Sionna Therapeutics Announces Phase 1 Initiation for SION-109 in Cystic Fibrosis

– SION-109 is a small molecule targeting the interface between the ICL4 region and the NBD1 domain of the CFTR protein; designed to complement the company’s NBD1 modulator portfolio –

– First subject dosed in Phase 1 clinical trial –

Boston, MA, January 24, 2024 — Sionna Therapeutics, a life sciences company dedicated to developing highly effective and differentiated treatments for cystic fibrosis (CF), announced that the first healthy subject has been dosed in a Phase 1 clinical trial of SION-109 following clearance of its Investigational New Drug application (IND) by the U.S. Food and Drug Administration (FDA).

SION-109 is a small molecule designed to target the interface between the intracellular loop 4 (ICL4) region and the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. It is a complementary mechanism to the company’s portfolio of compounds directly targeting NBD1, including SION-638, the first-in-class NBD1 stabilizer now in Phase 1.

“Our ability to target NBD1 correction is truly novel, and with SION-109 now entering clinical development, we have the opportunity to develop proprietary combination treatments that have the potential to achieve full CFTR correction,” said Mike Cloonan, President and Chief Executive Officer of Sionna. “Advancing our second investigational drug into Phase 1 is an achievement made possible by the tireless work of our research and development teams, who have deep experience in CF and a sharp focus on advancing these programs rapidly to deliver new options for people living with CF and their families.”

CF is caused by genetic mutations in the CFTR protein, an epithelial ion channel that is essential for producing healthy, freely flowing mucus in the airways, digestive system, and other organs. The most common mutation in CFTR, ΔF508, causes NBD1 to unfold at body temperature and severely impairs CFTR function. Complementary targets including ICL4 may enable combination treatments with the potential for full CFTR correction. It is estimated that 90 percent of people living with CF have the genetic mutation ΔF508... Sionna Therapeutics' Press Release - 

Ampliphi Biosciences : Encouraging Pre-Clinical Data on the Use of Phage-Based Therapies to Treat Bacterial Lung Infections

October 18, 2012 - AmpliPhi Biosciences Corporation (APHB.PK) [‘AmpliPhi’] presented encouraging data from a pre-clinical animal model study of acute infection with Pseudomonas aeruginosa at the 26th Annual North America Cystic Fibrosis Foundation Conference held 11-13 October in Florida, USA (NACFC). The data generated in collaboration with scientists at Institut Pasteur in Paris and using AmpliPhi’s phage discovery platform show that the company’s proprietary mix of bacteriophage is able to clear lung infection with efficacy comparable or superior to a high dose of an antibiotic shown to be effective against the infecting bacteria in the laboratory. Furthermore, the bacteriophage therapy rapidly eradicates bacteria in the oropharynx and lungs of the infected mice. In fact, its action appears to be faster than the antibiotic, showing efficacy at just six hours, with reduced dissemination of the infection... source : AmpliPhi Biosciences' press release -

Chugai Pharmaceutical : Approval of an Orphan Drug, “Pulmozyme®,” a Recombinant Human Deoxyribonuclease I (rhDNase)

March 30, 2012 (Tokyo) - Chugai Pharmaceutical Co., Ltd. [Main Office: Chuo-ku, Tokyo. Chairman & CEO: Osamu Nagayama (hereafter, “Chugai”)] announced that it has obtained approval on March 30, 2012, from the Ministry of Health, Labour and Welfare (MHLW), for Pulmozyme® Inhalation Solution 2.5mg (hereafter, “Pulmozyme®”)[recombinant human deoxyribonuclease I (rhDNase), generic name: dornase alfa] for the indication of “improvement of pulmonary function in patients with cystic fibrosis.” Pulmozyme® is designated as an orphan drug for this indication by the MHLW.

