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Piper Sandler analyst Adam Maeder points to a link to a PMA supplement dated July 1 that was posted to the FDA website to support his view that Inspire Medical Systems appears to have received approval for expanded labeling of its system to include 1.5 Tesla full-body MRI capabilities, though he also notes that has not been able to connect yet with the company to confirm. The analyst, who has heard multiple clinicians at the SLEEP meeting last month comment that the historical MRI labeling has been a barrier to Inspire Medical therapy adoption, believes some patients may be more inclined to move forward with a procedure with the new approval in-hand. Maeder keeps an Overweight rating and $310 price target on Inspire Medical shares.
Arrowhead Pharmaceuticals announced that it has dosed the first subjects in two Phase 1/2a clinical trials of ARO-MUC5AC and ARO-RAGE, the company's investigational RNA interference therapeutics designed to reduce production the of mucin 5AC and the receptor for advanced glycation end products , respectively, as potential treatments for various muco-obstructive and inflammatory pulmonary diseases. "Initiating clinical studies for ARO-MUC5AC and ARO-RAGE, Arrowhead's next generation pulmonary targeted RNAi therapeutic candidates, is a significant milestone for the company," said Chris Anzalone, Ph.D., president and chief executive officer of Arrowhead. "These are potentially important new medicines that address targets that have been difficult to drug using other modalities and are designed to treat muco-obstructive and inflammatory lung diseases in fundamentally new ways." AROMUC5AC-1001 is a Phase 1/2a, randomized, double-blinded, placebo-controlled study in up to 42 normal healthy volunteers and up to 16 patients with moderate-to-severe asthma. The single ascending dose portion of the study includes 3 sequentially enrolled NHV cohorts with escalating single-dose levels. The multiple ascending dose portion of the study includes 3 NHV cohorts and 2 asthma patient cohorts. The objectives of the study include the assessment of safety and tolerability, pharmacokinetics, and pharmacodynamics of ARO-MUC5AC in NHVs and patients with asthma.
Guggenheim analyst Debjit Chattopadhyay downgraded Alnylam to Neutral from Buy with no price target, telling investors that proprietary analyses, complemented by talks with key opinion leaders, point to "uncertainties in ATTR-CM" and "a challenged outlook" for the APOLLO-B trial. Chattopadhyay sees an unfavorable risk/reward given the view that the APOLLO-B outcome is "a coin flip," but hopes to be wrong with the cautious outlook as "we are believers in the superiority of silencers over stabilizers," the analyst tells investors.
Takeda (TAK) and Arrowhead Pharmaceuticals (ARWR) announced that results from a Phase 2 clinical study of investigational fazirsiran for the treatment of liver disease associated with alpha-1 antitrypsin deficiency, AATD, were recently published in the New England Journal of Medicine, NEJM, and presented in an oral presentation at The International Liver Congress 2022 - The Annual Meeting of the European Association for the Study of the Liver, EASL. The NEJM article was published online ahead of print and is titled, "Fazirsiran for Liver Disease Associated with Alpha-1 Antitrypsin Deficiency." The EASL presentation was titled, "Reduction of Intra-hepatic Z-AAT Synthesis by Fazirsiran Decreases Globule Burden and Improves Histological Measures of Liver Disease in Adults with Alpha-1 Antitrypsin Deficiency." Fazirsiran is a potential first-in-class investigational RNA interference therapy designed to reduce the production of mutant alpha-1 antitrypsin protein as a potential treatment for the rare genetic liver disease associated with AATD. Z-AAT accumulation is believed to be the cause of progressive liver disease in patients with AATD. "The exciting data on fazirsiran treatment from the open label AROAAT-2002 Phase 2 study in patients with AATD liver disease suggest a treatment effect and the potential to improve multiple downstream markers of liver health. We are also nearing completion of the Phase 2 SEQUOIA study and we look forward to further assessing the potential of fazirsiran in this larger placebo-controlled study," said Javier San Martin, M.D., Chief Medical Officer at Arrowhead. "Fazirsiran has shown a high level of activity across all patients studied and is representative of how the RNA interference pathway can be leveraged to reliably and consistently silence gene expression and potentially have a positive impact on patients with various genetic diseases."
