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Rocket Pharmaceuticals, Inc. RCKT announced that it has completed patient enrollment in a pivotal phase II study evaluating its investigational gene therapy candidate, RP-A501, for treating male patients with Danon disease.
Danon disease is a rare X-linked inherited disorder caused by mutations in the LAMP2 gene.
Per the company, an estimated 15,000 to 30,000 patients suffer from Danon disease in the United States and Europe. Currently, the only available treatment for the disease is cardiac transplantation, which is not considered curative and may lead to complications.
Shares of the company were up 13.5% on Sept. 17 following the announcement of the news.
Year to date, shares of Rocket have plunged 27.2% against the industry’s increase of 1%.
More on RCKT’s RP-A501 Danon Disease Study
The global multi-center phase II study is evaluating the safety and efficacy of RP-A501 in male patients with Danon disease.
The co-primary endpoint of the study is to check the improvements in LAMP2 protein expression and reductions in left ventricular mass as measured by the biomarker while assessing the efficacy of RP-A501. This will be used to support the accelerated approval for RP-A501.
The key secondary endpoint of the study is to observe the change in troponin. Other secondary endpoints include natriuretic peptides event-free survival to 24 months and treatment-emergent safety events.
Per management, RP-A501 is the first gene therapy product to demonstrate safety and efficacy in clinical studies for a cardiovascular condition.
The FDA and European Medical Agency previously granted regenerative medicine advanced therapy designation and PRIority MEdicines (PRIME) scheme designation to RP-A501, respectively.
RCKT’s Other Pipeline Development Activities
The company is developing its gene therapy candidate, Kresladi (marnetegragene autotemcel), to treat patients with severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic disorder.
In June 2024, the FDA issued a complete response letter (CRL) to Rocket’s biologics license application (BLA) seeking approval for Kresladi to treat LAD-I.
Per the CRL, the FDA sought "limited" additional information on the Chemistry Manufacturing and Controls (CMC), which is part of the BLA submission. The goal of CMC is to ensure that every batch of the final product is identical in terms of its safety, purity, potency and performance.
Rocket is also evaluating two other gene therapies, namely RP-L102 and RP-L301, for the treatment of Fanconi anemia and pyruvate kinase deficiency, respectively. Both candidates are in mid-stage development.
Zacks Rank & Stocks to Consider
Rocket currently carries a Zacks Rank #3 (Hold).
Some top-ranked stocks in the biotech sector are Illumina, Inc. ILMN, Krystal Biotech, Inc. KRYS and Fulcrum Therapeutics, Inc. FULC, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for Illumina’s 2024 earnings per share have moved up from $1.84 to $3.62. Earnings per share estimates for 2025 have improved from $3.22 to $4.43. Year to date, shares of ILMN have lost 6.5%.
ILMN’s earnings beat estimates in each of the trailing four quarters, the average surprise being 463.46%.
In the past 60 days, estimates for Krystal Biotech’s 2024 earnings per share have increased from $2.09 to $2.38. Earnings per share estimates for 2025 have improved from $4.33 to $7.31. Year to date, shares of KRYS have risen 52.6%.
KRYS’ earnings beat estimates in three of the trailing four quarters while missing on the remaining occasion, the average surprise being 45.95%.
In the past 60 days, estimates for Fulcrum Therapeutics’ 2024 loss per share have narrowed from $1.24 to 33 cents. Loss per share estimates for 2025 have narrowed from $1.71 to $1.14. Year to date, shares of FULC have plunged 48.3%.
FULC’s earnings beat estimates in each of the trailing four quarters, the average surprise being 393.18%.
Zacks Investment Research
NEW YORK, NY / ACCESSWIRE / September 18, 2024 / Bronstein, Gewirtz & Grossman, LLC is investigating potential claims on behalf of purchasers of Fulcrum Therapeutics, Inc. ("Fulcrum" or "the Company") (NASDAQ:FULC). Investors who purchased Fulcrum securities are encouraged to obtain additional information and assist the investigation by visiting the firm's site: bgandg.com/FULC.
Investigation Details
On September 12, 2024, Fulcrum announced that "its Phase 3 REACH trial evaluating losmapimod in patients with FSHD, did not achieve its primary endpoint of change from baseline in RSA with losmapimod compared to placebo. In addition, secondary endpoints did not achieve nominal statistical significance." Following this news, Fulcrum stock dropped almost 60% in morning trading on the same day.
