Friday, the FDA approved BioMarin's Roctavian (valoctocogene roxaparvovec-rvox) gene therapy for adults with severe hemophilia A without antibodies to adeno-associated virus serotype 5.
The analyst expects Roctavian's uptake to be (s)lower than consensus expectations, and it continues to model ~$65 million /$900 million in total Roctavian 2023/peak sales (vs. ~$83 million/ $1,400 million for consensus).
The analyst says its expectations remain unchanged since January 2023 (vs. ~$215 million /$1,600 million for consensus in January 2023).
The analyst also says that at this valuation (-25% since initiated coverage here), Roctavian constitutes an upside opportunity as it's primarily not priced in.
BMO analyst says Voxzogo's launch has been very successful in Achondroplasia and constitutes a significant growth driver for BioMarin.
At the same time, recent data from BridgeBio Pharma Inc
Infigratinib in Phase 2 suggests that Infigratinib can gain significant market share in Achondroplasia, especially given Infigratinib's clean safety and oral administration. 12-month Phase 2 Infigratinib data is awaited to assess its impact further.
Noting Roctavian's (s)low uptake and Infigratinib's impact, BMO sees ~20% upside in BioMarin. The analyst also says that Voxzogo is largely not priced in at these levels (~$86/share) and, therefore, Roctavian's contribution offers an upside opportunity moving forward.
Allarity Therapeutics, Inc. (“Allarity” or the “Company”) (Nasdaq: ALLR), a clinical-stage pharmaceutical company developing novel oncology therapeutics together with drug-specific DRP®companion diagnostics for personalized cancer care, today announced initial results from its European Phase 2 clinical trial evaluating the efficacy of IXEMPRA®in metastatic breast cancer (mBC) patients selected with the DRP®-IXEMPRA®companion diagnostic (CDx) candidate. Researchers prescreened mBC patients using Allarity’s DRP®-IXEMPRA®CDx, a complex transcriptomic signature comprising multiple mRNA biomarkers of drug response/resistance. Patients were assigned a DRP®-score, and those with scores above 67% were selected for treatment with IXEMPRA®.
Of the 36 patients screened with the DRP®-IXEMPRA® CDx, investigators identified five DRP® positive patients. Among the evaluable patients assessed up to the data evaluation cut-off, there were promising signs of clinical benefit in four out of four evaluable cases:
One partial responder (PR) (tumor shrinkage of 66%).
One partial responder (PR) (tumor shrinkage of 59%).
One patient experienced 24 weeks of stable disease.
One patient experienced 19 weeks of stable disease.
The first of two “safe sleeping” areas for the homeless in San Diego opened on Thursday as part of an effort to move people off the streets in the California city.
The plan was originally announced in June that the city’s Homeless Strategies and Solutions Department would expand its services to combat the ongoing homeless crisis by offering different living arrangements.
“We are bringing forward a comprehensive shelter strategy with detailed proposals for the short, medium and long terms that include expanded shelter, more safe parking and safe sleeping offerings,” San Diego Mayor Todd Gloria said at the time.
Almost one month later, a new tent campsite behind a barbed-wire fence opened at San Diego’s Central Operations Yard at 20th and B streets.
City officials are planning to pitch as many as 136 tents with the capacity to hold approximately 150 people in an effort to move them from downtown areas.
The site will be operated by the nonprofit organization Dreams for Change who previously took part in maintaining “safe parking” sites for people living in vehicles.
The area has been described as a “low barrier” shelter that prohibits residents from bringing in weapons, alcohol or drugs but does not require guests to be sober.
Homeless residents will also have access to restrooms, meals and mobile showers.
A larger safe-sleeping site is expected to open in the fall in a parking lot near the Naval Medical Center. The site will likewise offer access to meals and other services and hold up to 400 tents.
The site will be operated by the nonprofit organization Dreams for Change who previously took part in maintaining “safe parking” sites for people living in vehicles.Matthew Bowler/KPBS/Sipa USAThe area has been described as a “low barrier” shelter that prohibits residents from bringing in weapons, alcohol or drugs but does not require guests to be sober.Matthew Bowler/KPBS/Sipa USA
The move comes after the city council also voted to prohibit tent encampments in all public spaces. The ban is expected to go into effect later this month.
In 2019, the Department of Housing and Urban Development reported that San Diego and the surrounding county had the fourth-highest homeless population in the country.
Homeless residents will also have access to restrooms, meals and mobile showers.Matthew Bowler/KPBS/Sipa USA
In response, San Diego’s then-Mayor Kevin Faulconer signed off on an $11 million contract to construct three “bridge shelters” to house unsheltered citizens.
“We’re taking dramatic action to move homeless people off the streets and get them help,” Faulconer told Fox News. “Our strategy is to connect, support and shelter them.”
San Diego Mayor Todd Gloria at the opening of the new Safe Sleeping program.Matthew Bowler/KPBS/Sipa USAIn 2019, the Department of Housing and Urban Development reported that San Diego and the surrounding county had the fourth-highest homeless population in the country.CBS 8 According to data from the Downtown San Diego Partnership, nearly 2,000 people lived outside the city in April,the highest in more than a decade.
A more recent count put the number at over 2,100 people living on sidewalks or in vehicles.
Mayor Gloria also proposed a city budget that included $81.7 million for homelessness services, a nearly $20 million increase from the last fiscal year.
Philippine President Ferdinand Marcos Jr said on Wednesday initial local trials for an African swine fever vaccine had showed promising results, raising hopes that it will soon be available in the country to combat the deadly animal disease.
A number of vaccines have been tested worldwide as authorities rush to stop the spread of the highly contagious disease that ravages pig farms and the $250 billion global pork industry regularly. One vaccine candidate is close to approval in Vietnam.
Marcos, who also heads the agriculture ministry, said preliminary clinical trials showed the vaccine produced sufficient antibodies and was safe to use so far, but needed further testing.
In the Philippines, African swine fever remains a major concern for hog raisers as millions of pigs have already been culled since the first local outbreak was detected in 2019.
Since then, 66 out of the country's 81 provinces have experienced outbreaks. As of last month, there were active cases in 13 provinces, according to the Department of Agriculture.
The resulting reduced domestic pork supply has prompted the Philippines to ramp up meat imports as local prices shot up, adding pressure on inflation that remains outside the central bank's target range.
Marcos, in a speech at a livestock trade event, said a second phase of the vaccine trial was underway but warned against complacency.
"It is not a reason for complacency, as we are being continuously warned by those who have studied the vaccine," he said.
The Philippines has also made progress in the procurement of avian flu vaccines, having called on manufacturers to register their products with the Food and Drug Administration, he added.
India will break ground next month on its first semiconductor assembly plant and begin producing its first domestically manufactured microchips by the end of 2024, the Financial Times reported on Wednesday.
India's information technology minister, Ashwini Vaishnaw, said Micron Technology would start construction in August on a $2.75 billion chip assembly and test facility in Gujarat, the newspaper reported.
Atomico, Lakestar and General Catalyst join €60 million round
Startup provides full-body scans for skin and heart conditions
Neko Health, the medical diagnostics company co-founded by Spotify Technology SA Chief Executive Officer Daniel Ek, raised €60 million ($65.4 million) in venture capital to expand outside its home country of Sweden.
Skype co-founder Niklas Zennstrom will join the company’s board as a result of his investment vehicle, Atomico, participating in the round, as will Klaus Hommels, whose VC firm Lakestar led the early-stage investment. Palo Alto-based General Catalyst also participated. Neko Health didn’t disclose a valuation.
