Genaera Reports Positive Preliminary Two-Month Data from Phase II Study for Squalamine... [DTZHYMG]
Genaera Reports Positive Preliminary Two-Month Data from Phase II Study for Squalamine in Age-Related Macular Degeneration
PLYMOUTH MEETING, Pa., Jan. 10 /PRNewswire-FirstCall/ -- Genaera Corporation (Nasdaq: GENR) today announced positive preliminary clinical results, including improved vision, from a multicenter open-label U.S. Phase II clinical trial (MSI-1256F-207) with squalamine for the treatment of choroidal neovascularization associated with age-related macular degeneration (AMD), also known as "wet" AMD. Squalamine is the leading systemically delivered anti-angiogenic drug being developed to treat AMD. Data from this study are scheduled to be presented by Carl Regillo, MD, FACS, Professor of Ophthalmology, Thomas Jefferson University, School of Medicine and Wills Eye Hospital, Philadelphia, at the 28th Annual Meeting of the Macula Society in Key Biscayne Florida on February 25, 2005. Preliminary results from six patients treated with 40 mg of squalamine, each of whom suffered from wet AMD in both eyes, demonstrated that of 100% of eyes (n=12) had preserved or improved vision at week three (after two doses of squalamine), week five (end of therapy) and two months after initiation of therapy. The greatest degree of improvement at two months was a gain relative to baseline of 28 letters (5.6 lines), while the greatest degree of loss was 11 letters on the ETDRS chart. In clinical trials of therapies for wet AMD, gain or loss of less than 15 letters (three lines) on the ETDRS chart constitutes stable vision, while gain equal to or greater than 15 letters constitutes improved vision. All patients received four weekly doses of squalamine, with no further maintenance therapy. There have been no withdrawals from therapy or drug-related serious adverse events in the trial thus far. Data discussed in this press release are included in the Overview Presentation, under Investor Relations at http://www.genaera.com "These preliminary results appear promising," said Professor Regillo. "I very much look forward to taking part in the next steps of squalamine clinical development through participation in the MSI-1256F-208 and -209 studies, as well as assisting Genaera in the preparation of its Phase III studies starting later this year." "These U.S. Phase II trial results are comparable to our previously reported Phase I/II clinical results in which 100% of wet AMD patients had stable or improved vision, and provide additional evidence of squalamine's tremendous potential as a less invasive systemic anti-angiogenic therapy for this blinding eye disease," said Roy C. Levitt, MD, President and Chief Executive Officer of Genaera Corporation. "The results announced today are particularly important because all of these patients had both eyes affected with wet AMD and we were able to treat both eyes with squalamine, conveniently and safely. In contrast, other anti-angiogenic agents approved or in development would have required a needle injection into both eyes in these patients. About one third of wet AMD patients have both eyes affected at the same time." Dr. Levitt added, "This is one of three Phase II studies enrolling in the U.S. to evaluate squalamine in wet AMD. While these are preliminary results in a small number of patients, thus far our data suggest a dose response indicating that a 40 mg dose may provide clinical benefit beyond that provided by the lowest dose tested, 10 mg. Studies are ongoing to further evaluate whether there is a role for the10 mg dose. We look forward to presenting these data in greater detail at upcoming medical meetings and continuing to assess the potential for squalamine as a treatment for this debilitating condition." Clinical Trial Program Genaera is currently conducting three Phase II trials of squalamine in AMD at multiple sites throughout the United States. Recently, the FDA selected squalamine for participation in the Continuous Marketing Application (CMA) Pilot 2 program. In October 2004, the FDA granted squalamine Fast Track designation. MSI-1256F-207 is a Phase II pharmacokinetic and safety trial that will evaluate 18 patients with AMD at three different doses of squalamine (10 mg, 20 mg or 40 mg) over four months. In this multi-center, open-label, parallel group study, squalamine is administered intravenously at three doses, once weekly for four weeks. Depending on their response, patients may continue to receive squalamine for up to a year in a separate study (MSI-1256F-211). MSI-1256F-208 is a Phase II trial designed to evaluate the effects of three different doses of squalamine in combination with an initial Visudyne(R) treatment in 45 patients with AMD. Specifically, this study will evaluate the safety and effects of systemically administered squalamine before and after photodynamic therapy with Visudyne(R). Based on its mechanism of action, this squalamine pretreatment has the potential to improve the effect of Visudyne(R), and squalamine follow up treatment may inhibit the detrimental effects of the VEGF 'burst' that commonly occurs after Visudyne(R) treatment. The multi-center, randomized, controlled, masked study also includes monthly squalamine maintenance therapy through six months, along with an additional twelve months follow-up for each patient. MSI-1256F-209 is the cornerstone and largest of Genaera's three Phase II studies and is designed to evaluate the safety and efficacy of squalamine in 100 patients with AMD over a two-year period. This Phase II multi-center, randomized, double masked, controlled study will evaluate two dose levels of squalamine (20 mg or 40 mg) given once weekly for four weeks, followed by maintenance doses once every four weeks until week 48. At the end of 48 weeks of therapy, each patient will be followed for a further 12 months. Genaera anticipates using data analyses from this study in coordinating Phase III activities. For information about participation in