Rare

Protara Therapeutics Announces Third Quarter 2023 Financial Results and Business Update

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金曜日, 11月 3, 2023
CIS, Research, LM, LMS, Food, Rare, Injection, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Carcinoma in situ, Microcystic lymphatic malformation, Protaras, Cancer, Investment, BCG, Patient, Therapy, Macrocyst, Pharmaceutical industry, NMIBC

The Company remains on track to report preliminary results in the first half of 2024.

Key Points: 
  • The Company remains on track to report preliminary results in the first half of 2024.
  • General and administrative expenses for both the third quarter of 2023 and 2022 were $4.5 million.
  • For the third quarter of 2023, Protara reported a net loss of $9.9 million, or $0.87 per share, compared with a net loss of $7.7 million, or $0.68 per share, for the same period in 2022.
  • Net loss for the third quarter of 2023 included approximately $1.4 million of stock-based compensation expenses.

The North America Rare Disease Summit focused on the need for centralized data to improve the lives of 30 million people living with rare conditions

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木曜日, 11月 2, 2023
FDA, Rare, Organization, Translational research, Research, Access, Caregiver, Patient, Diagnosis, Translation, Pharmaceutical industry

NEW YORK, Nov. 02, 2023 (GLOBE NEWSWIRE) -- The North America Rare Disease Summit, organized by Bamberg Health, gathered over 40 specialists on Rare Diseases.

Key Points: 
  • NEW YORK, Nov. 02, 2023 (GLOBE NEWSWIRE) -- The North America Rare Disease Summit, organized by Bamberg Health, gathered over 40 specialists on Rare Diseases.
  • The debate focused on the role of public awareness and patients, government initiatives, access and financing to research and treatments, and the use of new tools in accelerating processes.
  • “We’re changing things inside the FDA, to accelerate the response, but it’s huge and it will take longer than we’d like”.
  • “We’re talking about millions of dollars and, now, most are funded by patient advocates, because for industry is not profitable.

Catalyst Pharmaceuticals Receives Two New U.S. Patent Allowances For FIRDAPSE®

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木曜日, 11月 2, 2023
CORAL, Fasting, Patent, Rare, United States Patent and Trademark Office, Orange Book, Catalyst Pharmaceuticals, Amifampridine, Approved Drug Products with Therapeutic Equivalence Evaluations, Lambert–Eaton myasthenic syndrome, Tablet, Patient, NAT2, Pharmaceutical industry, Catalysis

FIRDAPSE is the only approved treatment available in the U.S. for Lambert-Eaton myasthenic syndrome.

Key Points: 
  • FIRDAPSE is the only approved treatment available in the U.S. for Lambert-Eaton myasthenic syndrome.
  • "We are very pleased to receive these patent notifications from the USPTO.
  • "These patent allowances strengthen our cumulative understanding of the uniqueness of FIRDAPSE and bolster its strong intellectual property estate offering enhanced patent protection," stated Dr. Steven Miller, Chief Operating Officer and Chief Scientific Officer of Catalyst.
  • Preparations are already in motion to include these additional FIRDAPSE patents in the FDA Orange Book post-grant, bringing the total listed patent count to eight."

X4 Pharmaceuticals to Report Third-Quarter 2023 Financial Results and Host a Conference Call and Webcast on November 9, 2023

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木曜日, 11月 2, 2023
ID, Immune system, Webcast, Rare, Rare-earth element, Video game, Broadcasting

The company will host a conference call and webcast on the same day at 8:30 a.m.

Key Points: 
  • The company will host a conference call and webcast on the same day at 8:30 a.m.
  • The conference call can be accessed by dialing 1-877-451-6152 from the United States or 1-201-389-0879 internationally, followed by the conference ID: 13741400.
  • The live webcast will be accessible through the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com .
  • Following the completion of the call, a webcast replay will be available on the website.

Agios Reports Business Highlights and Third Quarter 2023 Financial Results

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木曜日, 11月 2, 2023
Anti-transglutaminase antibodies, Research, RISE, PKU, Research and development, IND, AGIO, Rare, Sickle cell disease, MDS, Public expenditure, Enrollment, SG, Pyruvate kinase deficiency, R, Society, Phenylketonuria, ASH, Thalassemia, PK, Security, Abstract, Phenylalanine hydroxylase, TMPRSS6, Agios Pharmaceuticals, PAH, Agios, Patient, Administration, Nursing, Cryptocurrency, Pharmaceutical industry, Medical device, Video game, Alnylam Pharmaceuticals

CAMBRIDGE, Mass., Nov. 02, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today reported business highlights and financial results for the third quarter ended September 30, 2023.

