Outset Medical receives FDA warning letter

 Outset Medical (Nasdaq: OM)

 announced that it received a warning letter from the FDA listing observations related to its Tablo system.

San Jose, California-based Outset previously disclosed an FDA form-483 on its annual report in February. That FDA form identified four inspectional observations resulting from an inspection concluded on Feb. 10, 2023. From there, Outset provided a response plan on March 3 and completed remediation workstreams to address the observations.

A new warning, dated July 5, raises two additional observations. Outset issued a press release outlining the new warning on July 7. As the markets opened today (July 10), shares of OM fell 14% at $17.60 apiece.

First, the FDA asserts that certain materials it reviewed (found on Outset’s website) promote continuous renal replacement therapy (CRRT). That modality falls outside the current indications for the Tablo hemodialysis system. Outset said it believes it effectively addressed this concern through labeling and promotional changes already underway.

The second observation asserts that TabloCart with prefiltration requires prior 510(k) clearance for marketing authorization. TabloCart, a Tablo accessory, launched in the third quarter of 2022. Sales have remained immaterial to Outset’s financial results. Outset said it intends to work collaboratively with the FDA to resolve this, with steps potentially including a 510(k) submission.

According to the news release, the warning letter does not request the restriction of Tablo’s manufacturing, production or shipment in the U.S. It also does not request the withdrawal of the system from the U.S. market.

Outset said it intends to fully cooperate with the FDA, including a response within 15 business days, to resolve the warning letter. However, it can’t give assurances of the FDA’s satisfaction with the actions it takes in response to the letter. Outset also offers no assurances as to the timing of any such resolutions.

Another hurdle to clear for Outset Medical?

In June 2022, Outset put a hold on shipments of the Tablo hemodialysis system for home use. The hold came as a result of a pending FDA review and clearance of a 510(k) submitted for changes made since the Tablo system’s original March 2020 clearance.

A month later, a shareholder lawsuit came forward claiming that the company and executives made false and misleading positive statements leading up to the shipment hold announcement. In August of 2022, the company said that it received the FDA 510(k) clearance and resumed Tablo shipments.

Shortly after the company resumed shipments, a class action securities lawsuit came through against Outset.

Despite the issues surrounding Tablo, following the resumption of shipments, Outset’s performance hit an upturn. The company secured a $300 million debt financing and landed on Deloitte’s list of fastest-growing companies. It also produced a string of strong financial performances, culminating in a Street-beating first quarter to open 2023.

https://www.massdevice.com/outset-medical-receives-fda-warning-letter/

NanoString: Positive Q2 Prelims

 Record Q2 Revenue of Over $44 Million, 37% Year on Year Growth and Above Upper End of Q2 Guidance Range -

- New Orders for CosMx Instruments Grew Sequentially and Year on Year -

NanoString Technologies, Inc. (NASDAQ:NSTG), a leading provider of life science tools for discovery and translational research, today reported preliminary financial and operational highlights for the second quarter ended June 30, 2023.

"We are excited to report that our teams delivered strong preliminary revenue results for the second quarter, with preliminary results exceeding consensus estimates and the upper end of our guidance ranges," said Brad Gray, President and CEO of NanoString. "Customers are focused on the potential for spatial analysis to advance science, and value the RNA-plex and protein capabilities our spatial platforms provide. During Q2, a competitor launched a campaign using a ruling in the Munich regional court to try to frighten customers to cancel orders for our CosMx Spatial Molecular Imager, even though the preliminary ruling was limited to Germany. Despite this challenge, through the strength of our technology and the hard work of our team, we fulfilled or retained approximately 97% of our cumulative CosMx instrument orders and captured orders for new CosMx instruments at a faster pace than in Q1."

https://finance.yahoo.com/news/nanostring-provides-preliminary-financial-operational-090000289.html

Biogen shares 'an under-owned turnaround story,' Jefferies analysts say

 Shares of Biogen Inc. (BIIB) , which last week received a milestone U.S. Food and Drug Administration approval for Alzheimer's treatment Leqembi, "remain an under-owned turnaround story," and the stock should benefit as sales gradually ramp up, Jefferies analysts wrote in a research note Sunday. The approval failed to give Biogen stock an immediate boost, as some analysts predicted a slow rollout for Leqembi and bottlenecks at infusion-therapy centers. But infusion center capacity is "not overly concerning," the Jefferies analysts wrote, and doctors are receptive to getting patients on Leqembi. The analysts rate Biogen shares a buy, with a $350 price target. Biogen stock is up 0.5% premarket on Monday.

https://www.morningstar.com/news/marketwatch/20230710135/biogen-shares-an-under-owned-turnaround-story-jefferies-analysts-say

EU probes Novo Nordisk drugs after reports of suicidal thoughts

 The European Medicines Agency (EMA) is probing Novo Nordisk's diabetes drug Ozempic and weight-loss treatment Saxenda after Iceland's health regulator flagged three cases of patients on the drugs thinking about suicide or self-harm.

Shares of the Danish drugmaker fell about 1.5% on Monday following the news.

An EMA safety committee is looking at adverse events raised by the Icelandic Medicines Agency, including two cases of suicidal thoughts potentially linked to Ozempic and Saxenda, the regulator said.

One additional patient on Saxenda reported thoughts of self-injury, the agency said.

