Agios Business Highlights and Second Quarter 2022 Financial Results

 Generated $3.1 Million in Net U.S. Revenue for First Full Quarter of PYRUKYND® (mitapivat) Sales –

– Brian Goff to Become Chief Executive Officer at Agios on August 8, 2022, Leveraging 30+ Years of Biopharma Experience, Including Significant Expertise in Rare Disease and Hematology; Current CEO Jackie Fouse to Become Chair of the Board –

– Actively Enrolling Patients in Five PYRUKYND® Pivotal Studies Across Thalassemia, Sickle Cell Disease and Pediatric Pyruvate Kinase (PK) Deficiency –

– $1.1 Billion of Cash, Cash Equivalents and Marketable Securities as of June 30, 2022 –

https://finance.yahoo.com/news/agios-reports-business-highlights-second-110000837.html

Adaptimmune Results and Business Update

  Reaffirming financial guidance; Company funded into early 2024 -

- ASCO data confirms afami-cel potential for synovial sarcoma; BLA submission on-track for Q4 2022 -

- Update from signal-finding Phase 1 SURPASS trial at the September ESMO 2022 Congress -

- Quarterly call to be held today, August 4, 2022, at 8:00 a.m. EDT (1:00 p.m. BST) -

The Company believes that its existing cash, cash equivalents and marketable securities, together with the additional payments under the Strategic Collaboration and License Agreement with Genentech, will fund the Company's current operations into early 2024, as further detailed in the Company's Quarterly Report on Form 10-Q for the three and six months ended June 30, 2022, to be filed with the Securities and Exchange Commission following this earnings release.

Conference Call Information

The Company will host a live teleconference and webcast to provide additional details at 8:00 a.m. EDT (1:00 p.m. BST) today, August 4, 2022. A live webcast of the conference call and replay can be accessed at https://www.gowebcasting.com/12004. Call in information is as follows: (800)-952-5114 (US or Canada) or +1 (416)-406-0743 (International and additional options available HERE). After placing the call, please ask to be joined into the Adaptimmune conference call and provide the confirmation code (5869059).

https://finance.yahoo.com/news/adaptimmune-reports-second-quarter-financial-113000359.html

Intellia Results and Recent Company Progress

 

• Completed dose-escalation portion of the ongoing Phase 1 study of NTLA-2001 in patients with transthyretin (ATTR) amyloidosis with cardiomyopathy; expects to present interim safety and serum TTR reduction data in 2H 2022
• Presented updated interim data from the dose-escalation portion of the polyneuropathy arm, establishing deep reductions of disease-causing protein were sustained through 12 months following a single dose of NTLA-2001
• Plans to present interim data for second in vivo CRISPR clinical candidate, NTLA-2002 for hereditary angioedema (HAE), in 2H 2022
• Moves to exclusively develop cell therapies leveraging its proprietary allogeneic platform; pivoting to an allogeneic version of NTLA-5001
• Ended the second quarter of 2022 with strong cash position of $907 million

https://www.biospace.com/article/releases/intellia-therapeutics-announces-second-quarter-2022-financial-results-and-highlights-recent-company-progress/

NanoViricides Starts Drug Development to Combat Monkeypox Virus

 NanoViricides, Inc. (NYSE American:NNVC) (the "Company"), a global leader in the development of highly effective antiviral therapies based on a novel nanomedicines platform, reports that it has begun drug development to combat the monkeypox virus.

Specifically, the Company has initiated a program to screen its library of broad-spectrum antiviral nanoviricides against certain poxviruses in order to develop broad-spectrum poxvirus therapeutics that act by mechanisms other than tecovirimat (TPOXX®).

https://finance.yahoo.com/news/nanoviricides-reports-begun-drug-development-104500770.html

Arbutus Results and Corporate Update

 Key milestones across our chronic hepatitis B virus (cHBV) and pan-coronavirus programs remain on track

Financially strong with a projected cash runway into the second quarter of 2024

Conference Call and Webcast Today at 8:45 AM ET

Arbutus will hold a conference call and webcast today, Thursday, August 4, 2022, at 8:45 AM Eastern Time to provide a corporate update. You can access a live webcast of the call through the Investors section of Arbutus’ website at www.arbutusbio.com. Alternatively, you can dial (800) 715-9871 or (646) 307-1963 and reference conference ID: 5109143.