As a result of the evaluation by the “Review Committee on Unapproved Drugs and Indications with High Medical Needs*” held on April 18, 2011, it was concluded that it is reasonable that dornase alfa be filed for approval in this indication based on available data, and on July 15, 2011, the filing was made using overseas data. In overseas clinical studies, administration of dornase alfa by inhalation is confirmed to be effective for the improvement of pulmonary function and reduction of the risk of serious infection of the respiratory tract in cystic fibrosis patients, compared to placebo... Chugai Pharmaceutical's Press Release -

NovaBiotics : plans for cystic fibrosis drug after EU grants orphan status

20th December 2011 - NovaBiotics reveals plans for cystic fibrosis drug after EU grants orphan status -

Bolstered by the an EU orphan drug designation for its drug candidate for cystic fibrosis, Lynovex (cysteamine), UK-based NovaBiotics revealed to Scrip that it plans to begin a first-in-man, safety/tolerability and evidence of efficacy study of the product in 2012, followed by other trials in 2013.

The company announced that the Committee on Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has granted orphan status to the novel dual mucolytic-antibacterial drug for the treatment of cystic fibrosis and added that it is "currently in discussions with a number of interested parties with a view to licensing or co-developing the drug".

NovaBiotics stated it would "now progress with follow-on orphan applications for paediatric use and also applications for orphan drug designation in the US".

"This novel drug candidate is expected to be tested in patients in 2012," the biotech firm highlighted. It told Scrip that the study slated for next year would test Lynovex when used in conjunction with conventional inhaled ABX (antibiotic) therapies in the first instance. In 2013, NovaBiotics said it would initiate a second and third trial to further assess efficacy.

The company confirmed that it intends to partner the product, and "as per our core business model [this is] most likely after the second clinical study". With respect to its finances, it said: "NovaBiotics remains fully equity funded (still pre-revenue) and this is how development has been/will be funded in the short-term for Lynovex, but as the programme develops towards final clinical studies, revenue from Novexatin and Luminaderm commercialisation deals will be reinvested into the Lynovex programme."... NovaBiotics' Press Release -

Novartis : Becoming Christopher a fictional video trailer about living with Cycstic Fibrosis

Lamellar Biomedical : First Orphan Drug Licence

June 16, 2011 - Provides 10-year Market Exclusivity for the treatment of Cystic Fibrosis - Glasgow biopharmaceutical firm, Lamellar Biomedical Ltd, launched four years ago to develop a new class of therapies for the prevention, treatment and control of severe respiratory disease, announced that their lead clinical candidate LMS-611 for the treatment of patients with cystic fibrosis has received Orphan Drug Designation from the European Commission. The designation follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) within the European Medicines Agency (EMA) earlier this month.

“The designation of LMS-611 as an orphan drug by the European Medicines Agency underscores the importance of developing effective treatments for patients with cystic fibrosis,” said Dr Iain McDougall, Chief Business Officer of Lamellar Biomedical Ltd. “To date we have been extremely encouraged by LMS-611’s preclinical results, and we continue to believe we are developing a therapy which will prove to be both safe and effective with the potential to offer a brand new way of treating the disease when we enter clinical trials early next year.”

Drugs for rare diseases are called “orphans” because the small number of patients they would treat means they are often not developed by the pharmaceutical industry. Today’s designation will make it easier for Lamellar Biomedical Ltd to attract both investment and development partners and facilitate LMS-611’s entry into clinical use. Lamellar Biomedical Ltd and its partners will also have ten years to exclusively market the Drug in the European Union.