The EMA's investigation is centered around medicines that contain the active ingredients, semaglutide or liraglutide, for weight loss.

The review comes weeks after the regulator had raised a thyroid cancer safety signal over several of Novo's products that contain semaglutide, the active ingredient used in diabetes drug Ozempic and obesity treatment Wegovy, which has seen soaring demand in the United States. Saxenda contains liraglutide.

Suicidal thoughts are not currently listed as a side effect in the EU product information of these treatments.

Side effects have hobbled several previous attempts by the drug industry to develop lucrative weight-loss drugs.

Sanofi's Acomplia, which never won a U.S. approval, was withdrawn in Europe in 2008 after being linked to suicidal thoughts.

Acomplia was designed to modify parts of the nervous system that regulate appetite. New weight-loss drugs such as Wegovy, however, regulate appetite by mimicking a gut hormone, and not directly interfere with brain chemistry.

The EMA said on Monday it would consider whether the review should be extended to include other medicines of the same class known as GLP-1 receptor agonists.

Patient safety is a top priority and the company takes all reports about adverse events very seriously, Novo Nordisk said.

Safety data did not show any "causal association" between the suicidal or self-harming thoughts and the drugs, it added.

https://www.yahoo.com/news/eu-probes-novos-weight-loss-110433807.html

Uptake of Leqembi for Alzheimer’s may be slow

 Full FDA approval of Eisai and Biogen’s Alzheimer’s disease therapy Leqembi may have unlocked the door to a broader rollout of the drug and potentially blockbuster sales, but commentators think uptake could initially be slow.

The regulatory milestone means that Medicare will cover most of the cost of the $26,500 per year anti-amyloid therapy, giving patients the option of accessing a disease-modifying drug for Alzheimer’s for the first time outside of a clinical trial.

The Centers for Medicare and Medicaid Services (CMS) indicated that it will foot the bill for 80% of the drug’s cost through Medicare Part B, which covers drugs administered to older patients in physicians’ offices or hospitals. That means patients will have to pay 20% of the approved amount for Leqembi once they meet their Part B deductible, either out of pocket or through secondary insurance.

The cost will have to be balanced against the modest slowdown in clinical decline that Leqembi (lecanemab) achieved in clinical trials, the risk of uncommon but potentially life-threatening side effects that are listed in a black box warning on its label, and additional healthcare costs stemming from clinic visits and diagnostic tests.

Analysts at Jefferies said the decision by the FDA to use its strongest warning was unexpected and could slow down Leqembi’s sales growth.

The antibody is administered by intravenous infusions in a doctor’s office or clinic every two weeks and will be available for people diagnosed as having early-stage Alzheimer’s, as well as for those with Alzheimer’s-related mild cognitive impairment and amyloid plaques in the brain.

Take-up of treatment could also be limited by access to infusion clinics, which can be patchy across the US, as well as to the PET scans needed to check amyloid levels in the central nervous system before treatment and every few months thereafter.

That could add logistical, as well as financial, barriers for some patients, as there is still some uncertainty around Medicare’s reimbursement policy for those tests, according to analysts at RBC Capital Markets cited by Barron's.

Other potential bottlenecks for treatment include the recommendation for genetic tests to look for the APOE4 gene mutation, as patients with two copies of that are at elevated risk of side effects from the drug, as well as the need to be enrolled in a patient registry that will be used to gauge the effects of Leqembi in a real-world setting over the coming years.

Patients organisations have said they are concerned that access to healthcare providers willing to participate in the registry programme may also be limited, particularly in rural areas and those without large, urban hospital systems.

Eisai – which is taking the lead on the commercialisation of Leqembi – said it has set up a patient assistance programme to provide the drug at no cost to “eligible uninsured and underinsured patients, including Medicare beneficiaries, who meet financial need and other programme criteria.”

It has previously said it expects sales of the drug to reach $7 billion by the end of the decade.

With full approval in hand for Leqembi, eyes are turning to Eli Lilly’s rival amyloid therapy donanemab, which is also gunning for full FDA approval and has the advantage of only needing to be infused once a month. The FDA is expected to decide on donanemab’s approval either later this year or early in 2024.

Another Biogen and Eisai drug – Aduhelm (aducanumab) – was given accelerated approval by the FDA in 2021, but questions about its efficacy led to CMS placing restrictions on its use and it never made any headway in the market.

https://pharmaphorum.com/news/uptake-leqembi-alzheimers-may-be-slow

JPMorgan lowers target for Bayer to 55 euros - 'Neutral'

 U.S. bank JPMorgan cut its price target for Bayer to 55 euros from 60 euros and left its rating at "Neutral." Value in the Leverkusen-based company's shares depends on the turnaround in the pharmaceuticals business, not a spin-off of the agricultural business, analyst Richard Vosser wrote in a research note presented Monday. Vosser is lowering his estimates and fears that annual targets will be trimmed further.

https://www.marketscreener.com/quote/stock/BAYER-AG-436063/news/JPMorgan-lowers-target-for-Bayer-to-55-euros-Neutral-44298433/

Marker: AML Candidate Granted Orphan Drug Designation in EU

 https://www.marketscreener.com/quote/stock/MARKER-THERAPEUTICS-INC-46699740/news/Marker-Therapeutics-Says-Acute-Myeloid-Leukemia-Treatment-Candidate-Granted-Orphan-Drug-Designation-44301139/