An archived webcast will be available on the Arbutus website after the event. Alternatively, you may access a replay of the conference call by calling (800) 770-2030 or (609) 800-9909, and reference conference ID: 5109143

https://www.biospace.com/article/releases/arbutus-reports-second-quarter-2022-financial-results-and-provides-corporate-update/

BridgeBio Results and Business Update

 Reported positive interim Phase 2 data for infigratinib in achondroplasia demonstrating a mean increase in annualized height velocity (AHV) of 1.52 cm/year among all Cohort 4 children 5 years of age and older, and announced addition of a 5^th cohort to the trial 

- Reported positive Phase 2 data for encaleret in autosomal dominant hypocalcemia type 1 (ADH1) and announced design of the Phase 3 pivotal study, which is expected to begin later this year

- Selected a next-generation KRAS G12C dual inhibitor development candidate and plans to be in the clinic in mid-2023

- Presented updated results from the open-label extension of the Phase 2 study of acoramidis in transthyretin amyloid cardiomyopathy (ATTR-CM), which continue to suggest long-term tolerability of acoramidis and stabilization of disease progression in ATTR-CM

- Reported positive data for five early-to-mid-stage genetic disease programs in pipeline, including primary hyperoxaluria type 1 (PH1) and recurrent kidney stone formation; Canavan disease; pantothenate kinase-associated neurodegeneration (PKAN) and organic acidemias; recessive dystrophic epidermolysis bullosa (RDEB); and venous, lymphatic and mixed venolymphatic malformations (VM, LM and VLM)

- Received positive opinion from the Committee for Medicinal Products for Human Use (CHMP) recommending approval in the European Union (EU) for NULIBRY® (fosdenopterin) for the treatment of molybdenum cofactor deficiency (MoCD) Type A

- Entered into exclusive license agreement with Bristol Myers Squibb for BBP-398, a potentially best-in-class SHP2 inhibitor, in oncology; BridgeBio is eligible to receive up to $905 million, including an upfront payment of $90 million received during the quarter and up to $815 million in additional milestone payments and royalties 

- Announced sale of Priority Review Voucher (PRV) for $110 million  

- Secured a two-year deferral for first principal payment on the Company’s senior debt 

- Ended quarter with $688.6million in cash, cash equivalents and marketable securities, providing financial runway into 2024 

https://www.biospace.com/article/releases/bridgebio-pharma-inc-reports-second-quarter-2022-financial-results-and-business-update/

Karyopharm Results and Recent Company Progress

 Achieved Second Quarter 2022 Total Revenue of $39.7 Million, Up 76% Versus Second Quarter 2021;

XPOVIO® (selinexor) Net Product Revenue of $29.0 Million, a 44% Increase Over Q2 2021 –

–  Received  Full Marketing Authorization from the European Commission Expanding Indication for NEXPOVIO® (selinexor) for Treatment of Adults with Multiple Myeloma Who Have Received at Least One Prior Therapy –

– Clinical Updates from Phase 1/2 Study of Selinexor in Combination with Ruxolitinib in Treatment-Naïve Myelofibrosis and Interim Data from Phase 2 Study Evaluating Eltanexor in Relapsed/Refractory MDS Expected in 2H 2022 –
 

– Company Provides Full Year 2022 Total Revenue Guidance of $155 Million to $165 Million Including Revised XPOVIO Net Product Revenue of $120 Million to $130 Million and non-GAAP R&D and SG&A Expense Guidance of $250 Million to $265 Million; Re-iterates Cash Runway to Early 2024 –

– Conference Call Scheduled for Today at 8:00 a.m. ET –

Karyopharm will host a conference call today, August 4, 2022, at 8:00 a.m. Eastern Time, to discuss the second quarter 2022 financial results and provide other business highlights. To access the conference call, please dial (888) 349-0102 (local) or (412) 902-4299 (international) at least 10 minutes prior to the start time and ask to be joined into the Karyopharm Therapeutics call. A live audio webcast of the call, along with accompanying slides, will be available under "Events & Presentations" in the Investor section of the Company's website, http://investors.karyopharm.com/events-presentations. An archived webcast will be available on the Company's website approximately two hours after the event.

https://www.biospace.com/article/releases/karyopharm-reports-second-quarter-2022-financial-results-and-highlights-recent-company-progress/