Key Points: 
  • Launch: Generated $7.4 million in U.S. net revenue for the third quarter of 2023, a 10 percent increase over the second quarter of 2023.
  • A total of 160 unique patients have completed prescription enrollment forms, representing an increase of 9 percent over the second quarter of 2023.
  • Net Loss: Net loss was $91.3 million for the third quarter of 2023 compared to $81.7 million for the third quarter of 2022.
  • ET to discuss third quarter 2023 financial results and recent business activities.

X4 Pharmaceuticals Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

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水曜日, 11月 1, 2023
Immune system, Employment, Rare, Pharmaceutical industry, Health insurance

BOSTON, Nov. 01, 2023 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced that, effective on October 31, 2023, the company issued inducement awards to new employees under the X4 Pharmaceuticals, Inc. 2019 Inducement Equity Incentive Plan (the “2019 Inducement Plan”).

Key Points: 
  • BOSTON, Nov. 01, 2023 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced that, effective on October 31, 2023, the company issued inducement awards to new employees under the X4 Pharmaceuticals, Inc. 2019 Inducement Equity Incentive Plan (the “2019 Inducement Plan”).
  • The 2019 Inducement Plan is used exclusively for the grant of equity awards to individuals who were not previously an employee of X4.
  • The inducement awards consist of options to purchase an aggregate of 454,500 shares of X4’s common stock.
  • These stock awards were granted as an inducement material to the new employees entering into employment with X4 in accordance with Nasdaq Listing Rule 5635(c)(4) and were approved by X4’s Compensation Committee of the Board of Directors.

Cellectis Announces Strategic Collaboration and Investment Agreements with AstraZeneca

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水曜日, 11月 1, 2023
Euronext, Gene editing, AstraZeneca, IND, Therapy, Rare, Alexion, Doctor of Philosophy, Research, Initial, MOU, UCART123, File, Collaboration, Investment, IP, Patient, Pharmaceutical industry, Security (finance), Cellectis

Under the terms of the Collaboration Agreement, AstraZeneca will leverage Cellectis’ proprietary gene editing technologies and manufacturing capabilities to design novel cell and gene therapy candidate products.

Key Points: 
  • Under the terms of the Collaboration Agreement, AstraZeneca will leverage Cellectis’ proprietary gene editing technologies and manufacturing capabilities to design novel cell and gene therapy candidate products.
  • As part of the Collaboration Agreement, 25 genetic targets have been exclusively reserved for AstraZeneca, from which up to 10 candidate products could be explored for development.
  • Pursuant to the Collaboration Agreement, Cellectis’ research costs under the collaboration will be funded by AstraZeneca and Cellectis will receive an upfront payment of $25M.
  • As a condition to the signing of the Collaboration Agreement, AstraZeneca has agreed to make an initial equity investment of $80M in Cellectis by subscribing for 16,000,000 ordinary shares, at a price of $5.00 per share (the “Initial Investment”).

Spexis Announces Changes to the Executive Committee

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水曜日, 11月 1, 2023
Knowledge, Railway Executive Committee, Trust, LR, Allschwil, Rare, Executive Committee, Therapy, Human resources, Management, Spexy Beast

Allschwil, Switzerland, Oct. 31, 2023 (GLOBE NEWSWIRE) -- Spexis AG (SIX: SPEX), a clinical-stage biopharmaceutical company focused on macrocycle therapeutics for rare diseases and oncology, today announced that Hernan Levett, currently Chief Financial Officer, has decided to pursue a new career opportunity outside the company. He will continue to support the company as a strategic advisor on financial transactions and will depart the company effective December 1, 2023. Martin Jakobovic, currently Head of Finance, will succeed Hernan Levett as interim Chief Financial Officer joining the Executive Committee on December 1, 2023. Additionally, Muriel Fleming, currently Head of Human Resources, will assume additional responsibilities related to administration and facilities, as well as joining the Executive Committee of Spexis also on December 1, 2023.

Key Points: 
  • He will continue to support the company as a strategic advisor on financial transactions and will depart the company effective December 1, 2023.
  • Martin Jakobovic, currently Head of Finance, will succeed Hernan Levett as interim Chief Financial Officer joining the Executive Committee on December 1, 2023.
  • Additionally, Muriel Fleming, currently Head of Human Resources, will assume additional responsibilities related to administration and facilities, as well as joining the Executive Committee of Spexis also on December 1, 2023.
  • “I would like to thank Jeff, the board of directors, our shareholders, and the entire Spexis team for their trust, support, and commitment.

