Sanofi's genetic disorder drug shows mixed results in late-stage trials

By Bhanvi Satija

Reuters

LONDON, Feb 2 (Reuters) - French drugmaker Sanofi said on Monday that its experimental genetic disorder treatment showed promise in a late-stage study of patients with ​a type of Gaucher disease, but failed to meet the main goal in ‌a separate trial.

The oral drug, venglustat, was being tested in patients with Fabry disease and type 3 Gaucher disease. Both ‌inherited conditions are caused by enzyme deficiencies that lead to the accumulation of toxic substances in the body.

Previous failures in trials of patients with Parkinson's disease and a type of acute kidney disease have prompted Sanofi to prioritize testing the drug in rare genetic disorders, where its mechanism of ⁠blocking the buildup of harmful fatty ‌molecules has yielded promising early-stage results.

Analysts have not projected future sales for venglustat as market expectations for its success remain low.

Sanofi is banking on ‍its late-stage pipeline and recent acquisitions to help drive sales growth in the next decade, after the top-selling eczema and asthma drug Dupixent it shares with Regeneron loses exclusivity.

Sanofi's bet could pay off if venglustat is ​eventually approved, making it the first such drug to target neurological symptoms and giving patients ‌an oral dosing option.

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"A daily pill could make a serious difference for Gaucher patients facing neurological challenges," said Sanofi research chief Houman Ashrafian.

But the drug's path to regulatory approval looks murky, especially for Fabry disease. Ashrafian said data from the Fabry disease study was still being analysed, while the company said it would work with global regulators to determine next steps.

STUDY DETAILS

The drug ⁠showed superior improvements in neurological symptoms such as speech ​and limb coordination for type 3 Gaucher disease patients, ​compared to those who received enzyme replacement therapy. It also demonstrated statistically significant improvements on three of four secondary goals of that study.

In patients with Fabry ‍disease, venglustat helped reduce ⁠neuropathic and abdominal pain, but not enough to declare statistical success. Sanofi suggested that may be due to a large placebo effect. The company said it helped reduce levels ⁠of plasma lyso-GL-3 in patients, which is an indicator of accumulated harmful fat molecules.

Sanofi already sells Fabrazyme, an enzyme‑replacement ‌therapy for Fabry disease, and markets Cerezyme and the oral drug Cerdelga for ‌Gaucher disease.

(Reporting by Bhanvi SatijaEditing by Bill Berkrot)

Sanofi's genetic disorder drug shows mixed results in late-stage trials

By Bhanvi Satija LONDON, Feb 2 (Reuters) - French drugmaker Sanofi said on Monday that its experimental gene...
Factbox-Which are the main parties in Thailand's election and what are their policies?

Feb 2 (Reuters) - Thailand is holding a general election on February 8 with three parties expected to dominate the contest to decide which will lead the Southeast Asian country's next government.

Following are details and key policy pledges of the main parties running in the election.

BHUMJAITHAI PARTY

Led by incumbent Prime Minister Anutin Charnvirakul, Bhumjaithai formed the last government in September after moving in swiftly ​to dismantle the Pheu Thai-led coalition following a court's sacking of then premier Paetongtarn Shinawatra. Bhumjaithai was not a contender in the 2023 and 2019 elections, taking 71 and 51 seats respectively, though enough ‌to earn spots in the coalition governments.

Bhumjaithai has brought in technocrats and will hope to benefit from its incumbency and defections from other parties to boost its share of the vote. It will likely need to form an alliance to lead the next government.

Key policies:

- Phase two ‌of a co-payment subsidy scheme that in stage one covered half of the cost of selected food and consumer goods

- Boost growth to above 3% through a "10-Plus Plan", including support for low-income earners, better income and care for seniors; community-based products; free education with job pathways, support for small businesses

- Construct border walls to prevent smuggling of illicit goods, illegal labour, proliferation of "grey" businesses

- Introduce volunteer soldiers posts with monthly salaries; create 100,000 jobs in a "One Village, One Volunteer Nurse" scheme

- Barter trade for major state purchases such as fighter jets and submarines, paid for with Thai farm goods

- National disaster fund to support co-pay insurance for 29.5 million households

- Cheap loans for buying electric motorcycles

- Boost small businesses through state procurement, ⁠cheap loans, credit guarantees, new market access

- Promote green economy initiatives, community solar cells, ‌and cap household electricity bills

PHEU THAI PARTY

Pheu Thai and its predecessors have dominated Thai politics for the past quarter-century through huge electoral war chests and populist policies that won massive working class support and loyalty.