What's Next?
If you are aware of any facts relating to this investigation or purchased Fulcrum securities, you can assist this investigation by visiting the firm's site: bgandg.com/FULC. You can also contact Peretz Bronstein or his client relations manager, Nathan Miller, of Bronstein, Gewirtz & Grossman, LLC: 332-239-2660.
There is No Cost to You
We represent investors in class actions on a contingency fee basis. That means we will ask the court to reimburse us for out-of-pocket expenses and attorneys' fees, usually a percentage of the total recovery, only if we are successful.
Why Bronstein, Gewirtz & Grossman
Bronstein, Gewirtz & Grossman, LLC is a nationally recognized firm that represents investors in securities fraud class actions and shareholder derivative suits. Our firm has recovered hundreds of millions of dollars for investors nationwide.
Attorney advertising. Prior results do not guarantee similar outcomes.
Contact
Bronstein, Gewirtz & Grossman, LLC Peretz Bronstein or Nathan Miller 332-239-2660 | info@bgandg.com
SOURCE: Bronstein, Gewirtz & Grossman, LLC
View the original press release on accesswire.comIf you're interested in broad exposure to the Healthcare - Biotech segment of the equity market, look no further than the Invesco Biotechnology & Genome ETF (PBE), a passively managed exchange traded fund launched on 06/23/2005.
Retail and institutional investors increasingly turn to passively managed ETFs because they offer low costs, transparency, flexibility, and tax efficiency; these kind of funds are also excellent vehicles for long term investors.
Investor-friendly, sector ETFs provide many options to gain low risk and diversified exposure to a broad group of companies in particular sectors. Healthcare - Biotech is one of the 16 broad Zacks sectors within the Zacks Industry classification. It is currently ranked 5, placing it in top 31%.
Index Details
The fund is sponsored by Invesco. It has amassed assets over $271.87 million, making it one of the average sized ETFs attempting to match the performance of the Healthcare - Biotech segment of the equity market. PBE seeks to match the performance of the Dynamic Biotechnology & Genome Intellidex Index before fees and expenses.
The Dynamic Biotech & Genome Intellidex Index seeks to provide capital appreciation by thoroughly evaluating companies based on a variety of investment merit criteria, including price momentum, earnings momentum, quality, management action, and value.
Costs
Cost is an important factor in selecting the right ETF, and cheaper funds can significantly outperform their more expensive counterparts if all other fundamentals are the same.
Annual operating expenses for this ETF are 0.58%, making it on par with most peer products in the space.
It has a 12-month trailing dividend yield of 0.05%.
Sector Exposure and Top Holdings
Even though ETFs offer diversified exposure that minimizes single stock risk, investors should also look at the actual holdings inside the fund. Luckily, most ETFs are very transparent products that disclose their holdings on a daily basis.
This ETF has heaviest allocation in the Healthcare sector--about 99.90% of the portfolio.
Looking at individual holdings, Gilead Sciences Inc (GILD) accounts for about 5.58% of total assets, followed by Illumina Inc (ILMN) and Regeneron Pharmaceuticals Inc (REGN).
The top 10 holdings account for about 48.18% of total assets under management.
Performance and Risk
So far this year, PBE return is roughly 8.14%, and was up about 20.51% in the last one year (as of 09/18/2024). During this past 52-week period, the fund has traded between $52.52 and $71.87.
The ETF has a beta of 0.80 and standard deviation of 23.55% for the trailing three-year period, making it a high risk choice in the space. With about 32 holdings, it has more concentrated exposure than peers.
Alternatives
Invesco Biotechnology & Genome ETF carries a Zacks ETF Rank of 3 (Hold), which is based on expected asset class return, expense ratio, and momentum, among other factors. Thus, PBE is a sufficient option for those seeking exposure to the Health Care ETFs area of the market. Investors might also want to consider some other ETF options in the space.
SPDR S&P Biotech ETF (XBI) tracks S&P Biotechnology Select Industry Index and the iShares Biotechnology ETF (IBB) tracks Nasdaq Biotechnology Index. SPDR S&P Biotech ETF has $7.70 billion in assets, iShares Biotechnology ETF has $7.73 billion. XBI has an expense ratio of 0.35% and IBB charges 0.45%.