squalamine clinical trials, patients and physicians may call Genaera's Clinical Trial Hotline at (800) 299-9156. About Squalamine Squalamine is a unique first in class synthetic small molecule administered systemically that directly interrupts and reverses multiple facets of the angiogenic process. Working within activated endothelial cells, squalamine inhibits growth factor signaling including VEGF, integrin expression, and reverses cytoskeletal formation, thereby resulting in endothelial cell inactivation and apoptosis. Systemically administered squalamine inhibits abnormal angiogenesis in rodent models of retinopathy of prematurity, and the development of choroidal neovascular membranes in rat models of AMD. Additional preclinical studies have demonstrated that systemic squalamine administration is effective in reaching abnormal ocular blood vessels in primates, and leads to partial regression and inhibition of new abnormal vessels in the eye. These results support that squalamine may have a role in the treatment of human choroidal neovascular membrane formation that underlies the pathology of wet AMD. About AMD Wet AMD resulting from angiogenesis is the leading cause of legal blindness among adults age 50 or older in the Western world. Approximately 25 to 30 million people are affected globally and this number is expected to triple over the next 25 years. AMD occurs in two types: the "dry" form and the more severe "wet" form. Wet AMD is caused by the growth of abnormal blood vessels, or choroidal neovascularization, under the central part of the retina, the macula. Dry AMD, or the avascular form is the more common and milder form of AMD, accounting for 85% to 90% of all cases. Dry AMD results in varying forms of sight loss and may or may not eventually develop into the wet form. Although the wet form of AMD accounts for only 10% to 15% of all AMD, the chance for severe sight loss is much greater. It is responsible for 90% of severe vision loss associated with AMD. Approximately 500,000 new cases of wet AMD are diagnosed annually worldwide. In North America alone, approximately 200,000 new cases of wet AMD are diagnosed each year. About Genaera Genaera Corporation is a biopharmaceutical company committed to developing medicines for serious diseases from genomics and natural products. Research and development efforts are focused on anti-angiogenesis and respiratory diseases. Genaera has three products in development addressing substantial unmet medical needs in major pharmaceutical markets. These include squalamine, an anti-angiogenesis treatment for cancer and eye disease; interleukin-9 antibody, a respiratory treatment based on the discovery of a genetic cause of asthma; and LOMUCIN(TM), a mucoregulator to treat the overproduction of mucus and secretions involved in many forms of chronic respiratory disease. For more information on Genaera, visit the company's website at http://www.genaera.com . This announcement contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks and uncertainties, known and unknown. Forward-looking statements reflect management's current views and are based on certain expectations and assumptions. You may identify some of these forward-looking statements by the use of words in the statements such as "anticipate," "believe", "continue," "develop," "expect," "plan" and "potential" or other words of similar meaning. Genaera's actual results and performance could differ materially from those currently anticipated and expressed in these and other forward-looking statements as a result of a number of risk factors, including, but not limited to: the risk that subsequent clinical trial results differ from the results announced today; the risk that squalamine ceases to meet the criteria for CMA2 Pilot or Fast Track designation at some point in the future; Genaera's history of operating losses since inception and its need for additional funds to operate its business; the costs, delays and uncertainties inherent in scientific research, drug development, clinical trials and the regulatory approval process; the risk that clinical trials for Genaera's product candidates, including squalamine may be delayed or may not be successful; the risk that Genaera may not obtain regulatory approval for its products, whether due to adequacy of the development program, the conduct of the clinical trials, changing regulatory requirements, different methods of evaluating and interpreting data, regulatory interpretations of clinical risk and benefit, or otherwise; Genaera's reliance on its collaborators, in connection with the development and commercialization of Genaera's product candidates; market acceptance of Genaera's products, if regulatory approval is achieved; competition; general financial, economic, regulatory and political conditions affecting the biotechnology and pharmaceutical industry; and the other risks and uncertainties discussed in this announcement and in Genaera's filings with the U.S. Securities and Exchange Commission, all of which are available from the Commission in its EDGAR database at www.sec.gov as well as other sources. You are encouraged to read these reports. Given the uncertainties affecting development stage pharmaceutical companies, you are cautioned not to place undue reliance on any such forward-looking statements, any of which may turn out to be wrong due to inaccurate assumptions, unknown risks, uncertainties or other factors. Genaera does not intend (and it is not obligated) to publicly update, revise or correct these forward-looking statements or the risk factors that may relate thereto. SOURCE Genaera Corporation 10Jan05 13:30 GMT Symbols: us;GENR Source PRN PR Newswire Categories: MST/I/BTC MST/I/DRG MST/I/HEA MST/L/EN MST/R/US/PA TGT/PRX
[The biggest loser in this deal could be DYAX, who is developing a competing drug for HAE in the U.S. together with GENZ. However, DYAX’s drug has yet to begin phase-3 trials. Meanwhile, Pharming, n.v., is wrapping up its own phase-3 program for HAE in Europe.
