- Emergent Biosolutions (NYSE:EBS): Q3 Non-GAAP EPS of $2.19 beats by $0.04; GAAP EPS of $0.73 misses by $1.03.
- Revenue of $385.2M (+23.5% Y/Y) misses by $53.31M.
- FY2020 guidance: Total revenues of $1,520M-$1,580M from previous guidance of $1,500M-$1,600M vs consensus of $1.52B ; Adjusted net income of $375M-$405M from previous guidance of $340M-$390M; Adjusted EBITDA of $575M-$615M from previous guidance of $535M-$600M.
- Press Release
- https://seekingalpha.com/news/3632865-emergent-biosolutions-eps-beats-0_04-misses-on-revenue-revised-fy20-guidance
Thursday, November 5, 2020
Emergent Biosolutions EPS beats by $0.04, misses revenue, revises FY20 guidance
Pfizer Vyndaqel heart disease drug zooms toward blockbuster land
All public attention for Pfizer these days is centered on its BioNTech-partnered COVID-19 vaccine. But while key phase 3 efficacy data for the experimental shot remain blinded, the company’s tafamidis franchise is cruising on a clear growth trajectory that now looks sure to take it to blockbuster status.
In the third quarter, tafamidis, marketed under the brands Vyndaqel and Vyndamax, sold $351 million, up 27% from its haul in the second quarter. Both the U.S. and international markets contributed to the growth.
As the drug’s market performance improves on every front, SVB Leerink analyst Mani Foroohar now projects the rare disease drug to reach $2.5 billion in 2021 global revenues, according to an investor note from last week.
Vyndaqel last May scored its long-awaited FDA nod to treat a rare heart disease called ATTR cardiomyopathy (ATTR-CM). Industry watchers immediately pegged its peak sales estimates in blockbuster territory, but both analysts and Pfizer management acknowledged that the drug’s success was dependent on the company’s ability to drive diagnosis rates.
Before Vyndaqel arrived, an estimated 1% to 2% of patients living with ATTR-CM were properly diagnosed. But Pfizer’s push to increase awareness and its use of noninvasive diagnostics are paying off. The U.S. diagnosis rate has steadily ticked upward to about 17% in Q3 from 15% in Q2, Pfizer CEO Albert Bourla told investors on a conference call last week.
As of the end of Q3, over 17,500 U.S. patients have been diagnosed, more than 12,000 have gotten a prescription, and about 7,300 have received the drug, Bourla said. The conversion rate of prescribed patients to treated patients has improved slightly to 63% from 62%, Foroohar noted. In Q3, 82% of diagnosed patients who were deemed eligible for Vyndaqel were able to get a prescription, according to Pfizer Biopharmaceuticals Group president, Angela Hwang. That figure also represented an increase from 78% in Q2.
Vyndaqel’s $225,000-a-year sticker recently drew criticism as physicians complained about patients not being able to afford the drug’s out-of-pocket cost, which is part of the reason why some patients didn’t eventually get the medicine.
Lawmakers have recently proposed bills that would cap out-of-pocket costs in Medicare Part D. During the conference call, Bourla said the current insurance benefits system in the U.S. needs to change. “It seems like there is a general recognition in both sides of the aisle,” he said. “We will continue working with all, and we hope that we will see a change in the design of these benefits that will reduce the cost that the Americans have to pay out-of-pocket when they go to collect their medicines.”
Outside the U.S., Pfizer in February won EU approval for Vyndaqel in ATTR-CM, about nine years after its initial approval there in ATTR-polyneuropathy, a neurologic form of ATTR. That green light helped Vyndaqel’s ex-U.S. sales tally swell 46% in Q3 over Q2 to reach $193 million.
Now, with Pfizer's Upjohn established medicines business on track to be folded in with Mylan in a new company called Viatris, Vyndaqel ranks fifth among Pfizer’s top-selling drugs by Q3 numbers. It trails only pneumococcal vaccine Prevnar 13, breast cancer drug Ibrance, Bristol Myers Squibb-shared anticoagulant Eliquis and JAK inhibitor Xeljanz.
Treating multiple sclerosis with an antigen-specific cell therapy
Existing multiple sclerosis therapies systematically modulate the immune system to dampen its erroneous attack on the protective myelin sheaths around nerve cells, which is the hallmark of the autoimmune disease. But this approach puts patients at a higher risk of infection.
Scientists at Thomas Jefferson University said they have found a way to train the immune system to tolerate self-antigens that trigger inflammatory responses in MS while leaving the rest of the immune system intact.
They isolated tiny sacs called extracellular vesicles from cells known as oligodendrocytes. The sacs contained myelin antigens, and when they injected those particles into mice, it suppressed MS, according to a new study published in Science Translational Medicine.