Piper Sandler analyst Do Kim keeps an Overweight rating on Ionis Pharmaceuticals (IONS) with a $55 price target after the company announced eplontersen met the co-primary and secondary endpoints for the Phase 3 NEURO-TTRansform study in patients with hereditary transthyretin-mediated amyloid polyneuropathy. The company released no detailed data but in a call with management, it confirmed eplonterson efficacy and safety was competitive with Alnylam's (ALNY) Amvuttra, Kim tells investors in a research note. He expects an FDA filing in the second half of 2022 to support approval in hATTR-PN in the second half of 2023.
Citi analyst David Lebowitz opens a "90-day Catalyst Watch" on Alnylam Pharmaceuticals after the company announced approval for Amvuttra for polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. The approval removes one overhang from the shares, and investors can now turn their attention to the upcoming Phase III APOLLO-B update for Onpattro, Lebowitz tells investors in a research note. The analyst believes the trial will report in Alnylam's favor and support FDA approval for the indication. He keeps a Buy rating on the shares with a $211 price target.
Piper Sandler analyst Edward Tenthoff reiterates an Overweight rating and $208 price target on Alnylam, noting that the FDA approved 25mg quarterly subcutaneous AMVUTTRA for the treatment of hereditary ATTR amyloidosis with polyneuropathy based on positive Phase III HELIOSA data. The analyst believes less frequent subcutaneous AMVUTTRA will cannibalize some intravenous ONPATTRO, but still grow the overall market for Alnylam. Alnylam maintained guided 2022 product sales of $870M-$930M, and Tenthoff inched up his forecast to $867M due to earlier approval and higher than anticipated price. The primary driver for Alnylam shares remains Phase III APOLLO-B data on ONPATTRO in mid-2022, he adds.
Alnylam Pharmaceuticals "announced that the FDA approved AMVUTTRA, or vutrisiran, an RNAi therapeutic administered via subcutaneous injection once every three months for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. The FDA approval is based on positive 9-month results from the HELIOS-A Phase 3 study, where AMVUTTRA significantly improved the signs and symptoms of polyneuropathy, with more than 50 percent of patients experiencing halting or reversal of their disease manifestations. The FDA approval of AMVUTTRA is based on positive 9-month results from HELIOS-A, a global, randomized, open-label, multicenter, Phase 3 study that evaluated the efficacy and safety of AMVUTTRA across a diverse group of patients with hATTR amyloidosis with polyneuropathy. 164 patients with hATTR amyloidosis were randomized 3:1 to receive either 25 mg of vutrisiran via subcutaneous injection once every three months or 0.3 mg/kg of patisiran via intravenous infusion once every three weeks for 18 months. The efficacy of AMVUTTRA was assessed by comparing the AMVUTTRA group in HELIOS-A with the placebo group from the landmark APOLLO Phase 3 study of patisiran, a randomized controlled study in a comparable patient population."
Piper Sandler analyst Do Kim raised the firm's price target on Ionis Pharmaceuticals (IONS) to $55 from $54 and keeps an Overweight rating on the shares. The analyst updated his model following the firm's conversation with management, saying the cadence of his quarterly estimates better aligns with Ionis expectations, with Q2 revenues similar to Q1 and H2 revenues more weighted to year-end. Kim added that he believes Alynylam's (ALNY) Onpattro APOLLO-B data in ATTR-Cm expected in mid-year 2022 could have potential read through to Ionis shares.
Truist analyst Richard Newitter lowered the firm's price target on Inspire Medical to $250 from $296 but keeps a Buy rating on the shares. His discussion with sleep specialists and ENT Inspire users at this week's SLEEP 2022 event have left him positive on Inspire volume trends and the hypoglossal nerve stimulation awareness, the analyst tells investors in a research note. Newitter adds that he remains positive on the company and its "durable" 30%-45% revenue growth with 85% or more gross margin profile.