Founded by divisive, currently jailed billionaire Thaksin Shinawatra, the party has powerful enemies in the royalist establishment. Six premiers from or allied with the Shinawatra ‍family have been removed by military coups and court rulings.

The 2023 election was the first since 2001 where Thaksin-backed parties did not win the most votes. Pheu Thai's support has been challenged of late following Paetongtarn's removal and its failure to fully implement its signature "digital wallet" handout programme.

Key policies:

- "Millionaire-maker" campaign that award nine daily prizes of 1 million baht ($31,556) each, targeting tax filers, seniors over 60, farmers, volunteers and buyers of goods and services

- Top-up incomes for those with annual earnings below 36,000 baht ($1,163)

- For rice, ​cassava, rubber and corn, set market price targets; guarantee farmers at least 30% profit in first year

- Coupons for up to 250 kg (551 pounds) of fertiliser and 150 kg of seeds per household

- Suspend principal and interest ‌for three years on loans up to 500,000 baht for farmers

- Convert land documents to full land title deeds for 33 million rai (about 13 acres)

- 500,000 baht ($16,150) in seed funding per startup, plus five‑year tax exemptions

- Add mental health services as a core benefit under a 30 baht universal healthcare scheme

- Establish a national credit guarantee agency to support small businesses

- Programme aimed at developing 20 million high‑skilled workers

- Affordable housing with low instalments and no downpayment for qualified first-time buyers

- Flat 20 baht rate for all trips on electric trains; two‑year motorcycle tax exemption for delivery riders

- Relief for holders of non-performing loans below specified thresholds; small loans from state banks to repay informal debt

PEOPLE'S PARTY

People's Party is the third incarnation of the progressive movement after its two popular predecessors were dissolved by courts, including Move Forward, which won most votes in the 2023 election but was blocked from forming a government by a Senate ⁠hand-picked by the military.

Surveys consistently show People's Party is Thailand's most popular party, suggesting it still has strong backing among young ​and urban Thais for its liberal policies and institutional reform agenda.

Though it is a contender to win, its efforts to form a government ​could again encounter turbulence, with long-term reforms it has previously pledged and some of its current manifesto posing a challenge to the interests of conservatives, powerful families and business groups with far-reaching political influence.

Key policies:

- Replace military conscription with a fully voluntary military with enlistment contracts of 4-8 years; reduce number of generals; improve welfare for lower-ranking soldiers

- Government guarantees on new ‍loans for small businesses and upgrades to national credit database

- ⁠Tighten screening for company registrations to tackle illicit businesses; crackdown on nominee structures and grey capital; increase penalties and shorten court processes

- Develop a southern port link system to cut logistics costs and strengthen southern economy

- Single transport ticket capping trip prices on urban rail, buses and ferries

- National single-wage formula adjusted annually based on living costs and economic conditions

- Provide 1,000 baht co-payment top-up for 12 million people

- Subsidise ⁠training for new skills; provide courses linked to labour market needs

- Raise VAT to 8% by 2028 and 10% by 2030, with compensation for low‑income households

- Reform to police to prevent buying/selling of police positions

- Limit working hours to five days or 40 hours per week, ‌with overtime pay required beyond

- Implement menstrual leave up to 3 days per month and annual compassionate/care leave up to 15 days

- Establish a 10 billion baht annual fund to buy and ‌absorb surplus farm produce before prices fall

($1 = 31.6900 baht)

(Compiled by Orathai Sriring and Martin Petty; Editing by Raju Gopalakrishnan)

Factbox-Which are the main parties in Thailand's election and what are their policies?

