Exagen Reports Prelim Q4 Testing Results

Exagen Inc. (Nasdaq: XGN), an organization dedicated to transforming the care continuum for patients suffering from autoimmune diseases, today reported preliminary, unaudited financial results for the quarter ended December 31, 2020.

Testing revenue for the three months ended December 31, 2020 is expected to be between approximately $10.5 million and $10.8 million, an increase of 11% and 15% compared with $9.5 million in the third quarter of 2020, and an increase of 10% to 13% compared with $9.6 million in the fourth quarter of 2019. Exagen delivered a total of approximately 28,600 AVISE CTD tests, including AVISE Lupus, in the quarter, with 1,690 ordering healthcare providers, a record number of 635 adopters and a sequential quarterly retention rate of 99% among adopting healthcare providers from the prior quarter.

The above financial results exclude SIMPONI^® revenue due to the nature of syndicated pharmaceutical data reporting timing. The company will provide SIMPONI revenue and consolidated revenue for the fourth quarter and full year ended December 31, 2020 when it reports its complete financial results for such periods, which is expected in March 2021. The preliminary, unaudited financial information presented in this press release is based on Exagen's current expectations and is subject to change as a result of, among other things, completion of financial closing procedures and the audit for the full year 2020.

“The fourth quarter of 2020 was strong for Exagen, despite continuing challenges from the COVID-19 pandemic. Our core testing business delivered record revenue, driven largely by record quarterly volumes for our flagship AVISE CTD test. It is also gratifying to note that we attained a record number of healthcare provider adopters in the quarter, along with another period of very high retention rates, which are indicative of the value healthcare providers place on our testing products. We believe we are firmly positioned as a leading provider of autoimmune-related testing services and have the momentum and capabilities to drive continued growth in 2021," said Ron Rocca, President and CEO of Exagen.

https://www.biospace.com/article/releases/exagen-inc-reports-preliminary-fourth-quarter-2020-testing-results/

Karyopharm Prelim Q4, Commercial Update Disappoints

 - Unaudited Total Revenues of between $35.0 Million and $36.0 Million for Fourth Quarter 2020 and between $108.0 Million and $109.0 Million for the Full Year 2020 --

-- XPOVIO® (selinexor) Unaudited Net Product Sales of between $20.0 Million and $20.5 Million for Fourth Quarter 2020 and between $76.0 Million and $76.5 Million for the Full Year 2020 --

-- Commercial Launch of XPOVIO in Expanded Multiple Myeloma Indication Fully Underway --

https://www.prnewswire.com/news-releases/karyopharm-announces-preliminary-unaudited-fourth-quarter-and-full-year-2020-total-revenues-and-provides-commercial-update-301204826.html

Alnylam Launches 5-Yr Growth Plan

 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced its new 5-year strategy “Alnylam P5x25” focused on the Company’s planned transition to a top-5 biotech (measured by market capitalization) in the next 5 years through: sustainable innovation yielding transformative medicines for rare and common diseases for patients around the world and delivery of exceptional financial performance. Alnylam P5x25 extends the Company’s decade-long heritage of providing longer term, 5-year business strategy guidance, the most recent of which was known as Alnylam 2020. In addition, Alnylam today reported preliminary fourth quarter and full year 2020 global net product revenues for ONPATTRO and GIVLAARI and provided additional updates on the Company’s commercial launches, including initial OXLUMO demand.

New 5-Year Strategy: P5x25
Alnylam ended last year exceeding all metrics for its Alnylam 2020 strategy, with 4 marketed products (versus 3), 12 clinical programs (versus 10), 6 of which are in late-stage development (versus 4), across 4 strategic therapeutic areas (versus 3).

The Company’s Alnylam P5x25 strategy is aimed at Alnylam’s transition to a top 5 biotech company, as measured by market capitalization, over the next 5 years.