Please see #msg-4691681 for some hideous photos of patients with HAE.]
>> Kos and Jerini Sign Exclusive Collaboration and License Agreement for Development, Marketing and Distribution of Icatibant in The United States and Canada
Monday November 7, 3:03 am ET
-- Kos to develop, market and distribute Icatibant in the U.S. and Canada
-- Icatibant, in final Phase III clinical trials for hereditary angioedema (HAE), represents next potential new product launch for both companies scheduled for the first half of 2007
-- U.S. Food and Drug Administration has granted Icatibant fast track designation and orphan drug status
-- Jerini plans further clinical development for Icatibant for additional indications of angioedema
-- Kos responsible for Icatibant's clinical development in asthma(inhalation) and refractory ascites in liver cirrhosis (intravenous)
CRANBURY, N.J. and BERLIN, Nov. 7, 2005 (PRIMEZONE) -- Kos Life Sciences, Inc., a subsidiary of Kos Pharmaceuticals, Inc. (NasdaqNM: KOSP ) and Jerini U.S. Inc., a subsidiary of Jerini AG (FSE:JI4), announced today the signing of an exclusive agreement for the development, marketing and distribution of Jerini's compound, Icatibant, a potent and specific peptidomimetic bradykinin B2 receptor antagonist, in the United States and Canada. The strategic partnership includes an upfront licensing payment of EUR 12 million (approx. $15 million), along with a EUR 10 million (approx. $12 million) equity investment out of a capital increase at the higher of the weighted average of the closing price of Jerini's shares during the last four trading days, or EUR 3.20 (approx. $3.84) per share (IPO price). Undisclosed milestone payments and sales royalties are also specified in the agreement. Hereditary angioedema (HAE) is the first of several diseases for which Icatibant offers a potential treatment. The agreement outlines collaboration in the HAE area as well as other forms of angioedema, asthma, and refractory ascites in liver cirrhosis (RAIL) indications. Proof-of-concept trials have already been completed in these indications.
HAE is a rare genetic disease which can be debilitating, painful and life-threatening and is characterized by recurrent local swelling at three main sites: subcutaneous tissue, the gastrointestinal tract, and the larynx. Laryngeal attacks can lead to upper airway constriction and to suffocation. HAE is caused by an activation of the kinin system leading to elevated levels of bradykinin (a naturally occurring peptide hormone). These elevated levels of bradykinin present in the affected areas that cause the symptomatic swelling attacks. Icatibant reduces the effects of such elevated levels of bradykinin, and if approved could offer HAE patients a novel treatment that will allow them the opportunity to self-administer the drug in the event of an attack. There are approximately 10,000 diagnosed HAE patients in the European Union and the U.S., but the disease is believed to be significantly under diagnosed. Experts estimate the HAE patient population could be as high as 75,000. In addition, Icatibant also offers a potential treatment for drug-induced and idiopathic angioedema, affecting a significantly larger patient group. RAIL is another potential indication for this product affecting approximately 300,000 patients.
``We are very pleased with Jerni's clinical development of Icatibant for the angioedema indication and are excited about this near-term commercial opportunity. Currently there are no approved treatment options for HAE in the U.S. or most other countries. We are hopeful that Icatibant may be able to fulfil a serious unmet medical need in this patient population,' commented Adrian Adams, President and Chief Executive Officer of Kos Pharmaceuticals, Inc. ``The product has received orphan drug and fast track review status by the FDA, and could potentially be launched in the first half of 2007. This strategic acquisition could provide entry into new specialty markets that are synergistic with our therapeutic focus in addition to leveraging Kos' established field force strengths and customertargets,' he continued. ``Our partnership with Jerini is an excellent example of Kos' expanded business model that includes making measured investments to build our R&D pipeline through scientific in-licensing and general corporate development. Kos is committed to exploring the potential of Icatibant in other bradykinin related diseases such as RAIL and asthma, indications that may provide additional medium-term opportunities for organic sales growth.'
