Moonlake wins in phase II HS bid

The IL-17 space is seeing plenty of action lately as news of Eli Lilly and Co.’s $2.4 billion buyout of Dice Therapeutics Inc. was followed more recently by favorable data from Moonlake Immunotherapeutics Inc. from the global phase II trial called Mira evaluating the efficacy and safety of the Nanobody sonelokimab in moderate to severe hidradenitis suppurativa. Sonelokimab, which consists of three heavy-chain domains covalently linked by flexible glycine-serine spacers, binds to IL-17A and IL-17F. The study findings were disclosed June 25, and shares of Moonlake (NASDAQ:MLTX) have risen 91% in the past five days. They were trading midday at $50.46, up $4.51.

Mendus to evaluate vididencel-oral-aza combo as maintenance therapy in AML

Later this year, Mendus AB plans to move its allogeneic cell-based cancer vaccine, vididencel, into a phase II combination trial with oral azacitidine to evaluate the regimen’s potential as a maintenance therapy in patients with acute myeloid leukemia (AML). The latter agent is the only approved maintenance therapy for AML patients who have already achieved complete remission after an initial cycle of intensive chemotherapy. These patients, particularly those with minimal residual disease, remain at high risk of relapse, however. Compared with placebo, oral azacitidine was recently shown to prolong overall survival and relapse-free survival by 9.9 months and 5.3 months, respectively. “It has not led to a durable response – it doesn’t lead to cure,” Jeroen Rovers, chief medical officer at Mendus, told a conference call audience. “We feel that this combination could benefit patients even more.”

Japan’s MHLW gives nod to Taiho’s FGFR inhibitor Lytgobi for biliary tract cancer

Japan’s Ministry of Health, Labour and Welfare approved Taiho Pharmaceutical Co. Ltd.’s Lytgobi (futibatinib) for unresectable biliary tract cancer harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions that has progressed after chemotherapy. An irreversible FGFR inhibitor from a series of small-molecule inhibitors of FGFR-1, -2, -3 and -4, Lytgobi (TAS-120) covalently binds to FGFR-2 and inhibits the signaling pathway. Other approved FGFR inhibitors on the market are reversible ATP-competitive inhibitors.

Afterthought or proof of efficacy? Adcom to ponder Ipsen post hoc analyses

After nearly 10 years in clinical development and just a few months after an EMA rejection, Ipsen SA will be making its case tomorrow before a U.S. FDA advisory committee for its ultra-rare bone disease drug, palovarotene. Much of the discussion at the Endocrinologic and Metabolic Drugs Advisory Committee meeting will focus on the use of post hoc analyses to support efficacy and the interpretability of trial results using a natural history study in fibrodysplasia ossificans progressiva as an external control. The EMA wasn’t impressed with the post hoc analyses, saying they were “neither scientifically nor clinically justified.” Safety concerns also will be part of the conversation.

Eubiologics to file for COVID-19 vaccine approval in Philippines with positive interim phase III data

Eubiologics Co. Ltd. said it would file for regulatory approval for its COVID-19 vaccine called Eucorvac-19 in the Philippines, after announcing positive interim phase III trial results on June 27. The latest data readout turns up the heat in a crowded COVID-19 vaccine arena dominated by big pharma and challenged by new entrants like rising Korean biopharma companies like Eubiologics. News sent the firm’s (KOSDAQ:206650) shares up 3.76% from the day prior, trading at ₩7,720 (US$5.94) on market closing of June 27.

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