Bottom Line
To learn more about this product and other ETFs, screen for products that match your investment objectives and read articles on latest developments in the ETF investing universe, please visit Zacks ETF Center.
Zacks Investment Research
LOS ANGELES, CA / ACCESSWIRE / September 17, 2024 / The Schall Law Firm, a national shareholder rights litigation firm, announces that it is investigating claims on behalf of investors of Fulcrum Therapeutics, Inc. ("Fulcrum" or "the Company") (NASDAQ:FULC) for violations of the securities laws.
The investigation focuses on whether the Company issued false and/or misleading statements and/or failed to disclose information pertinent to investors. Fulcrum announced on September 12, 2024, that "its Phase 3 REACH trial evaluating losmapimod in patients with FSHD, did not achieve its primary endpoint of change from baseline in RSA with losmapimod compared to placebo. In addition, secondary endpoints did not achieve nominal statistical significance." Based on this news, shares of Fulcrum fell by almost 60% in morning trading on the same day.
If you are a shareholder who suffered a loss, click here to participate.
We also encourage you to contact Brian Schall of the Schall Law Firm, 2049 Century Park East, Suite 2460, Los Angeles, CA 90067, at 310-301-3335, to discuss your rights free of charge. You can also reach us through the firm's website at www.schallfirm.com, or by email at bschall@schallfirm.com
The Schall Law Firm represents investors around the world and specializes in securities class action lawsuits and shareholder rights litigation.
This press release may be considered Attorney Advertising in some jurisdictions under the applicable law and rules of ethics.
CONTACT: The Schall Law Firm Brian Schall, Esq. 310-301-3335info@schallfirm.comwww.schallfirm.com
SOURCE: The Schall Law Firm
View the original press release on accesswire.comShares of Ascendis Pharma ASND rose 17% on Monday after it reported top-line results from the pivotal ApproaCH study that evaluated investigational once-weekly administered TransCon CNP in children aged two to 11 years with achondroplasia, the most common form of dwarfism.
ASND Drug Shows AGV Benefit in Achondroplasia Patients
The ApproaCH study met its primary endpoint of annualized growth velocity (AGV) at 52 weeks — children treated with TransCon CNP achieved a mean AGV of 5.89 cm/year compared with 4.41 cm/year in the placebo group.
Management also provided sub-group analyses of this data by age groups — children aged two to less than five years and another group aged five to 11 years. Patients in the first group who received TransCon CNP achieved a mean AGV of 6.07 cm/year compared with 5.06 cm/year in placebo. Those who received the drug in the second age group achieved a mean AGV of 5.79 cm/year compared with 4.02 cm/year on placebo.
Per Ascendis, treatment with the drug also demonstrated statistically significant improvement in other secondary endpoints, including height Z-score and changing from baseline AGV.
But Why Did BioMarin Stock Dip on TransCon CNP Data
There is only one drug approved to treat achondroplasia in the United States and Europe — Voxzogo — which is marketed by BioMarin BMRN. Since its launch in 2021, BMRN has been experiencing a rapid uptake for Voxzogo driven by strong prescription demand. The drug added nearly $337 million to BioMarin’s topline in the first half of 2024, up 67% year over year.
While it is difficult to compare the results of two different clinical studies directly, investors were likely impressed with the once-weekly dosing advantage of TransCon CNP over Voxzogo, which requires daily dosing. This major factor was not only responsible for Ascendis’ share price soaring but also sent BioMarin stock down nearly 18% after the results were announced.
Ascendis’ shares have moved up 10.8% in the year compared with the industry’s 1.0% growth.
Ascendis’ Future Plans for the Drug
Based on the ApproaCH study results, management intends to submit a regulatory filing seeking approval for TransCon CNP in achondroplasia with the FDA during first-quarter 2025. It also plans to submit a similar filing with the EMA by third-quarter 2025.
ASND’s Zacks Ranks
Ascendis currently carries a Zacks Rank #3 (Hold).
Ascendis Pharma A/S Price
Ascendis Pharma A/S price | Ascendis Pharma A/S Quote
Our Key Picks in the Biotech Sector
Some better-ranked stocks in the overall healthcare sector are Bioventus BVS and Illumina ILMN. Each stock presently sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for Bioventus’ 2024 earnings per share (EPS) have risen from 27 cents to 40 cents. Estimates for 2025 have increased from 43 cents to 45 cents during the same period. Year to date, shares of Bioventus have surged 109.9%.