Feb 2 (Reuters) - Thailand is holding a general election on February 8 with three parties expected to dominate the conte...
Russia's Medvedev says expiry of New START should alarm the world

MOSCOW, Feb 2 (Reuters) - Dmitry Medvedev, deputy chairman of Russia's ​Security Council, said that if ‌the New START treaty expired with ‌no replacement then the world should be alarmed that the biggest nuclear powers had no limits ⁠for probably the ‌first time since the early 1970s.

Reuters

"I don't want ‍to say that this immediately means a catastrophe and a nuclear war ​will begin, but it should ‌still alarm everyone," Medvedev told Reuters, TASS and the WarGonzo Russian war blogger in an interview at his residence outside Moscow.

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Arms ⁠control treaties, Medvedev said, ​played a crucial ​role not just in limiting the number of warheads but ‍also ⁠as a way to verify intentions and to ensure some ⁠element of trust between major nuclear ‌powers.

(Reporting by Guy Faulconbridge; Editing ‌by Tom Hogue)

Russia's Medvedev says expiry of New START should alarm the world

MOSCOW, Feb 2 (Reuters) - Dmitry Medvedev, deputy chairman of Russia's ​Security Council, said that if ‌the New STAR...
Wembanyama fuels early surge in late start as Spurs overcome travel woes to beat Magic

SAN ANTONIO (AP) — Victor Wembanyama had 25 points, eight rebounds and five blocks and San Antonio beat the Orlando Magic 112-103 on Sunday night in a game that started five hours late because of the Spurs' travel woes.

The Spurs were scheduled to leave Charlotte following a111-106 lossSaturday, but had to stay overnight because of thestorm that dropped nearly a foot of snow. The team then had a mechanical issue on its flight.

Devin Vassell added 16 points for San Antonio. Dylan Harper had 15 points, and De'Aaron Fox had 14 points and 10 assists.

Desmond Bane scored 25 points for Orlando, and Paolo Banchero had 19 points and 10 rebounds. Orlando had won two in a row.

The Magic had been in San Antonio since Saturday after a 130-120 home victory over Toronto. The Spurs weren't so lucky.

The game was scheduled to start at 3 p.m. but was pushed back to 6 p.m. and then 8 p.m. as the Spurs returned home nearly 24 hours later than planned.

The delayed start did not hinder the Spurs as they made their first three shots and seven of their first eight in building a double-digit lead five minutes in, Wembanyama made his first two attempts, forcing his way past Wendell Carter Jr. in the lane for a finger-roll layup and a two-handed slam.

Wembanyama was cleared to play a half hour before tipoff after being questionable due to left calf soreness.

San Antonio guard Stephon Castle missed the game with left adductor tightness.

Magic: At Oklahoma City on Tuesday night.

Spurs: Host Oklahoma City on Wednesday night.

AP NBA:https://apnews.com/hub/NBA

Wembanyama fuels early surge in late start as Spurs overcome travel woes to beat Magic

SAN ANTONIO (AP) — Victor Wembanyama had 25 points, eight rebounds and five blocks and San Antonio beat the Orlando Magi...
Chris Kreider scores 2 goals, Ducks beat slumping Golden Knights 4-3 to sweep season series

ANAHEIM, Calif. (AP) — Chris Kreider scored two goals, Lukas Dostal made 27 saves and the Anaheim Ducks swept their three-game season series with the Vegas Golden Knights with a 4-3 victory on Sunday night.

Cutter Gauthier scored and Ryan Poehling added an empty-net goal for the Ducks. Anaheim earned its eighth victory in 10 games overall despite nearly blowing an early 3-0 lead during a third period dominated by Vegas.

Mitch Marner and Ivan Barbashev scored goals in their third straight games for the Golden Knights, who have lost five straight and seven of eight. Vegas has gone on two five-game skids since Christmas, with an 8-2-0 surge sandwiched between them.

Tomas Hertl scored with 6 seconds to play and Adin Hill stopped 19 shots for Vegas.

