BioMarin Hemophilia Gene Therapy Meets Efficacy Endpoints At 2 Year Analysis

 BioMarin Pharmaceutical Inc (NASDAQ: BMRN) announced results from its ongoing Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, investigational gene therapy for severe hemophilia A.

• Annualized Bleeding Rate (ABR) was significantly reduced by 4.1 treated bleeds per year in the pre-specified primary analysis in participants from a prior non-interventional study.

• Valoctocogene roxaparvovec also significantly reduced the mean annualized Factor VIII infusion rate in the rollover population by 133 infusions per year or 98% from baseline.

• Overall, valoctocogene roxaparvovec has continued to be well tolerated.

• No participants developed inhibitors to Factor VIII, malignancy, or thromboembolic events.

• The European Medicines Agency validated BioMarin's resubmission of a marketing application. A Committee for Medicinal Products for Human Use and Committee for Advanced Therapies opinion is anticipated in 1H of 2022.

• In the U.S., BioMarin intends to submit two-year follow-up safety and efficacy data GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the FDA.

• The Company plans to meet with FDA to discuss the resubmission of a marketing application targeted for Q2 2022, followed by an expected six-month review by the FDA.

https://finance.yahoo.com/news/biomarins-hemophilia-gene-therapy-meets-112912447.html

Emergent BioSolutions prelim results, guidance

 

• Expects a re-baseline of operations with stable revenue contributions from core products and services business in 2022, guiding to total revenues of $1.45 billion at the midpoint and Adjusted EBITDA margin of 21.5% at the midpoint

• Reports preliminary 2021 total revenues of $1.78 billion at the midpoint and Adjusted EBITDA of $513 million, or 29%, at the midpoint, both in line with prior guidance given in November 2021

The Company is providing the following preliminary, unaudited financial results for full year 2021.

(in millions)	PRELIMINARY 2021 RESULTS (January 9, 2022)	PRIOR 2021 GUIDANCE (November 4, 2021)
Total Revenues	$1,770 - $1,790	$1,700 - $1,800
Net Income	$260 - $280	$260 - $295
Adjusted EBITDA (1)	$500 - $525	$500 - $550
Adjusted Net Income (1)	$315 - $335	$315 - $350

2022 FINANCIAL GUIDANCE

The Company is providing the following guidance of selected financial metrics for full year 2022.

(in millions)	FULL YEAR 2022 (As of January 9, 2022)
Total Revenues	$1,400 - $1,500
Adjusted EBITDA (1)	$280 - $340
Adjusted Net Income (1)	$135 - $180
Gross Margin %	47% - 51%
Product/Service Level Revenue
• Anthrax Vaccines	$280 - $300
• ACAM2000®	$190 - $210
• Nasal Naloxone Products*	$240 - $310
• CDMO Services	$430 - $480
• Other Products and Contracts and Grants	$200 - $260

* Includes revenues from the Company's branded NARCAN® Nasal Spray and revenues related to the authorized generic of NARCAN® Nasal Spray, a product licensed to Sandoz AG and launched in late 2021.

PRESENTATION WEBCAST
The Company will provide an update on the current business and discuss preliminary 2021 unaudited financial results, the financial guidance for 2022, and long-term goals during its presentation at the 40th Annual J.P. Morgan Healthcare Conference on January 10, 2022 at 8:15 AM Eastern time.

A live webcast of the presentation can be accessed through Emergent’s website. An on-demand replay of the webcast can also be accessed in the investors section after the presentation has concluded.

https://finance.yahoo.com/news/emergent-biosolutions-announces-2022-financial-190000094.html

Pluristem, Israeli Food Producer to Establish Cultured Food Platform

 

• Collaboration uniquely combines Pluristem’s technological expertise in mass-scale, cost-effective, consistent cell production with Tnuva’s power multipliers in consumer-packaged goods, research and development (R&D), consumer branding marketing, and sales and distribution with the goal to accelerate time to market
  
  

• Tnuva Group is Israel's largest food conglomerate, founded 96 years ago, with over $2 billion dollars in annual revenues
  
  

• The collaboration aligns with Pluristem’s strategy of leveraging its cell-based technology platform in key partnerships
  
  

• Pluristem to host online investor and analyst call on January 11 at 9:00 am EST (4:00 pm IST)

Pluristem to host online investor and analyst call on January 11, 2022 at 9:00 am EST (4:00 pm IST). Click here to register: https://veidan.activetrail.biz/pluristem

https://finance.yahoo.com/news/biotechnology-company-pluristem-israel-largest-080000542.html

Carisma to get $80M in cash and investment through new deal with Moderna

 Philadelphia cell therapy developer Carisma Therapeutics has entered into a research collaboration with Moderna that includes Carisma receiving an upfront payment of $45 million.