LMS-611 is developed from Lamellar’s proprietary inhaled LamellasomeTM delivery platform. The LamellasomeTM is based on the biological and biophysical resemblance to the natural lamellar bodies found in healthy lungs, and found to be absent in cystic fibrosis patients. By mimicking this missing body responsible for normal lung function, Lamellar Biomedical Ltd have already established an impressive package of preclinical data demonstrating significant potential as a mucus-altering treatment with the ability to fight and prevent infection... Lamellar Biomedical's Press Release -

Insmed : ARIKACE® Demonstrates Sustained Benefit Through Six Cycles of Treatment for Cystic Fibrosis Patients With Pseudomonas Lung Infections

June 10, 2011 - Insmed Incorporated (Nasdaq CM: INSM), a biopharmaceutical company, announced positive data through six treatment cycles (72 weeks total duration) of its Phase 2 clinical trial program for ARIKACE® (liposomal amikacin for inhalation) in cystic fibrosis (CF) patients with Pseudomonas lung infections. The data were presented at the 34th European Cystic Fibrosis Conference in Hamburg, Germany, by Predrag Minic, M.D., Professor of Pediatrics and Head of Pediatrics Pulmonology Department, Mother and Child Health Institute, Belgrade, Serbia, and co-lead investigator of the study.

The open label Phase 2 study was designed to evaluate ARIKACE over multiple treatment cycles in CF patients with Pseudomonas lung infections. The study enrolled 49 patients to receive ARIKACE 560 mg once daily for 28 days of therapy, followed by a 56-day off-treatment observation period. ARIKACE was administered using an optimized, investigational eFlow® Nebulizer System (PARI Pharma GmbH).

The data demonstrated that ARIKACE, delivered once-daily for 28 consecutive days, followed by 56 days off-treatment, for a total of six cycles, resulted in statistically significant improvement in lung function that was sustained over a 72 week period. Specifically, inhalation of 560 mg of ARIKACE produced a mean increase in pulmonary function (FEV1) of 11.7% at the end of the 28 day treatment period of the sixth cycle (p<0.0001).

During the course of the study, FEV1 improvement was also sustained at the end of 56 days off-treatment in each of cycles one through six, with an estimated relative increase in FEV1 of 5.7% (95% CI +3.0%, +8.5%; p=0.0001). Overall, ARIKACE was well-tolerated during all six cycles, with adverse events reported as consistent with those expected in a population of CF patients receiving inhaled medicines... Insmed's Press Release -

Centocor Ortho Biotech Acquires RespiVert Strengthens Pulmonary Focus

June 1, 2010 - Acquisition Bolsters Pulmonary Portfolio and Capabilities, Accessing Promising Pipeline of First-in-Class Compounds and Renowned Team of Respiratory Scientists – Centocor Ortho Biotech Inc. announced that it has acquired RespiVert Ltd., a privately held drug discovery company focused on developing small-molecule, inhaled therapies for the treatment of pulmonary diseases. The company’s lead compounds, RV-568 and RV-1088, narrow spectrum kinase inhibitors with a unique profile of anti-inflammatory activities, are progressing into clinical development as potential first-in-class treatments for moderate to severe asthma, Chronic Obstructive Pulmonary Disease (COPD) and Cystic Fibrosis (CF).


The clinical development of RV-568 and RV-1088 will be led by RespiVert in collaboration with scientists at Centocor Research and Development, Inc. The company is not disclosing financial terms... Centocor Ortho Biotech [JNJ] -

Johnson & Johnson Pharmaceutical Research and Development : FDA Approves New Drug Application for PANCREAZE

April 13,2010 - Pancreatic Enzyme Therapy Approved by FDA to Treat Exocrine Pancreatic Insufficiency - Johnson & Johnson Pharmaceutical Research and Development (J&JPRD) announced that the U.S. Food and Drug Administration (FDA) has approved a new drug application (NDA) for PANCREAZE™ (pancrelipase) delayed-release capsules for the treatment of exocrine pancreatic insufficiency due to cystic fibrosis or other conditions. Exocrine pancreatic insufficiency is the inability to properly digest and absorb food due to a lack of enzymes made by the pancreas that are important in the digestion of fats, proteins, and sugars... Johnson & Johnson's Press Release -

Bayer HealthCare : FDA Grants Orphan Drug Status to Ciprofloxacin Dry Powder Inhaler for the Treatment of Cystic Fibrosis

