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Tuesday, January 18, 2022

Dyne Therapeutics: FDA Clinical Hold on Application for DYNE-251 in Duchenne

  Dyne Therapeutics, Inc. (Nasdaq: DYN), a muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that the U.S. Food and Drug Administration (FDA) has placed on clinical hold its Investigational New Drug (IND) application to initiate a clinical trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) amenable to skipping exon 51. Dyne received a clinical hold letter from the FDA on Friday, January 14, 2022 requesting additional clinical and non-clinical information for DYNE-251. The Company expects to submit to the FDA its response with data from existing and ongoing studies in the second quarter of 2022, and if satisfactory to the FDA, to be dosing patients in a Phase 1/2 multiple ascending dose (MAD) clinical trial of DYNE-251 by mid-2022 in accordance with its current guidance. Dyne will work closely with the FDA to resolve the clinical hold as promptly as possible.

As previously announced, the Company expects to submit an IND for DYNE-101 in myotonic dystrophy type 1 (DM1) during the first quarter of 2022 and to be dosing patients in a planned MAD clinical trial by mid-2022.

https://finance.yahoo.com/news/dyne-therapeutics-announces-fda-clinical-130000899.html

MorphoSys Reports Preliminary 2021 Monjuvi U.S. Sales and 2022 Guidance

 

  • Preliminary 2021 Monjuvi U.S. net product sales of US$ 79.1 million ( € 66.9 million)

  • Anticipated 2022 Monjuvi U.S. net product sales in the range of US$ 110 to 135 million

MorphoSys AG (FSE:MOR; NASDAQ:MOR) today reported preliminary Monjuvi (R) U.S. net product sales for the full year of 2021 and provided financial guidance for 2022.

Preliminary Monjuvi (tafasitamab-cxix) U.S. net product sales are US$ 23.6 million (€ 20.5 million) for the fourth quarter and US$ 79.1 million (€ 66.9 million) for the full year of 2021. Fourth quarter and full year 2021 results will be published on March 16, 2022.

"We are pleased that many patients have benefitted from Monjuvi since launch and we expect to see continued growth in 2022," said Jean-Paul Kress, M.D., Chief Executive Officer of MorphoSys. "The updated structure of our 2022 financial guidance better reflects our business model and provides greater transparency to the investment community."

Full Year 2022 Financial Guidance:

Amounts in million

2022 Financial Guidance

2022 Guidance Insights

Monjuvi U.S. Net Product Sales

US$ 110m to 135m

100% of Monjuvi U.S. product sales are recorded on MorphoSys' income statement and related profit/loss is split 50/50 between MorphoSys and Incyte.

Gross Margin for Monjuvi U.S. Net Product Sales

75% to 80%

100% of Monjuvi U.S. product cost of sales is recorded on MorphoSys' income statement and related profit/loss is split 50/50 between MorphoSys and Incyte.

R&D expenses

€ 300m to 325m

2022 growth over 2021 will be driven primarily by investment in ongoing pivotal phase-3 studies, excluding transaction/restructuring/other charges related to Constellation acquisition recorded in 2021.

SG&A expenses

 155m to 170m

  • 51% to 56% of mid-point of SG&A expenses represents Monjuvi U.S. selling costs of which 100% are recorded in MorphoSys' income statement. Incyte reimburses MorphoSys for half of these selling expenses.
    For 2022, we anticipate a year-over-year decline in SG&A, excluding transaction/restructuring/other charges related to Constellation acquisition recorded in 2021.

  • Additional information related to 2022 Financial Guidance:

  • Tremfya royalties will continue to be recorded as revenue without any cost of sales in MorphoSys' income statement. These royalties, however, will not contribute any cash to MorphoSys as 100% of the royalties will be passed on to Royalty Pharma.

  • MorphoSys anticipates receiving royalties for Minjuvi (R) sales outside of the U.S. Guidance for these royalties is not being provided as MorphoSys does not receive any sales forecasts from its partner Incyte.

  • MorphoSys does not anticipate any significant cash-accretive revenues from the achievement of milestones in 2022. Milestones for otilimab are passed on to Royalty Pharma. Milestones from all other programs remain with MorphoSys at 100%.

  • MorphoSys anticipates sales of commercial and clinical supply of tafasitamab outside of the U.S. to its partner Incyte. Revenue from this supply is recorded in the "Licenses, milestones and other" category in MorphoSys' income statement. These sales result in a zero gross profit/margin. As such, MorphoSys does not provide guidance for these sales.

  • While R&D expense is anticipated to grow year-over-year due to investments in three pivotal studies, the growth is partially being offset by the consolidation of research/discovery activities.

  • SG&A expense guidance range reflects savings from synergies following the acquisition of Constellation and streamlined commercialization efforts.

  • Anticipated foreign exchange (USD/EUR) to impact operating expenses (R&D and SG&A) negatively by approximately 3%.

