John-Knee
5 años hace
Alexion to Acquire Achillion
2019-10-16 06:30:00 AM ET (BusinessWire)
--- Provides opportunity to enhance treatment for PNH patients experiencing extravascular hemolysis (EVH), potential first-in-class C3 glomerulopathy (C3G) therapy & promising development platform for Factor D inhibition in additional alternative pathway complement-mediated rare diseases -
--- Initial all-cash transaction for $6.30 per share; total transaction of up to $8.30 per share with potential additional contingent considerations -
--- Conference call and webcast scheduled for today, October 16, 2019, at 8:00 a.m. EDT -
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) and Achillion Pharmaceuticals, Inc. (NASDAQ:ACHN) today announced that they have entered into a definitive agreement for Alexion to acquire Achillion, a clinical-stage biopharmaceutical company focused on the development of oral small molecule Factor D inhibitors to treat people with complement alternative pathway-mediated rare diseases, such as paroxysmal nocturnal hemoglobinuria (PNH) and C3 glomerulopathy (C3G). Achillion currently has two clinical-stage medicines in development, including danicopan (ACH-4471) in Phase 2 and ACH-5228 in Phase 1.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20191016005257/en/
"Alexion has demonstrated the transformative impact that inhibiting C5 can have on multiple rare and devastating diseases. However, we believe this is just the beginning of what's possible with complement inhibition," said Ludwig Hantson, Ph.D., Chief Executive Officer of Alexion. "Targeting a different part of the complement system - the alternative pathway - by inhibiting Factor D production addresses uncontrolled complement activation further upstream in the complement cascade, and importantly, leaves the rest of the complement system intact, which is critical in maintaining the body's ability to fight infection. We believe this approach has the opportunity to help patients with diseases not currently addressed through C5 inhibition. We look forward to applying our nearly three decades of complement and development expertise to unlock the potential of oral Factor D inhibitors and bring these benefits to patients."
"We have established great momentum - discovering and advancing several small molecules into clinical development that have the potential to treat immune-related diseases associated with the alternative pathway of the complement system," said Joe Truitt, President and Chief Executive Officer at Achillion. "Having already demonstrated proof-of-concept and proof-of-mechanism with our lead candidate, danicopan (ACH-4471), in PNH and C3G, respectively, we believe there is significant opportunity for Factor D inhibition in the treatment of other diseases as well. Alexion is an established leader in developing medicines for complement-mediated diseases, and we look forward to working together to accelerate our objective of bringing novel therapies to patients as quickly as possible and ensuring that the broad promise of this approach is fully realized. We thank our employees, investigators and partners for their incredible work and commitment."
Transaction Details The initial consideration of approximately $930 million, or $6.30 per share of Achillion common stock, will be funded with cash on hand. As part of the acquisition, Alexion will also be acquiring the cash currently on Achillion's balance sheet. As of September 30, 2019, this was approximately $230 million; the actual amount will be determined as of the transaction close. The transaction includes the potential for additional consideration in the form of non-tradeable contingent value rights (CVRs), which will be paid to Achillion shareholders if certain clinical and regulatory milestones are achieved within specified periods. These include $1.00 per share for the U.S. FDA approval of danicopan and $1.00 per share for ACH-5228 Phase 3 initiation.
Alexion's acquisition of Achillion is subject to the approval of Achillion shareholders and satisfaction of customary closing conditions and approval from relevant regulatory agencies, including clearance under the Hart-Scott Rodino Antitrust Improvements Act. Pending these approvals, the transaction is expected to close in the first half of 2020.
Conference Call Alexion will host a conference call and webcast today, October 16, 2019, at 8:00 a.m. EDT to discuss the acquisition. To participate in this call, dial (866) 762-3111 (USA) or (210) 874-7712 (International), passcode 5426458, shortly before 8:00 a.m. EDT. A replay of the call will be available for a limited period of time following the call. The audio webcast can be accessed on the Investors page of Alexion's website at: http://ir.alexion.com.
About Factor D Factor D is an essential serine protease and critical control point in the alternative pathway (AP) of the complement system, a part of the innate immune system. Achillion's complement platform is focused on advancing oral small molecules that inhibit the AP and can potentially be used in the treatment of immune-related diseases in which complement AP plays a critical role. Potential indications currently being evaluated for these compounds include PNH, C3G and immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN).
About Paroxysmal Nocturnal Hemoglobinuria (PNH) PNH is a chronic, progressive, debilitating and life-threatening ultra-rare blood disorder characterized by hemolysis (destruction of red blood cells) that is mediated by uncontrolled activation of the complement system, a component of the body's immune system. Patients with PNH may experience a wide range of signs and symptoms, such as fatigue, difficulty swallowing, shortness of breath, abdominal pain, erectile dysfunction, dark-colored urine and anemia. The most devastating consequence of chronic hemolysis is thrombosis, which can occur in blood vessels throughout the body, damaging vital organs and causing premature death. PNH is primarily a disease of intravascular hemolysis (IVH), where the red blood cell destruction occurs within the blood vessels. C5 inhibition addresses the complications of IVH and the increases in LDH that cause thrombosis and even death in patients with PNH. However, a small portion of patients - less than 10 percent - receiving a C5 inhibitor continue to experience clinical extravascular hemolysis (EVH), where the red blood cell destruction occurs outside the blood vessels. As a result, these patients are transfusion dependent despite treatment but do not have bone marrow failure or aplastic anemia. Inhibiting Factor D in the alternative pathway (AP) of the complement system offers the possibility of selectively blocking AP activity and protecting against the destruction of RBCs, while leaving the rest of the complement system intact to fight infection.