March 11, 2010 – Bayer Schering Pharma AG, Germany, announced that an orphan drug designation has been granted by the U.S. Food and Drug Administration (FDA) for the ciprofloxacin dry powder inhaler (DPI) for the management of chronic pulmonary infections due to Pseudomonas aeruginosa in cystic fibrosis (CF) patients. A similar designation has already been granted by the European Medicines Agency (EMA)... Bayer HealthCare's Press Release -

Genta : to Support Initiation of New Clinical Trial using Ganite® as Treatment for Life-Threatening Infections in Patients with Cystic Fibrosis

December 2, 2009 - Genta Incorporated (OTCBB: GETA) announced that the Company will supply Ganite® (gallium nitrate injection) for a new clinical trial that will be initiated in patients with cystic fibrosis (CF) who may develop serious infections.

Infection is the most common cause of death in CF patients. Frequently, these infections are due to bacteria known as Pseudomonas aeruginosa, and patients are commonly treated with years of antibiotic therapy to control such infections. However, prolonged antibiotic use has greatly increased resistance due to genetic mutations. In addition, Pseudomonas has evolved protective mechanisms known as "biofilms" that encase colonies of bacteria and prevent antibiotics from reaching infection sites. Bacteria in biofilms are far more resistant to being killed. Recent information has suggested that gallium may be used as an anti-bacterial agent against Pseudomonas, in part due to its ability to disrupt biofilms... Genta's Press Release -

Eurand : ZENPEP(TM) (Pancrelipase) Delayed-Release Capsules

Nov 30, 2009 - First and Only FDA-Approved Pancreatic Enzyme Product Clinically Proven in Patients as Young as One Year Old - Eurand N.V. (NASDAQ: EURX), a global specialty pharmaceutical company, announced the commercial availability of ZENPEP(TM) (pancrelipase) Delayed-Release Capsules, a pancreatic enzyme product (PEP) indicated for the treatment of exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF) or other conditions. The U.S. Food and Drug Administration (FDA) approved ZENPEP on August 27, 2009.

Eurand has also completed the hiring, training and deployment of its sales organization to promote ZENPEP, including a specialty CF sales force composed of 16 Eurand sales people targeting the 120 accredited cystic fibrosis centers, satellites and other specialists who treat CF. In addition, the Company has contracted with Innovex, a Quintiles company, to promote ZENPEP to PEP prescribers who commonly treat patients with EPI associated with other conditions such as chronic pancreatitis, pancreatic cancer and gastric surgery. These 49 Innovex sales professionals will call on key gastroenterologists, internal medicine specialists, family-practice physicians and oncologists... Eurand's Press Release -

Sequenom : Launch of SensiGene Cystic Fibrosis Carrier Screening Test

Sept. 17, 2009 - SEQUENOM, Inc. (Nasdaq: SQNM) announced that the company's first laboratory developed test, the SensiGene(TM) Cystic Fibrosis (CF) Carrier Screening test, has been launched. This test is available exclusively through the Sequenom Center for Molecular Medicine, a next-generation CLIA (Clinical Laboratory Improvement Amendments, 1988) molecular diagnostic laboratory and a wholly-owned subsidiary of Sequenom, Inc... Sequenom's Press Release -

Osmetech : FDA clears its Cystic Fibrosis Genotyping Test

9 July 2009 - Osmetech plc (OMH.L), the fast growing international molecular diagnostics company announces that it has received US Food & Drug Administration (‘FDA’) 510(k) clearance for its eSensor® Cystic Fibrosis Genotyping Test for use on the eSensor® XT-8 System. This multiplex test is used for cystic fibrosis carrier screening for adults of reproductive age, as an aid in newborn screening for cystic fibrosis, and as a confirmatory diagnostic test for cystic fibrosis in newborns and children... Osmetech's Press Release -

Alnara Pharmaceuticals Licenses Worldwide Rights to Liprotamase from Cystic Fibrosis Foundation