NeuroMetrix gets breakthrough tag for Chemotherapy Induced Peripheral Neuropathy device

 NeuroMetrix, Inc. (Nasdaq: NURO) today announced that its Quell® technology has received Breakthrough Designation from the U.S. Food and Drug Administration (FDA) for reducing moderate to severe symptoms of chemotherapy induced peripheral neuropathy that have persisted for at least 6-months following the end of chemotherapy.

About 650,000 cancer patients receive chemotherapy annually in the United States. CIPN is a disabling complication that occurs in many patients treated with common chemotherapeutic drugs such as vincristine, paclitaxel and cisplatin. Symptoms include burning/shooting pain, tingling, cramping, and numbness in the hands and feet. CIPN is also associated with impaired balance, walking, and sleep, decreased quality of life, and increased risk of falls. Approximately 30% of patients experience chronic CIPN, defined as symptoms lasting longer than 3-6 months after the last chemotherapy treatment. There are no FDA approved treatments for CIPN, and those that are used have limited effectiveness and have side effects.

Quell is an advanced, non-invasive, neuromodulation technology that is covered by 19 U.S. utility patents. It is the only wearable neuromodulator that is enabled by a proprietary microchip that provides precise, high-power nerve stimulation in a form factor the size of a credit card. The Quell device utilizes position and motion sensing to automatically adjust stimulation for an optimal patient experience both day and night. The device supports Bluetooth® low energy (BLE) to communicate with smartphone and smartwatch apps that help the patient personalize and control their treatment.

https://finance.yahoo.com/news/neurometrix-receives-fda-breakthrough-device-140000796.html

Monday, January 17, 2022

Canada's vaccine mandate for cross-border truckers is now in effect

 Industry experts and leaders remained concerned about the country's supply chain as the federal government's new vaccine mandate for truck drivers came into effect Saturday following days of confusion around the rules.

The mandate, which will require Canadian truckers to quarantine if unvaccinated when crossing the border into Canada, led to a number of questions and corrections around who would be exempt and how.

Now, with the vaccine requirement in place, concerns persist about the impact this mandate will have on the North American supply chain.

"I think you probably won't see that movement … that the government's looking for," retail expert Bruce Winder told CTV News Channel on Saturday when asked if the effort will encourage truckers to get vaccinated.

The Canadian Trucking Alliance has said between 10 and 15 per cent of cross-border commercial drivers could be lost if the mandate takes effect.

American Trucking Associations has argued that a misapplied mandate would fuel a surge in driver turnover and attrition, with fleets losing as much as 37 per cent of their current workforce.

There are 120,000 Canadians and 40,000 licensed drivers in the U.S. who operate cross-border, the Canadian Trucking Alliance says, while about 70 per cent of the $648 billion in trade between the two countries moves by truck.

Under the vaccine mandate, unvaccinated or partially-vaccinated non-Canadian truckers will be turned away if they aren't able to show proof of vaccination or a valid medical exemption to the COVID-19 vaccines. The U.S. plans to have a similar mandate come into effect for drivers crossing into the country starting Jan. 22.

"I know what the government's trying to do with managing the hospital capacity, but they could find themselves with a very tough situation if Canadians rise up with inflation and food insecurity, or major manufacturers slow down, lay off people," Winder said.

The mandate throws a "major wrench" in the Canadian and North American supply chains, he added, with grocers, food producers, the auto parts industry and building materials among the sectors expected to be most affected.

"I really hope that we're not at the stage where you see food insecurity, where you're actually going to grocery stores and there's nothing on the shelf," Winder said.

"That could be the worst-case scenario."

Mike Millian, president of the Private Motor Truck Council of Canada, told CTV News Channel on Saturday that there were as many 23,000 vacancies at the end of the third quarter of 2021, with his group's own studies showing that roughly 20 per cent of Canadian truck drivers operating across the border are unvaccinated.

Although the Canada Border Services Agency said earlier this week that unvaccinated Canadian truckers would be exempt from having to quarantine or provide proof of a negative COVID-19 molecular test when coming back, the federal government later corrected this and said they would still need to quarantine upon their return.

Millian had hoped unvaccinated Canadian truckers, who were dispatched after the CBSA's announcement, would be granted an exemption from the quarantine rules.

But after speaking with government officials on Friday, Millian told CTV's Power Play that the request had been denied.

However, Canadian drivers will still be allowed to finish their deliveries before heading into quarantine.

"They bring our food in, our heating fuels and our groceries, everything that we need, our medical supplies," Millian said.

"If we remove a fifth of that workforce, we're going to see shortages on shelves and we're going to see inflation of prices, because the cost to bring this stuff here is going to go up."

https://www.ctvnews.ca/canada/canada-s-vaccine-mandate-for-cross-border-truckers-is-now-in-effect-1.5741561

Texas abortion ban remains in effect after appeals court ruling

 A federal appeals court rejected a request from Texas abortion clinics to return their challenge against the state’s controversial abortion ban, leaving in place the strictest ban in the country.