Spero Therapeutics to Present at Upcoming November Investor Conferences

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水曜日, 11月 1, 2023
W Miami Hotel, Rare, Evercore, MDR, Conference, Infection, Lotte New York Palace Hotel, Kimpton, Spero, Therapy, Pharmaceutical industry, Online shopping

CAMBRIDGE, Mass., Nov. 01, 2023 (GLOBE NEWSWIRE) -- Spero Therapeutics, Inc. (Nasdaq: SPRO) (Spero), a multi-asset clinical-stage biopharmaceutical company, focused on identifying, developing and commercializing treatments in high unmet need areas involving rare diseases and multi-drug resistant (MDR) bacterial infections, today announced that Sath Shukla, President and Chief Executive Officer, will be available for one-on-one meetings at the Truist Securities BioPharma Symposium, to be held at the Lotte New York Palace in New York, NY, on November 8-9, 2023 and will present and be available for one-on-one meetings at the 6th Annual Evercore ISI HealthCONx Conference to be held at the Kimpton EPIC Miami Hotel in Miami, FL, on November 28-30, 2023.

Key Points: 
  • CAMBRIDGE, Mass., Nov. 01, 2023 (GLOBE NEWSWIRE) -- Spero Therapeutics, Inc. (Nasdaq: SPRO) (Spero), a multi-asset clinical-stage biopharmaceutical company, focused on identifying, developing and commercializing treatments in high unmet need areas involving rare diseases and multi-drug resistant (MDR) bacterial infections, today announced that Sath Shukla, President and Chief Executive Officer, will be available for one-on-one meetings at the Truist Securities BioPharma Symposium, to be held at the Lotte New York Palace in New York, NY, on November 8-9, 2023 and will present and be available for one-on-one meetings at the 6th Annual Evercore ISI HealthCONx Conference to be held at the Kimpton EPIC Miami Hotel in Miami, FL, on November 28-30, 2023.
  • Details are as follows:
    The webcast for the Evercore ISI event may also be accessed through Spero Therapeutics’ website ( www.sperotherapeutics.com ) on the “Events and Presentations” page under the “Connect” tab.
  • Replays will be available on the website following the conclusion of each event.

Spero Therapeutics Appoints Esther Rajavelu as Chief Financial Officer and Chief Business Officer

Retrieved on: 
水曜日, 11月 1, 2023
Principal, Research, DSM-IV codes, Bank, RSU, Rare, Compensated emancipation, Biotechnology, BofA Securities, MDR, Form, University, Infection, MBA, BA, Wharton School of the University of Pennsylvania, Wesleyan University, Employment, Wall Street, Interim, Deutsche Bank, UBS, CFO, Towers Perrin, M&A, Therapy, Patient, CBO, Fortune 500, Pharmaceutical industry, Spero

CAMBRIDGE, Mass., Nov. 01, 2023 (GLOBE NEWSWIRE) -- Spero Therapeutics, Inc. (Nasdaq: SPRO), a multi-asset clinical-stage biopharmaceutical company, focused on identifying, developing, and commercializing treatments in high unmet need areas involving rare diseases and multi-drug resistant (MDR) bacterial infections, today announced the appointment of Esther Rajavelu as Chief Financial Officer and Chief Business Officer.

Key Points: 
  • CAMBRIDGE, Mass., Nov. 01, 2023 (GLOBE NEWSWIRE) -- Spero Therapeutics, Inc. (Nasdaq: SPRO), a multi-asset clinical-stage biopharmaceutical company, focused on identifying, developing, and commercializing treatments in high unmet need areas involving rare diseases and multi-drug resistant (MDR) bacterial infections, today announced the appointment of Esther Rajavelu as Chief Financial Officer and Chief Business Officer.
  • Ms. Rajavelu will succeed Stephen DiPalma, who has served as the company’s Interim CFO since August 1, 2023.
  • Esther brings a proven track record in corporate finance, from both industry and Wall Street, bringing expertise in life science growth strategies, investor relations, financing, and M&A,” said Sath Shukla, Spero’s Chief Executive Officer.
  • Ms. Rajavelu will formally assume the role of Principal Financial Officer on the day after the filing of such Form 10-Q.