Specifically, the Company intends to end 2025 with the following profile**:

• Patients: Over 0.5 million on Alnylam RNAi therapeutics globally
• Products: 6 or more marketed products in rare and prevalent diseases
• Pipeline: Over 20 clinical programs, with 10 or more in late stages and 4 or more INDs per year
• Performance: ≥40% revenue CAGR through YE 2025
• Profitability: Achieve sustainable non-GAAP profitability within the period

“We executed well on our Alnylam 2020 strategy, exceeding all pre-set metrics and transitioning into a global, multi-product commercial company with a robust clinical pipeline and an organic product engine delivering sustainable innovation, a profile that has rarely been achieved in biotech history. It was especially gratifying to cap 2020 with positive Phase 3 HELIOS-A results for vutrisiran, which is set to become our 5th RNAi therapeutic to reach the market, if approved,” said John Maraganore, Ph.D., CEO of Alnylam Pharmaceuticals. “We are now thrilled to launch our new chapter with Alnylam P5x25, which is aimed at Alnylam’s planned transition to a top 5 biotech in market capitalization based on a proven and high-yielding technology for disruptive medical innovation and a foundational track record of commercial execution. Indeed, with Alnylam P5x25, we expect to sustainably and organically create and commercialize transformative rare and common disease medicines benefiting hundreds of thousands of patients around the world while delivering strong financial performance and profitability, resulting in a leading biotech profile.”

2020 Preliminary Commercial and Financial Performance*
ONPATTRO® (patisiran), a commercial-stage RNAi therapeutic targeting transthyretin (TTR) for the treatment of polyneuropathy in adult patients with hATTR amyloidosis.

• Preliminary global net product revenues for the fourth quarter and full year 2020 were approximately $90 million and $306 million, respectively.
  • Q4 results represent approximately 10 percent growth compared to Q3 and include 10 percent growth in the U.S. market segment driven by new patient demand.
  • Further, the full year ONPATTRO revenues reached the high end of the previously shared guidance range of $295 million - $310 million and represent over 80 percent growth from full year 2019.
• As of year-end 2020, about 1,350 patients worldwide were receiving commercial ONPATTRO.

GIVLAARI® (givosiran), a commercial-stage RNAi therapeutic for the treatment of adults with acute hepatic porphyria (AHP).

• Preliminary global net product revenues for the fourth quarter and full year 2020 were approximately $22 million and $55 million, respectively.
  • These results represent greater than 30 percent quarter over quarter growth.
• As of year-end 2020, the product’s first full year of launch, over 200 patients are receiving commercial drug.

OXLUMO™ (lumasiran), a commercial-stage RNAi therapeutic for the treatment of primary hyperoxaluria type 1 to lower urinary oxalate levels in pediatric and adult patients.

• For the period following European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) approval of OXLUMO, in late November 2020, strong initial U.S. demand was observed with 8 Start Forms received through year-end.
• Preliminary global net product revenues for the fourth quarter were approximately $0.3 million representing initial patient demand in Europe

Finally, the Company today reported that it expects its full year 2020 non-GAAP operating loss to be substantially improved relative to the prior year, marking 2019 as Alnylam’s peak non-GAAP operating loss year as the Company transitions towards a self-sustainable financial profile.

Further, at December 31, 2020, Alnylam had preliminary cash, cash equivalents, and marketable securities of approximately $1.9 billion, as compared to $1.5 billion at December 31, 2019. The Company balance sheet was strengthened by the 2020 strategic financing collaboration with Blackstone.

https://www.businesswire.com/news/home/20210110005034/en/Alnylam-Launches-%E2%80%9CAlnylam-P5x25%E2%80%9D-Strategy-for-Planned-Transition-to-a-Top-Five-Biotech-in-Market-Capitalization-Over-Next-Five-Years

Street cautiously optimistic on J&J’s ‘one-dose’ Covid vaccine

 Health care systems around the world are struggling to cope with rising numbers of Covid-19 infections as they race against the clock to vaccinate the vulnerable. 

The three vaccines currently approved for use by major Western economies all require two separate jabs and given supplies are limited, governments are considering contentious tactics like stretching the length of time between doses to get at least one dose to as many people as possible. 