The 5th Circuit Court of Appeals temporarily transferred the case to the Republican-controlled Texas Supreme Court in a 2-to-1 decision, according to The Washington Post, postponing the decision for months. 

The move will allow the Texas abortion ban, which has led to a large reduction in abortions in the state, to remain in place.

The ban restricts access to abortion after about six weeks into pregnancy, when a lot of women do not know they're pregnant yet.

“This court reasonably seeks the Texas Supreme Court’s final word on the matter,” wrote Judge Edith Jones, joined by Judge Stuart Kyle Duncan, nominees of former Presidents Reagan and Trump, respectively. 

However, Judge Stephen Higginson disagreed with his colleagues' "second-guessing redundancy" and said the situation "deepens my concern that justice delayed is justice denied, here impeding relief ordered by the Supreme Court," according to the Post.

Abortion providers said that the latest decision is a tactic to delay the law from being discussed.

The news comes after a December Supreme Court procedural ruling effectively prolonged efforts by the abortion providers to block the law. 

The controversial issue has also crossed state lines, with the Supreme Court discussing a Mississippi abortion ban that bars the procedure after 15 weeks. The debate has led conservative judges to hint at their support for overturning Roe v. Wade.

https://thehill.com/homenews/state-watch/590108-texas-abortion-ban-remains-in-effect-after-appeals-court-ruling

Walensky says she will improve CDC messaging amid criticism

 Centers for Disease Control and Prevention (CDC) Director Rochelle Walensky in a new interview with The Wall Street Journal committed to communicating agency coronavirus pandemic policy more clearly in the future, but defended its recent isolation guidance changes.

Walensky, who is now one year into her tenure as CDC director, said she should have made it more clear to the public that agency guidelines and recommendations can change quickly depending on the nature of the virus.

“I think what I have not conveyed is the uncertainty in a lot of these situations,” Walensky said in the interview, which was published Monday. 

While she vowed to improve communication, Walensky also defended the update to the agency's isolation and quarantine guidance, which shortened the recommended time people should isolate if infected with COVID-19 or quarantine if exposed.

At the end of December, the CDC announced that infected Americans would need to isolate for only five days, not 10, if they were no longer experiencing symptoms or if their symptoms were improving, and that a negative test result would not be required to end the isolation period.

Confusion and criticism ensued, as public experts said the agency erred by not specifying the need for a negative test before leaving isolation. Walensky and the administration spent the next week fielding criticism, and top officials hinted that a change was coming.  

The agency then added language to say a person could take a test if they want one and it was available, but insisted one was not necessary. Walensky has explained that rapid tests are not intended to be used to test out of isolation.  

She has cited rapidly evolving science for the need to make such a change. She testified during a Senate hearing last Tuesday that the changes represent “swift science-based action to address the very real possibility of staffing shortages.”

But Walensky admitted much of the research justifying shortening the isolation period came before the omicron variant took over as the dominant strain.

"We felt the need to take action before we had omicron-specific data,” she told The Wall Street Journal.

Walensky also said she intends to address gaps in national collection of public health data, and said she has the Biden administration's support to modernize data collection efforts.

Earlier this month, Walensky held her first solo press briefing since taking the helm. Almost all her other briefings have come as part of the White House's COVID-19 task force press conferences, where she appears with other administration officials rather than with agency scientists.

Walensky told the Journal that she plans to hold more solo media briefings in the coming months.

https://thehill.com/policy/healthcare/590059-walensky-says-she-will-improve-cdc-policy-messaging

Canadian Regulator Approves Use of Pfizer Oral Drug for Covid-19

 Pfizer Canada said Monday that Canada's health regulator has approved the use of its Covid-19 oral treatment for adults over the age of 18.

The pharmaceutical and biotechnology company said that Health Canada has authorized the use of PAXLOVID, also known as the nirmatrelvir tablets and ritonavir tablets, for the treatment of mild-to-moderate Covid-19.

The drug is designed to block the activity of one of the virus's key enzymes, which it uses to replicate itself.

Health Canada approved the drug based on data from Pfizer's phase 2/3 EPIC-HR trial for adults with Covid-19 at a higher risk of seeing their illness progress.

The company noted that additional phase 2/3 trials are still in process for adults at standard risk.

In December, Pfizer said it had reached an agreement with the Canadian government to supply 1 million treatment courses of the treatment in 2022.

"Pfizer is ready to begin delivery in Canada immediately to help get PAXLOVID into the hands of appropriate patients as quickly as possible," Pfizer Canada Hospital Business Unit Lead Kevin Mohamed said.

https://www.marketscreener.com/quote/stock/PFIZER-INC-23365019/news/Canadian-Regulator-Approves-Use-of-Pfizer-Oral-Drug-for-Covid-19-37567821/