Logic B o Phase 1/2 Trial Put on Hold

 LogicBio Therapeutics, Inc. (Nasdaq: LOGC), a clinical-stage genetic medicine company, today provided an update on the LB-001 clinical development program. The U.S. Food and Drug Administration (FDA) has notified the company that its Phase 1/2 SUNRISE clinical trial of LB-001 in pediatric patients with methylmalonic acidemia (MMA) has been placed on clinical hold. The company will host a conference call and webcast this morning to discuss this update.

To date, four patients have been dosed in the SUNRISE trial with LB-001, LogicBio's investigational, single-administration, adeno-associated virus (AAV) genome editing therapy. In accordance with the FDA-cleared protocol, the first two patients dosed were in the older age group (3 to 12 years old) and received 5 x 1013 vg/kg of LB-001. These first two patients are doing well, have not experienced drug-related serious adverse events (SAEs), and are being monitored in accordance with the protocol.

As previously disclosed, the third patient dosed in the SUNRISE trial, who received 5 x 1013 vg/kg of LB-001 and is in the younger age group (6 months to 2 years old), experienced a drug-related SAE, which was categorized as a case of thrombotic microangiopathy (TMA). TMA has been previously reported in association with other AAV genetic therapies. The patient was hospitalized and responded well to intravenous fluids and parenteral nutrition. Following this SAE, the company implemented additional safety measures in the SUNRISE trial, and reported the SAE to the FDA and the Data Safety Monitoring Board for the trial (DSMB). In December 2021, the company announced that the SAE experienced by the third patient had resolved.

In January 2022, the fourth patient dosed in the SUNRISE trial, who received 5 x 1013 vg/kg of LB-001 and is in the younger age group, experienced a drug-related SAE, which was categorized as a case of TMA. The patient is being closely followed by the patient's care team and has been steadily improving. The company reported the SAE to the FDA and the DSMB. The FDA subsequently notified the company that the SUNRISE trial has been placed on clinical hold. LogicBio will be working closely with the FDA and the DSMB to determine the next steps for the SUNRISE trial and the LB-001 program.

Conference Call and Webcast Details

LogicBio will host a conference call and webcast today, Wednesday, February 2, 2022, at 8:30 a.m. ET to discuss the program update. To listen to the conference call, please dial +1 (833) 519-1335 (domestic) or +1 (602) 585-9978 (international) using conference ID number 7545016. A live webcast of the call can be accessed via the Investors section of the company's website at https://investor.logicbio.com. A webcast replay will be available following the call and archived for approximately 30 days.

https://finance.yahoo.com/news/logicbio-therapeutics-provides-lb-001-110000095.html

Intuitive Surgical upped to Buy from Neutral by UBS

 Target to $325 from $317

https://finviz.com/quote.ashx?t=ISRG

Novo Nordisk shares rise on upbeat outlook

 Shares in Novo Nordisk rose more than 3% on Wednesday after the Danish drug developer delivered a better-than-expected earnings outlook for 2022 despite missing fourth quarter operating profit expectations.

The world's largest diabetes drug developer expects to generate sales growth of between 6% and 10% in local currencies in 2022 and deliver operating profit growth of 4% to 8%.

"This reflects an underlying unchanged positive growth momentum for Novo Nordisk," Sydbank analyst Soren Lontoft told Reuters, adding that he would be raising his estimates.

Jefferies analysts said Novo's operating profit forecast reflected lower margin pressure than feared.

Novo reported fourth quarter operating profit of 13.63 billion Danish crowns ($2.06 billion), an increase of 16% over the corresponding period last year, but below an average of 14.36 billion forecast by analysts in a Refinitiv poll.

Higher sales and distribution costs in addition to higher research and development spending had impacted the results, Lontoft said.

"It reflects perhaps a more normalized world with increasing absence of restrictions and COVID-19, which has led to increased marketing spending," he added.

Sales for the fourth quarter, however, came in at 38.3 billion crowns, above analysts' estimates of 37.9 billion, driven by sales of new diabetes drug, Ozempic, which rose 64% to 33.7 billion in 2021.

OVERCOMING SUPPLY ISSUES

Novo was overwhelmed by the demand for its Wegovy obesity drug following its release to the U.S. market in June, leading to product shortages.

In December, a contract manufacturer halted deliveries and manufacturing temporarily after issues relating to good manufacturing practice

"Since December, we have worked hard to optimise our internal capacity," Chief Executive Lars Joergensen told reporters on Wednesday.

"Consequently, we now expect that internal capacity in the first half of 2022 will be close to the demand of around 20,000 weekly total scripts as seen in the U.S. market at the end of 2021," Joergensen added.

https://finance.yahoo.com/news/novo-nordisk-posts-q4-operating-064425977.html

World's 1st trial deliberating infecting young adults with Covid found to be safe

 The world's first human challenge trial in which volunteers were deliberately exposed to Covid-19 to advance research into the disease was found to be safe in healthy young adults, one of the companies running the study said on Wednesday.