Because existing MS therapeutics suppress the immune system in a systemic way, scientists have been trying to find alternative therapies that target the disease in an antigen-specific way. This approach requires understanding which myelin self-antigens are involved in MS. Problem is, disease-causing antigens can differ among patients or change over time in the same patient.
“There are many possible immune-activating antigens in the myelin sheath, but the biggest hurdle is that we don't know which component of myelin is triggering the immune response in MS patients,” Abdolmohamad Rostami, M.D., Ph.D., the study’s senior author, explained in a statement. “Previous studies have used single myelin antigens or combinations of antigens to prevent auto-immunity in animal models, but in humans they have had limited success.”
Rostami and colleagues sidestepped the need to pinpoint specific myelin antigens by turning to oligodendrocytes, which produce the myelin sheath. The team showed that oligodendrocytes produced extracellular vesicles that contained almost all the relevant myelin antigens.
“The neat thing about these EVs is that they give us an opportunity to treat the disease in an antigen-specific way, without having to know the exact identity of the target antigen,” Rostami said. “It covers all the bases.”
The team tested the vesicles’ efficacy in three MS mouse models representing chronic and relapsing-remitting disease. The drug ameliorated clinical disease both when injected before symptoms developed and after disease onset.
The treatment tamped down the population of infiltrating CD45+ and CD4+ T cells in the central nervous system, the researchers found. The infused vesicles were preferentially taken up by some white blood cells, including monocytes and neutrophils. And the monocytes that absorbed the vesicles pumped up the expression of several anti-inflammatory molecules, such as PD-L1 and IL-10, further helping suppress MS.
The effects of the therapy depended solely on myelin antigens but not other components on the vesicles, the researchers showed. Human embryonic kidney cell-derived vesicles didn’t have any therapeutic benefits and didn’t decrease the number of CD4+ T cells, but genetically edited versions that expressed myelin oligodendrocyte protein did.
Oligodendrocytes’ role in myelin formation was previously leveraged in another way by researchers at the University of Rochester Medical Center to treat MS. That team used human glial progenitor cells to generate new oligodendrocytes, which restored myelin in mouse models of MS.
In another study aimed at replacing destroyed myelin in MS, a team from the VA Maryland Health Care System and the University of Maryland School of Medicine found that some CD34-expressing stem cells can transform into oligodendrocytes.
Rostami’s team at Thomas Jefferson University also isolated the vesicles from human oligodendrocytes and confirmed that they contained therapeutically relevant myelin antigens. The researchers are working on patenting the approach, which they hope will advance toward clinical trials.
“This is a huge advantage of our antigen-specific method over current therapies, which are like a sledgehammer to the immune system, and what makes it so novel,” Rostami said.
https://www.fiercebiotech.com/research/treating-multiple-sclerosis-antigen-specific-cell-therapy
CRISPR, Vertex show CRISPR/Cas9 gene-editing therapy works in more patients
CRISPR Therapeutics and Vertex have presented updated data on their CRISPR/Cas9 gene-editing therapy in sickle cell disease and beta thalassemia, showing patients continue to benefit from the drug. Yet, hemoglobin levels fell in two of the seven patients in the latest update, raising early questions about the durability of CTX001.
One year ago, CRISPR and Vertex linked CTX001 to improvements in two hemoglobinopathy patients. The results were a milestone for gene editing, but CTX001 needs to build on the early success if it is to emerge as a viable rival to bluebird bio’s LentiGlobin gene therapy, which is now set to be submitted for FDA approval in late 2022.
The LentiGlobin delays create an opportunity for CTX001. The question is whether CRISPR and Vertex can take it. Ahead of the American Society of Hematology's (ASH's) annual meeting, the partners shared new data that shed light on the potential prospects of CTX001.
Fetal hemoglobin levels rose in all seven patients after administration of CTX001 and have remained elevated for up to 15 months of follow-up. The five beta thalassemia patients last needed packed red blood cells in the first month or two after receiving CTX001, having previously more than 10 units a year, and neither of the sickle cell patients have suffered vaso-occlusive crises since being treated.
There are potential points of concern in the data, though, particularly given the likelihood of CTX001 facing competition. Hemoglobin fell from 12.5 to 11.6 in one beta thalassemia patient between the fifth and sixth months. The drop followed a decrease from 12.9 to 12.5 between the fourth and fifth months.
Investigators also tracked a decline in hemoglobin levels in one of the sickle cell patients. Levels fell from 11.8 to 10.3 between month nine and month 12.
The declines happened in two of the three patients with the longest follow-up, raising the question of whether similar trends will play out in other subjects in the coming months. Those concerns are countered by data on the first beta thalassemia patient, whose hemoglobin level is higher than ever 15 months after treatment with CTX001.
Safety is another concern. In an update earlier this year, CRISPR and Vertex revealed they had seen seven serious adverse events across three patients, suggesting bluebird’s LentiGlobin may have the edge in terms of safety. The ASH update discussed a new patient who suffered four serious adverse events that may be related to CTX001.