Moderna (NASDAQ: MRNA) will also invest $35 million in Carisma in the form of a convertible note, and in return get options for up to 12 targets for potential cancer therapies.

Under the partnership, the two companies will work together to discover, develop and commercialize potential cancer treatments. The treatments will incorporate Carisma's in vivo engineered chimeric antigen receptor monocyte (CAR-M) technology.

Based in Cambridge, Massachusetts, Moderna specializes in messenger RNA (mRNA) therapeutics including the Covid-19 vaccine it developed last year.

Carisma is pioneering cell therapies involving macrophages, which are a type of cell found in stationary form in tissue, or as a mobile white blood cell, at sites of infection. The company's researchers have developed a way to genetically engineer macrophage cells with chimeric antigen receptors, or CARs, to direct a therapy to tumor cells. Its in vivo approach means cells do not need to be manipulated outside the body then re-infused in patients.

Steven Kelly, CEO of Carisma, said Moderna’s deep expertise in mRNA and lipid nanoparticle technologies — which involve delivering genetic drugs without using deactivated viruses — "opens up a potentially game-changing opportunity for engineered macrophages."

"By combining Carisma’s expertise in engineered macrophage biology and Moderna’s pioneering in vivo mRNA delivery technologies, we are excited about the potential of this novel therapeutic approach for treating cancer," Kelly said.

Stephen Hoge, president of Moderna, said the partnership is part of his company's strategy to partner with companies with deep biological expertise to further expand the reach of Moderna’s technology.

Under the partnership agreement, Carisma, a University of Pennsylvania spinout founded in 2016, will also receive research funding and be eligible to receive undisclosed development, regulatory, and commercial milestone payments. Carisma will also get royalties on net sales of any products that are commercialized under the agreement.

Carisma will be responsible for the "discovery and optimization" of development candidates. Moderna will lead the clinical development and commercialization of therapeutics that result from the agreement.

https://www.bizjournals.com/philadelphia/news/2022/01/10/carisma-therapeutics-philadelphia-moderna-cancer.html

Lucira Prelims and Business Update

 Lucira Health, Inc. ("Lucira") (Nasdaq: LHDX), a medical technology company focused on the development and commercialization of transformative and innovative infectious disease test kits, today announced preliminary revenue results for the fourth quarter and full year 2021.

Preliminary, unaudited revenue for the fourth quarter 2021 is expected to be between $58 million and $60 million, reflecting growth of approximately 300% over the unaudited third quarter of 2021. Preliminary unaudited revenue for full year 2021, the first year of commercialization, is expected to be between $90 million and $92 million.

Based on sales pipeline visibility and manufacturing capacity, Lucira projects revenue for 2022 in excess of $450 million.

https://finance.yahoo.com/news/lucira-health-reports-preliminary-revenue-120000661.html

BioCryst soars on prelim revenue, outlook

 —ORLADEYO preliminary net revenue of $45.6 million for Q4 2021 and $122 million for FY 2021—

—ORLADEYO net revenue expected to more than double in 2022 to no less than $250 million; Company expects ORLADEYO to become the market leader in HAE prophylaxis therapy with peak sales of $1 billion—

—Enrollment of PNH patients has begun in REDEEM-1 and REDEEM-2 pivotal trials of BCX9930, oral Factor D inhibitor for complement-mediated diseases—

https://finance.yahoo.com/news/biocryst-announces-preliminary-full-2021-120000643.html

OrthoPediatrics prelim Q4 revenue seen up 31%

 OrthoPediatrics Corp. (“OrthoPediatrics” or the “Company”) (Nasdaq: KIDS), a company focused exclusively on advancing the field of pediatric orthopedics, today announced preliminary unaudited revenue for the fourth quarter and full year ended December 31, 2021.

Preliminary unaudited fourth quarter 2021 revenue is expected to be $24.8 million, up 31%, when compared to $18.9 million in the fourth quarter of 2020. Preliminary domestic revenue grew 11% while international revenue increased 363%. OrthoPediatrics’ preliminary unaudited full year 2021 revenue is expected to be $98.0 million, representing annual growth of 38%. Preliminary full year domestic revenue grew 24% while international revenue increased 150%.

The Company plans to release its fourth quarter and full year 2021 financial results in early March 2022. The quarterly and annual preliminary revenue estimates for 2021 included in this press release are prior to the completion of review and audit procedures by the Company’s independent registered public accounting firm and are therefore subject to adjustment.

https://finance.yahoo.com/news/orthopediatrics-announces-preliminary-unaudited-revenue-120100707.html