The data supports the safety of this model which could theoretically provide a "plug and play" platform for testing therapies and vaccines using the original Covid-19 strain as well as variants of the virus, Open Orphan (ORPH.L), which carried out the study, said in a statement.

Open Orphan is running the project, launched a year ago, with Imperial College London, the UK government's vaccines task force and the clinical company hVIVO.

The trial infected 36 healthy male and female volunteers aged 18-29 years with the original SARS-CoV-2 strain of the virus and closely monitored them in a controlled quarantined setting. They will be followed up for 12 months after discharge from the quarantine facility.

No serious adverse events occurred, and the human challenge study model was shown to be safe and well tolerated in healthy young adults, the company said.

With the model established, it said it should be able to contract or conduct Covid-19 human challenge studies in 2022, subject to individual ethics and regulatory approvals.

https://www.gulf-times.com/story/709138/World-s-1st-trial-deliberating-infecting-young-adu

CMS' Biogen decision could spell problems for Lilly, Roche Alzheimer's drugs, half of surveyed neurologists say

 Neurologists in the U.S. are agreeing with a new draft decision from the Centers for Medicare & Medicaid Services (CMS) that restricts reimbursement of Biogen’s controversial Alzheimer’s disease drug, Aduhelm, to Medicare patients enrolled in approved clinical trials only. In fact, half of those surveyed said CMS' decision would hit how they prescribe the rival drugs waiting in the wings that are also included in CMS' decision.

The CMS verdict was handed down Jan. 11, and, that week, Spherix sent out a survey to 75 neurologists and conducted follow-up calls to feel out the mood.

The decision, which is yet to be finalized, “could effectively spell the end for Aduhelm,” RBC Capital Markets analyst Brian Abrahams said in a note to clients mid-January.

That’s because older people covered by Medicare make up the bulk of the Alzheimer’s patient population, and restrictive access for them would be a serious blow to Aduhelm’s market potential, which had been pegged by analysts at Evaluate as reaching $4.9 billion at peak.

CMS' draft decision also applies to other anti-amyloid monoclonal antibodies (mAbs) still in development, including Eli Lilly’s donanemab, Roche’s gantenerumab and Biogen’s follow-up mAb, lecanemab, which is in development with Eisai.

Lilly is the most well known drug on the horizon, according to Spherix's report based on its survey. “Among the three late-phase emerging anti-amyloid mAbs in development for Alzheimer’s disease, unaided awareness is highest with Lilly’s donanemab,” the report notes.

Yet, around half of those surveyed said that should the CMS’ decision stick and be held for other mAbs, this would negatively impact their prescribing behavior. “One in two neurologists [48%] anticipate that CMS’ proposed NCD decision being finalized as such would have a negative impact on their willingness to prescribe donanemab as well as the other anti-amyloid mAbs lecanemab and gantenerumab.”

The CMS decision is expected to get finalized by April and could yet be changed, but that could be a big hit for these drugs, with donanemab and gantenerumab expected to hit 2026 sales of $6 billion and $2.5 billion, respectively, according to estimates by Evaluate made prior to the CMS decision.

There was overall a “lack of consensus” from neurologists when it came to these drugs given how similar they are to Aduhelm, though most are waiting for more data to make a decision as to whether they should be used and reimbursed.

One neurologist said to Spherix: “If Lilly had very strong data on clinical benefit [with donanemab], not just clearing of amyloid, and on the side-effect profile, I hope that CMS would change its decision for that particular drug.”

Biogen is working on a confirmatory trial for Aduhelm, a mandate of its FDA accelerated approval, though results are not expected soon. In December 2021, Biogen said it anticipates starting to screen patients this May and that the trial would likely take around four years to complete. Lilly, meanwhile, expects to have a first readout from its ongoing trial by 2023 and is also running a head-to-head against Aduhelm, with a readout slated for year-end.

“Given neurologists’ mixed opinions, it is perhaps not surprising that they believe that positive phase 3 data on cognitive and functional outcomes with acceptable safety from ongoing trials with late-phase agents would likely motivate CMS to loosen coverage in a revised reimbursement decision,” the report said.

Overall, the neurologists agreed with the CMS that Aduhelm should be restricted, with 55% agreeing and 33% neutral (while 15% disagreed), and generally saw the CMS in a positive light. The same could not be said for the FDA, however.

The agency approved Aduhelm nearly one year ago with what was, initially, a very broad label despite there being serious questions over efficacy and safety. In fact, in November 2020, the drug was rejected by the FDA’s expert advisory panel.   

Since then, the FDA narrowed Aduhelm's label, and Biogen ended up slashing the drug's price in half from $56,000 at launch. But the damage was already done. “Neurologists’ confidence in the agency has been eroded in the past year,” the report found.

Conversely, their confidence in CMS has remained on par (or has improved) over the past year.