March 25, 2009 - Novel, Oral, Pancreatic Enzyme Replacement Therapy with Phase 3 Data Offers Potential Advantages – Alnara Pharmaceuticals, a pharmaceutical company developing novel, non-systemic orally-delivered protein therapeutics for the treatment of metabolic diseases, announced a licensing agreement with the Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the nonprofit affiliate of the Cystic Fibrosis Foundation, for the worldwide rights to liprotamase, a novel, oral, porcine-free pancreatic enzyme replacement therapy (PERT) that has already completed a Phase 3 efficacy trial. Under the terms of the agreement Alnara will obtain exclusive worldwide rights to develop and market liprotamase.
Liprotamase (formerly known as ALTU-135 and Trizytek) is designed to treat malabsorption that is the result of pancreatic insufficiency associated with conditions like cystic fibrosis (CF) and chronic pancreatitis (CP), in which patients require a significant amount of pancreatic enzyme supplements with each meal. Patients with pancreatic insufficiency cannot properly absorb and digest food due to their disease. Pancreatic enzyme replacement therapy is a lifesaving treatment involving the administration of pancreatic enzymes, and liprotamase, a novel, oral, porcine-free treatment, offers potential advantages over the existing standard of care. Currently, patients are treated with porcine-derived pancreatic enzymes, which vary in potency and stability and have a high pill burden. Alnara believes liprotamase has the potential to overcome these challenges by providing a first-in-class and best-in-class PERT...

...

About Alnara
Alnara Pharmaceuticals, Inc. is dedicated to developing novel protein therapeutics for the treatment of metabolic diseases. The company's innovative approach focuses on designing effective protein therapies that can be orally delivered directly to the gastrointestinal tract without being absorbed into the bloodstream. The company is committed to bringing breakthrough new treatments to patients with unmet medical needs. Based in Cambridge, Massachusetts, Alnara is backed by top-tier venture investors, including Frazier Healthcare Ventures, Third Rock Ventures and Bessemer Venture Partners... [PDF] Alnara Pharmaceuticals' Press Release -

Novartis and Nektar Therapeutics Pulmonary Business

Novartis' Press Release :
Novartis to acquire Nektar Therapeutics pulmonary business unit, strengthening respiratory drug delivery capabilities
October 21, 2008 - Novartis has strengthened its capabilities for developing medicines that can be delivered via the lungs through a definitive agreement to acquire the pulmonary drug delivery business unit of Nektar Therapeutics (NASDAQ: NKTR) for USD 115 million in cash.

Important capabilities gained from Nektar, which has approximately 140 associates who will join Novartis and remain in San Carlos, California, include product formulation and delivery expertise along with a broad device platform. This transaction excludes Nektar's inhalation programs for insulin, vancomycin, ciprofloxacin and amikacin.

Novartis will utilize these skills to build on existing capabilities and accelerate its pipeline targeting life-threatening diseases such as chronic obstructive pulmonary disease (COPD), asthma and cystic fibrosis as well as the life-cycle management of late-stage development projects that are closer to regulatory submissions. Potential applications will also be assessed in other areas of the Novartis healthcare portfolio... Novartis' Press Release -


Nektar's Press Release :
Nektar Sells Pulmonary Business to Novartis For $115 Million and Nektar Retains Key Pulmonary Programs Including Amikacin Inhale, Inhaled Vancomycin, and Ciprofloxacin Inhalation Powder
Oct 21, 2008 - Nektar Therapeutics (Nasdaq: NKTR) announced that Novartis AG has agreed to acquire from Nektar specific pulmonary delivery assets, technology, and intellectual property for $115 million in cash.

"This agreement will allow us to focus our efforts on the development of novel therapeutics using our PEGylation and conjugate chemistry-based drug development platforms," said Nektar President and CEO Howard W. Robin. "The transaction also strengthens our balance sheet and significantly reduces expenses. It is a perfect example of our ability to monetize assets that had little future value for Nektar."