BVS’ earnings beat estimates in three of the last four quarters and missed the mark on one occasion. Bioventus delivered a four-quarter average earnings surprise of 102.86%.
In the past 60 days, estimates for Illumina’s 2024 EPS have risen from $1.84 to $3.62. Estimates for 2025 have increased from $3.22 to $4.43 during the same period. Year to date, Illumina’s shares have lost 7.0%.
Earnings of Illumina beat estimates in each of the last four quarters. Illumina delivered a four-quarter average earnings surprise of 463.46%.
Zacks Investment Research
NORTHAMPTON, MA / ACCESSWIRE / September 17, 2024 / IlluminaThe quality control stability scientist embraces family, sports, work, and her leadership role in Illumina's iLatinx employee resource group
Originally published on Illumina News Center
Karina Katalina Moreno has taken a circuitous route to her American dream. She grew up in Monterrey, Mexico, about 140 miles (225 km) south of Laredo, Texas, and got her degree in biotech engineering at Tecnológico de Monterrey. Following a brief stop in Barcelona, Spain, she earned her master of science degree at the University of Edinburgh.
Family brought Moreno back to Monterrey. Her mother was diagnosed with lung cancer, and she moved in to help care for her parents and three siblings. She worked as a science teacher at a local high school and helped her mom get to and from treatment.
"After my mom passed away, I thought, okay, this is life, I knew it was coming," she says. "She was diagnosed with stage three lung cancer, and the doctors told us she wouldn't have a lot of time. She was a real fighter, and stayed with us for three more years. But 13 days after my mom died, my dad passed from COVID. At that point I felt lost. My life changed so much in two weeks that I didn't know what I was going to do with my life."
Coming to Illumina Moreno had worked in pharma and cosmetics but always wanted to pursue a career in biotech. In 2023, a friend encouraged her to apply at Illumina and, after a rigorous interview process, she was hired as a quality control stability scientist. In this role, she focuses on measuring reagent stability, which has already paid big dividends in extended expiration dates.
She's been with Illumina for about a year and a half, but Moreno is already making her mark. She is well known in Building 6, at Illumina's San Diego campus, for her seemingly endless supply of Mexican candy-it's part of her ongoing mission to share her culture.
iLatinx Moreno is the treasurer of iLatinx, a growing employee resource group (ERG) at Illumina. The group has more than a hundred active members and hosts many events that focus on building community: attending San Diego Padres and Wave games, celebrating Hispanic Heritage Month, and working with the Make-a-Wish Foundation and the Clearity Foundation.
The group has also committed to mentoring young women. For the past two summers, iLatinx has partnered with the Women at Illumina Network ERG to sponsor Girls Who Lead days at the company.
"We have around 60 girls who come to Illumina and go to the lab with us," Moreno says, "but we try to involve them in other activities, as well, because not everyone works in the lab. Some people handle money or are in charge of production. We want to show them all their options."
The iLatinx group has big plans for this year's Hispanic Heritage Month, which runs from September 15 to October 15: a career panel, salsa classes, a mariachi band, and lots of treats. They highlight different countries each year: Last time it was Mexico and Peru; this year will be Colombia and Venezuela, among others.
"People like to learn about the different cultures at Illumina, and we try to find people from those places who can share their flags, food, and local customs," Moreno says. "We want everyone to know they don't have to be Latino to be part of this community. They only need to like the food, music, and culture."
Her safe space An avid athlete, Moreno has been active in team handball since she was young and now plays on teams in San Diego and Baja California. At Illumina, she participates in flag football, basketball, and softball. When she's not working or playing, she often visits her family in Monterrey. But looking at the life she's built, she is incredibly grateful to have found a home in San Diego and Illumina.
"Everybody is really open [at Illumina], and that makes me feel great," says Moreno. "I have this accent and nobody cares, and I can wear a flower shirt, which is typical in Mexico, or sing in Spanish, and nobody is going to say anything. For me, it's a really safe space."
Karina Katalina Moreno at the Paris Olympics, summer 2024. | Photo courtesy of Moreno.
View additional multimedia and more ESG storytelling from Illumina on 3blmedia.com.
Contact Info: Spokesperson: Illumina Website: https://www.3blmedia.com/profiles/illumina Email: info@3blmedia.com
SOURCE: Illumina
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