A neurologist interviewed by the firm, who was not named, said: “The FDA is very concerning. The [AdComm] recommended against [Aduhelm's approval], and then the FDA went around and said yes, so you lose a lot of confidence right now.

“My confidence in CMS is much better, much improved. I think they did the right thing. I think this is necessary. Too expensive and too potentially harmful to just willy-nilly start using these medications. We need to know definitively that these are really beneficial.”

 The CMS will take comments on its decision through Feb. 10.

https://www.fiercepharma.com/marketing/half-neurologists-unlikely-to-prescribe-lilly-roche-alzheimer-s-drugs-if-cms-biogen

Novartis' Sandoz reportedly the target of $25B buyout bid from Carlyle and Blackstone

 As Novartis reviews its generics business, the list of potential Sandoz suitors has grown. The deal could become one of the biggest generic industry acquisitions ever—assuming an offer makes it across the finish line.

Investor groups Blackstone and Carlyle could join forces on a massive $25 billion bid for Sandoz, Bloomberg reports, citing people close to the matter. The private equity firms are discussing a potential joint offer for the Novartis unit, the publication said. Separately, Advent International, Hellman & Friedman and KKR & Co. are sizing up their own approaches, Bloomberg’s anonymous sources said.

Sandoz is a sizable target, so it could make sense for investor groups to merge their bids. But it’s early yet, and the suitors may decide against teaming up or proceeding with bids altogether, Bloomberg said.

The interest in Sandoz comes as Novartis takes a magnifying lens to the business. In October, the Swiss drugmaker launched a strategic review of its generics arm, leaving all options on the table. Novartis said it would provide an update by the end of 2022.

It’s not unusual for private equity firms to unite on the hunt for big acquisitions, Bloomberg notes. Blackstone, Carlyle and Hellman & Friedman in June agreed to buy medical supply company Medline Industries for a whopping $30 billion, for example. Hellman & Friedman also partnered with Bain Capital in November to snap up medical IT outfit Athenahealth for $17 billion.

Meanwhile, Sandoz has already attracted some interest. In November, German newspaper Handelsblatt reported that Swedish-based investment group EQT and Germany’s Struengmann family were weighing a joint move to buy the Novartis unit for $21.6 billion. Interestingly, Struengmann twins Thomas and Andreas are no strangers to Novartis, having sold it their generics maker Hexal back in 2005. They’re also primary investors in Pfizer’s COVID-19 vaccine partner BioNTech.

That same month, Novartis CEO Vas Narasimhan confirmed interest in the company: “There have been various requests for more information, but no concrete offers,” he told German magazine WirtschaftsWoche. 

Whether it’s $21 billion or $26 billion, a successful Sandoz buyout could eclipse every other M&A deal from last year. 2021 saw a relative lull in merger and acquisitions activity by drugmakers—though 2022 seems poised for a rebound.

https://www.fiercepharma.com/pharma/novartis-sandoz-reportedly-target-26b-buyout-bid-from-carlyle-and-blackstone

Novartis forecasts 2022 sales and profit growth; Sandoz review continues

 Novartis forecast its sales and core operating profit would grow at a mid-single-digit rate this year, as the Swiss pharmaceuticals group nears a decision on whether to keep or sell its generics business Sandoz.

The pharmaceuticals company in October raised the prospect of divesting Sandoz after years of revamping the business, as price pressures mount in the off-patent drug sector.

In its earnings statement on Wednesday, Novartis reiterated that it would provide an update by the end of 2022 as it explores a possible sale but also an option to retain the off-patent drugs unit.

Media reports have cited interest from private equity firms, in particular and Chief Executive Vas Narasimhan has said the asset was attracting interest from various suitors.

In a media call on Wednesday, the CEO said all options were still on the table.

"We don’t have a bias towards any of these options at the moment. We are doing the work to finish the carve-out financials to provide that to relevant parties and we’ll see what proposals come back," the CEO said.

Core operating income for the fourth-quarter ended Dec. 31 gained 9% to $3.8 billion, as higher drug sales offset in increase in marketing and development costs.

Revenues from arthritis and psoriasis drug Cosentyx gained 13% to $1.24 billion, slightly below average analyst expectations of $1.3 billion, based on Refinitiv data.

Novartis' revenues from heart failure treatment Entresto jumped 34% to $949 million, broadly in line with the market consensus.

It forecast Sandoz sales would be broadly in line with the 2021 level of $2.5 billion, while the division's core operating income was expected to fall at a low-to-mid-single-digit rate.

Despite plans to buy back up to $15 billion worth of shares until the end of next year, Novartis has said it would retain enough spending power to buy companies and technologies, back its own research efforts and pay attractive dividends.

It proposed raising its dividend 3.3% to 3.10 Swiss francs ($3.37) per share, the 25th consecutive increase since its creation.

https://www.marketscreener.com/quote/stock/NOVARTIS-AG-9364983/news/Novartis-forecasts-2022-sales-and-profit-growth-Sandoz-review-continues-37717327/