Novartis will assume ownership of certain dry powder and liquid pulmonary formulation and manufacturing assets, including capital equipment and manufacturing facility capital lease obligations. The agreement also transfers to Novartis approximately
140 Nektar personnel, as well as certain intellectual property and manufacturing methods. Additionally, Nektar will transfer manufacturing and royalty rights to the Tobramycin inhalation powder (TIP) program, already partnered with Novartis... Nektar's Press Release -

REGENERX : RGN-457 for Cystic Fibrosis

October 01, 2008 - REGENERX BIOPHARMACEUTICALS, INC. (AMEX:RGN) announced that it is seeking a strategic partner to assist in the development of RGN-457 for the treatment of cystic fibrosis (CF). RGN-457 is based on thymosin beta 4 (TB4) peptide formulated as an inhaled therapeutic agent to address this patient population. CF is a life-threatening, hereditary disease that impairs the patient's ability to breathe due to the accumulation of thick and sticky mucus secretions in the airways of the lungs. There are estimated to be 30,000 and 40,000 CF patients in the U.S. and Europe, respectively. It is, therefore, considered an "orphan" disease in both territories. In 2006, the predicted median age of survival for patients with cystic fibrosis was 37 years... REGENERX BIOPHARMACEUTICALS' Press Release -

Gilead Complete Response Letter from U.S. Food and Drug Administration for Aztreonam Lysine for Inhalation, an Investigational Treatment for CF

Sept. 16, 2008 - Gilead Sciences, Inc. (Nasdaq:GILD) announced that the company has received a complete response letter from the U.S. Food and Drug Administration (FDA) for its New Drug Application (NDA) for aztreonam lysine for inhalation, an investigational therapy in development for people with cystic fibrosis who have Pseudomonas aeruginosa (P. aeruginosa).

In this letter, the FDA informed Gilead that the review of the company's NDA has been completed. The agency stated that they cannot approve the application in its current form and an additional clinical study will be required. Gilead will continue its dialogue with the FDA to determine whether further analyses of existing data could lead to approval, or whether the company will need to conduct the additional study as stated in the letter... Gilead Sciences' Press Release -

Arriva Pharmaceuticals : Protein Production Patent

1 October 2008 - Arriva Pharmaceuticals, a biopharmaceutical company focused on developing anti-inflammatory therapies, announced that it had been granted a patent in the U.S. for methods to produce certain proteins in yeast. Among the proteins whose production is covered by the patent (United States Patent 7,419,801) is recombinant alpha 1-antitrypsin (rAAT), the protein used in Respriva™, the company’s lead product.

“This patent extends our patent portfolio and strengthens our intellectual property estate for Respriva™,” said Sue Preston, chief executive officer of Arriva. “As we move forward with the development of Respriva™, we have multiple competitive advantages that leave us well positioned in the global market.”

Respriva™ is an inhaled rAAT. Arriva is currently conducting clinical trials with Respriva™ as replacement therapy in hereditary emphysema (HE). Patients with HE have a congenital deficiency in naturally occurring alpha 1-antitrypsin (AAT). Respriva™ also has the potential to ameliorate lung disease in other indications, such as chronic obstructive pulmonary disease (COPD), asthma and cystic fibrosis... [PDF] Arriva Pharmaceuticals' Press Release -

Pharmaxis : Second Pivotal Phase 3 Trial In Cystic Fibrosis

4 September 2008 - Pharmaceutical company Pharmaxis (ASX:PXS, NASDAQ:PXSL) announced that it has enrolled the first subject into its second pivotal Phase 3 clinical trial evaluating Bronchitol in cystic fibrosis sufferers.
The Phase 3 trial is being conducted in 41 hospitals across North America, Argentina and Germany, and is the final clinical step before Pharmaxis seeks approval to market Bronchitol for cystic fibrosis in the United States... [PDF] Pharmaxis' Press Release -