Such details could matter, given CTX001 is still likely to cede a head start to LentiGlobin. Yet, CRISPR sees CTX001 as a best-in-class treatment and, with bluebird stumbling, thinks the drug is well placed to capture a sizable slice of the market.
“CRSP has mentioned they are working on bridging the development timeline gap (~1-2 yrs now) w/ BLUE, and they expect BLUE's earlier launch and investment in developing+educating the mkt could benefit CRSP w/o loss of a bolus of pts,” analysts at Jefferies wrote in a note to investors.
Hologic’s diagnostic sales rocket 375% as COVID-19 demand continues to rise
After seeing its overall revenues slip last quarter, Hologic’s COVID-19 testing sales have continued to skyrocket—powering the company’s most recent quarterly income to over $1.3 billion.
The performance of Hologic's coronavirus tests, including kits for its high-throughput Panther and Panther Fusion systems, offset declines seen in its other businesses as the pandemic once again began to surge to new levels. The company saw sales drops in its diagnostic offerings for blood, cytology and newborn testing, as well as among its gynecological surgery, mammography and osteoporosis screening divisions.
Even so, it managed to post year-over-year sales growth of 55.6% in the final quarter of its 2020 fiscal year. Hologic's molecular diagnostics division alone brought in $818.9 million for the period, marking a 375.8% gain and nearly eclipsing the $865.8 million in total revenues the company generated in the final quarter of fiscal 2019.
“We capped off an unprecedented fiscal year with remarkable financial results in our fourth quarter,” CEO Steve MacMillan said in a statement. “These results were driven by the tireless efforts of our Diagnostics, European and supply chain teams to provide COVID-19 tests, and by steady improvement in our other businesses compared to the June quarter.”
And the company isn't stopping there. The U.S. government earlier this week issued Hologic a $119 million contract to help the company increase its domestic test manufacturing capacity, saying that demand for COVID-19 diagnostics has outpaced the company’s ability to supply its Panther tests. The money will be used to expand production lines in Wisconsin, Maine and California, with the goal of providing 13 million tests per month by January 2022.
The contract followed an FDA move from late September to expand its emergency authorizations for the laboratory testing systems, allowing them to be used for pooled testing and the screening of people who have shown no symptoms of COVID-19.
The fourth-quarter performance marks a turnaround for Hologic after a 3.5% revenue drop to $822.9 million in Q3, a dip the company attributed in part to the 2019 sale of its Cynosure aesthetics business. At the time, Hologic's molecular diagnostics revenue was up 169.3% compared to the previous year, but the company saw large declines across the board elsewhere as patients postponed medical procedures and trips to the clinic.
Endo EPS beats by $0.38, beats on revenue, raises FY20 guidance
- Endo (NASDAQ:ENDP): Q3 Non-GAAP EPS of $0.52 beats by $0.38; GAAP EPS of -$0.30 beats by $0.22.
- Revenue of $634.86M (-13.0% Y/Y) beats by $84.23M.
- CF Ops: $77M (+133.3%).
- 2020 guidance: Revenues: $2.75B-$2.80B from previous guidance of $2.60B-$2.70B vs. $2.7B Consensus; non-GAAP EPS: $2.50- $2.55 from previous guidance of $2.00 - 2.15 vs. $2.14 consensus; non-GAAP EBITDA: $1.30B-$1.32B from previous guidance of $1.19B-$1.23B; Adjusted gross margin of ~67.0% from previous guidance of ~66.5% - ~67.0%; Adjusted interest expense of ~$525M from previous guidance of ~$530M-~$535M; Adjusted effective tax rate of ~13.5% from previous guidance of ~14.0% - ~15.0%.
https://seekingalpha.com/news/3632720-endo-eps-beats-0_38-beats-on-revenue-raises-fy20-guidance
Sarepta Exondys 51 and Vyondys 53 sales up 23% in Q3
- Sarepta Therapeutics (NASDAQ:SRPT) Q3 results:
- Revenues: $143.9M (+45.4%); product sales: $121.4M (+22.6%); collaboration revenue: $22.5M (primarily from Roche).
- Net loss: ($196.5M) (-55.6%); loss/share: ($2.50) (-47.1%).
- Quick assets: $1,816,1M (+61.5%).
- Q4 consensus: loss/share of ($1.78) on revenues of $145.3M.
- Data readout on SRP-5051 by year-end.
- U.S. marketing application for Amondys 45 (casimersen) for DMD patients amenable to exon 45 skipping currently under FDA review with an action date of February 25, 2021.
- Dosing to begin this quarter in new 10-subject study evaluating the safety and expression of commercial process material for SRP-9001, its gene transfer therapy for DMD. Preliminary data should be available in early 2021.
- https://www.blogger.com/blog/post/edit/3207539574676545063/5543057765168700814
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