Kreider put Anaheim up 2-0 with his first multigoal game since Nov. 6 for the Ducks, who acquired him last June from the New York Rangers.

He opened the scoring late in the first when a puck from Poehling hit his leg and went in. He tipped home his 19th goal early in the second.

Gauthier scored his 24th goal a few minutes later, beating Hill at the near post for Anaheim's fifth power-play goal in five games.

Marner took advantage of a lucky deflection when Mark Stone's pass from behind the net ricocheted off two Ducks and went straight to him.

Vegas took control in the third period, and Barbashev tapped home a stationary puck in the crease after Dostal couldn't smother Jack Eichel's shot.

Anaheim right wing Troy Terry returned from an 11-game absence with an upper-body injury, and center Mason McTavish also returned after missing five games.

The Ducks had their eighth consecutive sellout as they returned from a five-game road trip to begin a nine-game homestand. Anaheim doesn't play another road game until March 10.

Golden Knights: Host Vancouver on Wednesday.

Ducks: Host Seattle on Tuesday.

AP NHL:https://apnews.com/NHL

Chris Kreider scores 2 goals, Ducks beat slumping Golden Knights 4-3 to sweep season series

ANAHEIM, Calif. (AP) — Chris Kreider scored two goals, Lukas Dostal made 27 saves and the Anaheim Ducks swept their thre...
Lightning rally from 4-goal deficit to beat Bruins 6-5 in Stadium Series thriller

TAMPA, Fla. (AP) — Nikita Kucherov scored the tying goal in the third period, Jake Guentzel scored the only goal in the shootout, and the Tampa Bay Lightning rallied from a four-goal deficit to beat the Boston Bruins 6-5 in front of 64,617 fans at an NHL Stadium Series game at Raymond James Stadium.

Kucherov finished with a goal and three assists, Brandon Hagel had a goal and two assists, while Oliver Bjorkstrand, Darren Raddysh and Nick Paul all scored power-play goals after Tampa Bay fell behind 5-1 in the second period.

Andrei Vasilevskiy finished with 29 saves.

Tampa Bay's four-goal comeback is the largest in NHL outdoor game history and the largest comeback victory in franchise history.

Morgan Geekie had a pair of goals and three points for the Bruins. Viktor Arvidsson, Alex Steeves and Matthew Poitras also scored for Boston, which improved to 11-1-1 in the past 13 games. Charlie McAvoy had a pair of assists while Jeremy Swayman finished with 41 saves.

The game featured the first goalie fight in outdoor game history when Vasilevskiy and Swayman exchanged blows at center ice in the second period.

HURRICANES 3, KINGS 2, OT

RALEIGH, N.C. (AP) — Sebastian Aho scored early in overtime to lead Carolina to a win over Los Angeles.

Brandon Bussi made 11 saves to continue his dominant rookie season, while Jordan Staal and Alexander Nikishin also scored for the Hurricanes, who have earned at least a point in eight straight games (6-0-2).

Samuel Helenius and Quinton Byfield scored for the Kings and Anton Forsberg made 31 saves. The Kings wrapped up their road trip with a 3-1-1 record with one game (last Monday at Columbus) postponed due to severe winter weather.

Awinter stormdumped snow all around North Carolina but Raleigh was mostly spared, which made it easier for about 14,000 fans to make it to the arena for the afternoon start.

A day after squandering a three-goal lead in a4-3 overtime loss at Washington, Aho made sure the Hurricanes didn't blow a 2-0 lead against the Kings. He beat Forsberg 1:25 into the overtime period after the Kings' goalie had made two tough saves on Seth Jarvis.

DUCKS 4, GOLDEN KNIGHTS 3

ANAHEIM, Calif. (AP) — Chris Kreider scored two goals, Lukas Dostal made 27 saves and Anaheim swept their three-game season series with Vegas.

Cutter Gauthier scored and Ryan Poehling added an empty-net goal for the Ducks. Anaheim earned its eighth victory in 10 games overall despite nearly blowing an early 3-0 lead during a third period dominated by Vegas.