A one-shot vaccine could significantly improve our ability to fight the virus — and we may have one soon.

J&J’s late-stage trial 

Johnson & Johnson is expected to deliver preliminary late-stage trial results for its one-dose Covid vaccine candidate by the end of January. If its jab is proven to be safe and effective, the company aims to deliver at least 1 billion doses by the end of the year. 

The J&J vaccine was developed by the company’s Belgian unit, Janssen Pharmaceutica, and is based on viral adenovirus vector technology, the same approach used to create the University of Oxford-AstraZeneca vaccine. This type of shot is easier to scale up than those developed by Pfizer-BioNTech and Moderna which are based on messenger RNA technology.

Health care analyst Adam Barker at Shore Capital said in an email to CNBC last week: “The J&J vaccine is more like the AstraZeneca vaccine, but it uses only one dose. So we know this approach works (viral-vector) and it targets the spike protein. We know that target works too. But, we’ll have to see what one dose does.”

Morgan Stanley’s health care team said in a research note published last week that J&J’s vaccine offers “unique elements and efficacy could surprise to the upside relative to AstraZeneca driving confidence in pandemic response and market recovery.”

The investment bank is confident in the safety profile of the vaccine given early trial data, “along with the prior success and safety profile demonstrated in their Ebola vaccine as well as in investigational use in HIV, RSV and Zika.”

A report by the Tony Blair Institute for Global Change, which is founded by the former British prime minister, calls the AstraZeneca and Johnson & Johnson jabs “the two workhorse vaccines” because these should be deliverable at scale and are easier to administer than the mRNA shots. 

With J&J’s technology, the vaccine is estimated to remain stable for at least three months at normal refrigerated temperatures so does not require new infrastructure to transport.

Expected timeline 

J&J completed enrolment of its 45,000-participant phase three clinical trial for its single-dose vaccine candidate on December 17. Preliminary data from the trial is expected to be available by the end of the month.

If the data indicates the vaccine is safe and effective, the company expects to submit an Emergency Use Authorization application to the U.S. Food and Drug Administration in February. Other health regulatory applications around the world are expected to be made in parallel.

Supply agreements  

The company has committed to selling the vaccine on a not-for-profit basis for emergency pandemic use. 

J&J entered into an agreement with the U.S. in August 2020 to deliver 100 million doses of the vaccine following approval or emergency use authorization by the FDA, and the option to purchase up to 200 million additional doses under a subsequent agreement.

The U.K. negotiated a deal in August to initially purchase 30 million doses of the J&J vaccine with an option to purchase up to 22 million additional doses. The EU signed a deal with J&J in October for the supply of up to 400 million doses.

J&J has also agreed to provide up to 500 million doses of its vaccine as part of an agreement in principle with The Vaccine Alliance (Gavi), which is responsible for equitable access to vaccines, including to lower-income countries via COVAX. These doses will be distributed through 2022 if the vaccine candidate is approved for use. 

“If J&J’s Ad26 platform is able to confer 80%+ efficacy via a single-dose regimen, given the vaccine’s favourable handling requirements and significant manufacturing scale, we would view this as a compelling outcome,” said Morgan Stanley.

As for what governments should do in the meantime, Jonathan Reiner, professor of medicine and surgery at the George Washington University School of Medicine & Health Sciences argues: “The J&J vaccine is why we should not abandon the two-dose strategy for Pfizer-BioNTech and Moderna. We’re likely to have all the vaccine we need. We need to focus on getting the vaccines into arms.”

https://www.cnbc.com/2021/01/11/johnson-johnson-covid-vaccine-analysts-are-cautiously-optimistic.html

BioMarin Has Mixed Longer Data on Hemophilia Gene Therapy

 BioMarin announced one-year follow-up data from a phase III study on its pipeline candidate, valoctocogene roxaparvovec in patients with hemophilia A.

With a mean follow-up of 71.6 weeks, data from the phase III study – GENEr8-1 – showed that treatment with a single dose (6e13 vg/kg) of the gene therapy candidate significantly reduced annualized bleeding rate (ABR) by 84% and the mean annualized factor VIII (a clotting protein) infusion rate in the rollover population by 99%. Meanwhile, valoctocogene roxaparvovec demonstrated superiority to current standard of care, FVIII prophylactic therapy. 

