Amicus Therapeutics (NASDAQ:FOLD) has initiated rolling marketing application to the FDA for AT-GAA for the treatment of late onset Pompe disease (LOPD), an inherited metabolic disorder caused by the buildup of glycogen in the body's cells due to mutations in the gene that codes for an enzyme called acid alpha-glucosidase, leading to organ dysfunction.
The rolling submission allows submission of portions of the regulatory application as they are completed, rather than waiting until every section of the application is complete.
Earlier this year, the FDA authorized Amicus to proceed with a rolling BLA submission.
AT-GAA consists of cipaglucosidase alfa (ATB200), an enzyme replacement therapy with optimized carbohydrate structures, administered in conjunction with miglustat (AT2221), an orally administered stabilizer of cipaglucosidase alfa.
Amicus has submitted the nonclinical component of the cipaglucosidase alfa application and is on-track to submit the chemistry, manufacturing and controls component, and the final clinical module in 1H of 2021.
The company will separately submit miglustat application at the same time as the remainder of the cipaglucosidase alfa BLA.
AT-GAA received FDA Breakthrough status for LOPD. Top-line data from ongoing Phase 3 PROPEL study of AT-GAA in LOPD is on-track for Q1 of 2021.
Recently, under the priority review status, the FDA accepted Sanofi's application for long-term enzyme replacement therapy for Pompe disease. Agency's action date is May 18, 2021.
A Phase 3 clinical trial, ACTIVATE, evaluating Agios Pharmaceuticals' (NASDAQ:AGIO) mitapivat in adults with pyruvate kinase (PK) deficiency who do not receive regular transfusions met the primary endpoint.
Specifically, 40% of treated patients achieved a hemoglobin response at weeks 16, 20 and 24 compared to 0% in the control group (p<0.0001).
Key secondary endpoints were also met.
No new safety signals were observed.
Complete results will be submitted for presentation at the European Hematology Association Virtual Congress in June 2021.
The company plans to file marketing applications in the U.S. and Europe in 2021.
Mitapivat is an orally available activator of wild-type and mutated pyruvate kinase-R enzymes.
TG Therapeutics (NASDAQ:TGTX)initiates its rolling U.S. marketing application seeking approval of ublituximab, combined with umbralisib, for the treatment of chronic lymphocytic leukemia (CLL), a Fast Track- and Orphan Drug-tagged indication. The company expects to complete the filing in H1 2021.
Ublituximab is an anti-CD20 monoclonal antibody. Umbralisib is a dual inhibitor of enzymes PI3K-delta and CK1-epsilon. The company calls the doublet therapy "U2."
Its U.S. marketing applications for umbralisib for marginal zone lymphoma and follicular lymphoma are currently under FDA review with action dates of February 14, 2021 and June 15, 2021, respectively.
Dr. Reddy's Laboratories (NYSE:RDY)announces the start of an adaptive Phase 2/3 clinical trial in India evaluating Russia's Sputnik V COVID-19 vaccine. The study will be conducted by JSS Medical Research.
An adaptive study allows for modifications to the trial and/or statistical models while the study is in process.
The company is partnering with the Biotechnology Industry Research Assistance Council (BIRAC), Department of Biotechnology (DBT) for advisory support and to use BIRAC’s clinical trial centers for the vaccine.
The Russian Direct Investment Fund (RDIF) recently announced the results from the second interim analysis of a large-scale Phase 3 study that showed 91.4% efficacy at day 28 after the first dose and over 95% 42 days after the first dose. Over 40K volunteers are participating.
In September, RDY and RDIF entered into a partnership to conduct clinical trials of the vaccine and the rights for distribution of the first 100 million doses in India.
The FDA approves Roche's (OTCQX:RHHBY+1.9%) Xolair (omalizumab) as add-on maintenance treatment of nasal polyps in adults who fail to adequately response to corticosteroids.
The anti-IgE antibody was first approved in 2003 for asthma in patients at least 12 years old.
Goldman Sachs analysts are pounding the table on three stocks in particular, noting that each could surge over 40% in the year ahead. After running the trio throughTipRanks’ database, we found out that the rest of the Street is also standing squarely in the bull camp.
As we have all learned from coronavirus pandemic, some new thing in medical science can make huge impact on our world. Codiack aims to turn that principle to good. This research-oriented pharmaceutical aims to turn exosome therapeutics into a whole new class of medicines. Exosomes are the degradation mechanism RNA, and can transfer genetic material around a body.
And therein lies the potential. Codiack has developed a design platform for the engineering of exosome proteins capable of carrying and protecting drug molecules through cell walls. In effect, the proteins will mimic the pathways used by viruses – but are non-viral, and are designed to carry a ‘payload’ of therapeutic agents. If successful, exosome therapy offers doctors the ability to design a drug that will deliver specific agents to specific cells to fight specific disease.
Codiack is involved in all aspects of exosome therapeutics, from design to manufacturing, and currently has an active pipeline of agents – seven, in all – in various stages of discovery, preclinical testing, and the beginnings of Phase 1 trials.
In the biosciences, success or failure is all about that pipeline, and in its diverse, active pipeline of agents in a new sector of biotechnological pharmaceuticals, Codiack has a fine resource to attract investors. To get those investors, the company went public this past October, selling 5.5 million shares at an opening price of $14.10 per share.
Among the healthcare name's fans is Goldman Sachs analyst Graig Suvannavejh. The analyst wrote, “Biopharma industry interest in exosomes has long been high, but engineering them for a specific function and manufacturing at scale have both proven challenging. Among a field of multiple competitors, CDAK has made the most significant progress on both fronts, and as such we view their technology platform as best-in-class.”