``Kos is the ideal partner for the U.S. and Canadian markets, and this agreement meets all of our goals. Our strong financial position of approximately $120 million after our recent IPO enabled us to structure a transaction that includes significant royalty, sales and regulatory milestone payments, which allows us to participate substantially in the potential long-term success of Icatibant. Kos is an outstanding specialty pharmaceutical company and its exceptional growth and performance distinguish it as a market leader.
Kos has a strong U.S. infrastructure with a specialized sales force covering the complete range of medical areas needed to realize the full potential of Icatibant in the planned 2007 U.S. and Canadian launch,`` said Jens Schneider-Mergener, Chief Executive Officer of Jerini AG. 'In addition, our agreement with Kos expands the therapeutic potential of Icatibant to even larger indications that may bring considerable upside for us in the future.``
Jerini will retain the commercial rights to Icatibant outside the U.S. and Canada and continues to be responsible for Icatibant's HAE clinical Phase III trials and regulatory approval. Phase III clinical trials are currently being conducted in the U.S., Canada, Europe, and several other countries. Results from these trials are expected to be reported in mid- 2006, with filing for market approval in the U.S. and Europe also planned in 2006. Kos and Jerini will collaborate on the global branding and positioning of Icatibant in HAE and other forms of angioedema. Jerini will also continue to be responsible for the supply of Icatibant.
Icatibant has been shown to be safe and well tolerated in over 1,000 individuals. Phase II clinical results in HAE demonstrated a 100% response rate to the drug. Currently, Icatibant is in Phase III clinical trials, supported by a Special Protocol Assessment (SPA) from the FDA. These trials include the treatment of life-threatening laryngeal attacks as part of an open-label extension study. After treatment with Icatibant, all these patients experienced rapid onset of symptom relief, and no further treatment was necessary. Jerini plans additional clinical testing for idiopathic and drug-induced angioedema with Icatibant in 2006.
About Icatibant
Icatibant is a potent and specific peptidomimetic bradykinin B2 receptor antagonist. It has exhibited a strong safety profile in over 1,000 individuals. Icatibant is being tested in clinical trials for treatment of hereditary angioedema and refractory ascites in liver cirrhosis as well as in preclinical models for severe burn injuries.
About Icatibant and refractory ascites in liver cirrhosis (RAIL)
RAIL is an extremely painful and debilitating disease caused by the massive accumulation of excess fluid in the intraperitoneal cavity, which often develops during liver decompensation in chronic liver cirrhosis. Most patients in the decompensated stage die within three years. The most prevalent causes of liver cirrhosis are alcohol abuse, viral hepatitis (B, C), and autoimmune chronic active hepatitis
About Kos Pharmaceuticals, Inc.
Kos Pharmaceuticals, Inc. is a fully integrated specialty pharmaceutical company engaged in developing, commercializing, manufacturing and marketing proprietary prescription products for the treatment of chronic diseases with a particular focus on the cardiovascular, metabolic and respiratory disease areas. The Company's principal product development strategy is to reformulate existing pharmaceutical products with large market potential to improve safety, efficacy, and patient compliance. Kos' strategy also includes making increased, measured investments in new chemical entity research through in-house and sponsored research, scientific in-licensing and general corporate development activities. The Company currently markets Niaspano and Advicoro for the treatment of cholesterol disorders, Azmacorto for the treatment of asthma, CardizemoLA for the treatment of hypertension and angina, and Teveteno and TevetenoHCT for the treatment of hypertension. Kos has a strong and growing research and development pipeline including proprietary drug delivery technologies in solid-dose, inhalation and aerosol metered-dose device administration to help fuel sustained, organic sales growth into the future.
About Jerini AG
Jerini AG is a pharmaceutical company based in Berlin, Germany focusing on the discovery and development of peptide-derived peptidomimetic and small molecule therapeutics. Having recognized the potential of peptides as natural starting molecules for drug discovery, the company has developed state-of-the-art technologies to identify and transform peptides into drugs. The company's lead product, Icatibant, is a peptidomimetic and is currently in Phase III clinical trials for the treatment of hereditary angioedema. <<
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