In a subset of patients who were dosed more than two years ago, the decline in Factor VIII expression was at a slower rate compared to that at the end of one year with continued hemostatic efficacy.

https://finance.yahoo.com/news/biomarin-bmrn-reveals-1-data-133701387.html

GeoVax Gets NIH Grant to Advance COVID-19 Vaccine Development

  GeoVax Labs, Inc. (NasdaqCM: GOVX), a biotechnology company developing immunotherapies and vaccines against cancers and infectious diseases, announced today that the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), has awarded the Company a Small Business Innovative Research (SBIR) grant in support of its development of a vaccine against SARS-CoV-2, the virus that causes COVID-19.

The Phase 1 grant, titled, “Preclinical Development of GV-MVA-VLP Vaccines Against COVID-19,” will support the ongoing design, construction and preclinical testing of GeoVax’s vaccine candidates in preparation for human clinical trials. The efficacy testing will be performed in collaboration with the University of Texas Medical Branch (UTMB).

GeoVax is leveraging its GV-MVA-VLP™ platform to address the global need for an effective and safe SARS-CoV-2 vaccine. Unique among other vaccines under development, the experimental GeoVax candidates are specifically designed to provide a broader and more long-lived level of protective immunity against SARS-CoV-2 while avoiding the potential side effects that can limit vaccine utility and acceptance. GeoVax’s vaccine candidates will be tested for antigen expression and genetic stability under conditions designed to simulate those in manufacturing, which will demonstrate the likely suitability of each vaccine construct as a candidate for full-scale production and clinical testing.

https://finance.yahoo.com/news/geovax-awarded-nih-grant-advance-140000738.html

China says WHO team to probe COVID-19 origins will arrive Thursday

 A World Health Organization (WHO) team of international experts tasked with investigating the origins of the COVID-19 pandemic will arrive in China on Jan. 14, Chinese authorities said on Monday.

Lack of authorisation from Beijing had delayed the arrival of the 10-strong team on a long-awaited mission to investigate early infections, in what China’s foreign ministry called a “misunderstanding”.

The National Health Commission, which announced the arrival date, delayed from its early January schedule, did not detail the team’s itinerary, however.

WHO chief Tedros Adhanom Ghebreyesus welcomed the news.

“We look forward to working closely with our (Chinese) counterparts on this critical mission to identify the virus source & its route of introduction to the human population,” Tedros wrote on Twitter.

He previously said he was “very disappointed” when experts were denied entry earlier this month, forcing two members of the team to turn back.

China has been accused of a cover-up that delayed its initial response, allowing the virus to spread since it first emerged in the central city of Wuhan late in 2019.

The United States has called for a “transparent” WHO-led investigation and criticised its terms, which allowed Chinese scientists to do the first phase of preliminary research.

Ahead of the trip, Beijing has been seeking to shape the narrative about when and where the pandemic began, with senior diplomat Wang Yi saying “more and more studies” showed it emerged in multiple regions.

A health expert affiliated with the WHO said expectations should be “very low” that the team will reach a conclusion from their trip to China.

While other countries continue to struggle with infection surges, China has aggressively doused flare-ups.

Sunday’s 103 new cases were mainland China’s biggest daily increase in more than five months, as new infections rise in the province of Hebei, surrounding the capital, Beijing.

Shijiazhuang, capital of Hebei, went into lockdown and Hebei closed down some sections of highways in the province to curb the spread of the virus.

Wangkui county, under the jurisdiction of Suihua city in Heilongjiang province, reported eight new asymptomatic cases and moved on Monday to close all non-essential businesses, banned people from leaving the city and blocked all non-essential traffic, state television reported on Monday.

https://www.reuters.com/article/us-health-coronavirus-china-who/china-says-who-team-to-probe-covid-19-origins-will-arrive-thursday-idUSKBN29G0BT