"Given share underperformance (-37%) since the IPO, we find risk/reward highly compelling at current levels, and with key 2021 data sets to provide potential de-risking and positive share inflection," the analyst concluded.
Suvannavejh rates CDAK a Buy, and his $29 price target shows the extent of his confidence – it implies a 222% upside for the coming year. (To watch Suvannavejh’s track record, click here)
Overall, Codiack has a Strong Buy from the analyst consensus – 3 reviewers have put up Buy ratings in recent weeks. The stock is selling for $8.90, and its $24 average price target implies a 166% one-year upside potential. (See CDAK stock analysis on TipRanks)
Acrutis is a pioneering researcher in the treatment of dermatological disease. Arcutis is involved in discovering the next generation of dermatological treatments – an important niche, especially when one realizes that one common ailment, psoriasis, has not seen an FDA approval for a novel treatment in over two decades.
The company is leveraging recent advances in immunology and inflammation to find new approaches to skin treatment. The goal is to make it easier for patients and doctors together to manage conditions like psoriasis, alopecia, atopic dermatitis, seborrheic dermatitis, and vitiligo, to name just a few.
The company's lead candidate, ARQ-151 (roflumilast cream), is about to enter a phase 3 trial for atopic dermatitis, and is in an advanced phase 3 stage in Plaque Psoriasis.
Arcutis has recently issued an update on positive data from the Phase 2 trials of ARQ-151 in atopic dermatitis. The drug is a once-daily treatment, and has demonstrated significant patient relief from symptoms, especially itching and itching-related sleep problems.
This is another stock in Suvannavejh’s coverage universe. The Goldman analyst is impressed by developments in the company’s pipeline work, noting: “ARQT provided an update on the outcome of its end-of-Phase 2 meetings with the FDA, following their Phase 2a trial of ARQ-151 in atopic dermatitis (AtD). Feedback from regulators was broadly encouraging, in particular, acknowledging the robust long-term safety data being generated by ARQT for ARQ-151 in plaque psoriasis…”
Accordingly, Suvannavejh rates ARQT a Buy, and sets a $36 price target that indicates room for 40% upside growth in 2021. (To watch Suvannavejh’s track record, click here)
Arcutis has 2 recent Buy reviews, making the consensus rating a Moderate Buy. The stock’s average price target is $37, suggesting a 44% upside from current levels. (See ARQT stock analysis on TipRanks)
With the last stock, we move from medical research to medical care. Specifically, Oak Street Health is a primary care clinic operator, and part of the Medicare Network. The company has operations and clinics in Illinois, Indiana, Michigan, Pennsylvania, and Ohio, along with New York, North Carolina, Rhode Island, Tennessee, and Texas. It has been in operation for eight years, and went public this past summer, holding the IPO in August.
In the third quarter, the company’s first as a publicly traded entity, OSH brought in $217.9 million in revenue. The revenue number was up 56% from the year-ago quarter. Earnings per share matched expectations, at 15 cents.
The company’s expansion proceeds apace, and in October, Oak Street entered New York by opening, in Brooklyn, its 70th location. A planned expansion in Texas, involving a partnership with Walmart, is also proceeding as planned, and Oak Street has opened its first Walmart Community Clinic the Dallas-Fort Worth area city of Carrollton.
Robert Jones, covering this stock for Goldman, set a $74 price target to back his Buy rating. At currently levels, this target implies an upside of ~58% in the next 12 months. (To watch Jones’ track record, click here)
“Results suggest operations are still on track, with few incremental updates since the 2Q call, where management noted a resumption of center openings, (pivoted) marketing efforts, and in-person visits despite COVID. In 3Q, OSH opened 13 new centers and is on track for 73-75 by end of year… The company maintained that it is continuing to operate at a high level in places with elevated COVID case counts like Chicago and Detroit,” Jones noted.
All in all, the Strong Buy analyst consensus rating OSH is based on 8 reviews, breaking down to 7 Buys and just a single Hold. The stock is selling for $46.94, and its $61.29 average price target suggests it has a ~31% upside for the coming year. (See OSH stock analysis on TipRanks)
Already the nation’s largest health insurance company, UnitedHealth is poised to grow its top line by about 8%. UnitedHealth operates commercial, Medicaid and Medicare Advantage plans via its UnitedHealthcare health insurance unit and is also expanding its medical care provider business across the country under its fast-growing Optum unit.
As the company began its annual investor day virtually Tuesday morning, UnitedHealth said its 2021 outlook will include revenues of “$277 billion to $280 billion, net earnings of $16.90 to $17.40 per share, and adjusted net earnings of $17.75 to $18.25 per share.”
This growth will follow an updated 2020 performance that included an updated projection Tuesday of 2020 revenues expected to be about $257 billion, “with net earnings to approach $15.90 per share and adjusted net earnings to approach $16.75 per share.”
UnitedHealth chief executive David Wichmann said the company’s strategy will continue to push a move to value-based medical care models while escalating the use of technology to make sure patients get care that they need when they need it. As one example in his opening statements, he said patients will get more digital coaching along with advancements in telehealth.
“United is how we will deliver the future of health,” Wichmann told analysts and investors.
The meeting also marked the return of Sir Andrew Witty, who left UnitedHealth Group as head of Optum earlier this year on a leave of absence to a position at the World Health Organization to aid the development of vaccines against Covid-19. “I look forward to the kind of innovation this company can uniquely deliver,” Witty, who is president of UnitedHealth and CEO of Optum, said speaking from the United Kingdom after he was introduced by Wichmann.