Mitch Marner and Ivan Barbashev scored goals in their third straight games for the Golden Knights, who have lost five straight and seven of eight. Vegas has gone on two five-game skids since Christmas, with an 8-2-0 surge sandwiched between them.

Tomas Hertl scored with 6 seconds to play and Adin Hill stopped 19 shots for Vegas.

Kreider put Anaheim up 2-0 with his first multigoal game since Nov. 6 for the Ducks, who acquired him last June from the New York Rangers.

Lightning rally from 4-goal deficit to beat Bruins 6-5 in Stadium Series thriller

TAMPA, Fla. (AP) — Nikita Kucherov scored the tying goal in the third period, Jake Guentzel scored the only goal in the ...
2 measles infections confirmed at Texas facility where 5-year-old detained in Minnesota was held

Two detainees were infected with measles at a Texas immigration facility where 5-year-old Liam Conejo Ramos and his father had been held, a Department of Homeland Security official said Sunday.

NBC Universal A dense crowd of hundreds of people wearing raincoats and hoods is seen from an aerial perspective. Many of them are holding signs. (Brenda Bazán / AP)

The two patients at Dilley Immigration Processing Center were quarantined, alongside anyone else who may have made contact, DHS spokesperson Tricia McLaughlin said in a statement. "All movement" within the facility was halted, she said.

The detainees in question, who were not identified, were in good hands, she said. "This is the best healthcare than many aliens have received in their entire lives," McLaughlin said.

Dilley Immigration Processing Center is where 5-year-old Liam and his father, Adrian Alexander Conejo Arias, had been helduntil they were released Saturday. It wasn't clear whether they may have been in contact with the infected detainees. An attorney for the family did not respond Saturday night to a request for comment.

Measles can be spread through the air through breathing, talking, coughing and sneezing, and it can remain an active infectious agent on surfaces. The infection — noted by rashes, red eyes, fever, coughing, and red bumps at the hairline — can be especially dangerous for infants, children, pregnant women, the elderly and people with compromised immune systems, according to the Centers for Disease Control and Prevention.

Hundreds of protesters holding signs stand in the middle of a road in a dusty landscape. Law enforcement vehicles and officers on foot stand opposite them in the distance. (Moises Avila / AFP via Getty Images)

McLaughlin said medical staff members were monitoring detainees' conditions and would "take appropriate and active steps to prevent further infection."

Liam and his father were taken into custody on Jan. 20 after having encountered immigration agents upon returning home from the boy's preschool in Minnesota.

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A lawyer for the family said they came to the United States in 2023 and were pursuing asylum under "established protocols" when father and son were arrested. McLaughlin said previously that Liam had been abandoned, but his mothertold Noticias Telemundothat immigration enforcement agents used the boy as "bait" in an attempt to detain her.

A judge on Saturdayordered father and son to be releasedfrom the Texas facility about 70 miles southwest of San Antonio, where other families are detained together. It's also known as the South Texas Family Residential Center.

McLaughlin said the Texas Department of State Health Services confirmed the measles cases.

As part of a civil case over immigrant detention, Ben Thomas, chief of staff for Rep. Joaquin Castro, D-Texas, submitted a declaration to federal court that said Castro's office was also informed of the measles infections by Immigration and Customs Enforcement on Saturday.

The infections add to a national surge in measles, of which 588 cases had been confirmed in the U.S. as of Thursday, according todata from the CDC. On Saturday, Los Angeles County health officials announced the discovery of the second measles case in the region since the start of the year.

Experts areconcerned that the infection has become endemic, or an annual occurrence, despite virtual elimination in 2000 thanks to effective vaccination.

Experts blame the latest infections on declining vaccination rates.

Before measles vaccinations became available in the early 1960s, the infection wasresponsible for about 400 to 500 deathsand 48,000 hospitalizations every year in the U.S. Three people died of measles in the country last year, according to the CDC.

2 measles infections confirmed at Texas facility where 5-year-old detained in Minnesota was held

Two detainees were infected with measles at a Texas immigration facility where 5-year-old Liam Conejo Ramos and his fath...

 

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