About C3 Glomerulopathy (C3G) C3G is an ultra-rare kidney disease for which there is no approved treatment. The disease is characterized by the deposition of C3 protein fragments in the filtering units (glomeruli) of the kidney, caused by overactivation of the complement alternative pathway (AP). Over time, the chronic deposition of C3 fragments results in permanent kidney damage and kidney failure. Today, C3G patients are treated with steroids and broad-acting immunosuppressants to slow the progression of kidney damage. Oral Factor D inhibitors have demonstrated proof-of-mechanism to interrupt the overactivation of the AP and reduce C3 fragment deposition, providing a potential treatment approach for targeting the underlying cause of C3G.
About Alexion Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases through the discovery, development and commercialization of life-changing therapies. As the global leader in complement biology and inhibition for more than 20 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) as well as the first and only approved complement inhibitor to treat atypical hemolytic uremic syndrome (aHUS), anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D). In addition, the company is developing several mid-to-late-stage therapies, including a second complement inhibitor, a copper-binding agent for Wilson disease and an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases as well as several early-stage therapies, including one for light chain (AL) amyloidosis and a second anti-FcRn therapy. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of hematology, nephrology, neurology, and metabolic disorders. Alexion has been named to the Forbes' list of the World's Most Innovative Companies seven years in a row and is headquartered in Boston, Massachusetts' Innovation District. The company also has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: www.alexion.com.
[ALXN-G]
About Achillion Pharmaceuticals Achillion is a clinical-stage biopharmaceutical company focused on advancing its oral small molecule complement inhibitors into late-stage development and commercialization. Research has shown that an overactive complement system plays a critical role in multiple disease conditions including the therapeutic areas of nephrology, hematology, ophthalmology and neurology. Achillion is initially focusing its drug development activities on complement-mediated diseases where there are no approved therapies or where existing therapies are inadequate for patients. Potential indications being evaluated for its compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G), and immune complex membranoproliferative glomerulonephritis (IC-MPGN). The company has received Breakthrough Therapy designation for danicopan for treatment in combination with a C5 monoclonal antibody for patients with paroxysmal nocturnal hemoglobinuria (PNH) who are sub-optimal responders to a C5 inh ibitor alone. Each of the product candidates in the company's oral small molecule portfolio was discovered in its laboratories and is wholly owned. To advance its investigational product candidates into Phase 3 clinical trials and commercialization, the company plans to work closely with key stakeholders including healthcare professionals, patients, regulators and payors. More information is available at http://www.achillion.com.
John-Knee
5 años hace
News: Achillion Receives Breakthrough Therapy Designation from FDA for Danicopan for Treatment of Paroxysmal Nocturnal Hemoglobinuria
2019-09-25 07:00:02 AM ET (GlobeNewswire)
- Danicopan Phase 2 PNH combination therapy topline data expected Q4 2019 -
- Initiation of Phase 3 planned for early 2020 -
Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN), a clinical-stage biopharmaceutical company dedicated to transforming the lives of patients and families affected by complement-mediated diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for danicopan (ACH-4471) for treatment in combination with a C5 monoclonal antibody for patients with paroxysmal nocturnal hemoglobinuria (PNH) who are sub-optimal responders to a C5 inhibitor alone. The FDA's decision was based on positive safety and efficacy data from the ongoing danicopan Phase 2 PNH combination trial. Interim data was reported at the New Era of Aplastic Anemia and PNH Meeting in May 2019. The top line data from this combination trial is expected in the fourth quarter of 2019.
"The FDA's granting of Breakthrough Therapy designation for our lead oral factor D inhibitor, danicopan, underscores the urgent need for new treatment options for patients living with PNH," said Joe Truitt, President and Chief Executive Officer at Achillion. "Danicopan, with its demonstrated ability to limit both intravascular and extravascular hemolysis with oral administration, has the potential to benefit a significant number of patients with PNH that continue to have an unmet medical need on standard of care. We appreciate the review and decision by the FDA and plan to work closely with the Agency in advancing the development of danicopan into Phase 3 in early 2020."
FDA Breakthrough Therapy designation is designed to expedite the development and review of medicines for serious or life-threatening conditions. Receiving Breakthrough Therapy designation from the FDA indicates preliminary clinical evidence has demonstrated the drug may provide substantial improvement on at least one clinically significant endpoint compared with currently available therapy. The benefits of this Breakthrough Therapy designation include more intensive guidance from FDA on an efficient drug development program, access to a scientific liaison to help accelerate review time and eligibility for Accelerated Approval and Priority Review if relevant criteria are met. Danicopan (ACH-4471) has previously received orphan drug designation for the treatment of PNH in 2017.
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH is a rare, acquired blood disease caused by a somatic mutation resulting in the absence of key receptors, CD55 and CD59, on the surface of red blood cells (RBCs). The alternative pathway (AP) of the complement system recognizes these unprotected RBCs as foreign and destroys them in the circulatory system (intravascular hemolysis) and in the liver or spleen (extravascular hemolysis). The current standard of care for PNH targets intravascular hemolysis by inhibiting C5 complement protein (C5), leaving some patients with persistent extravascular hemolysis from early phases of complement activation (AP Activity) which C5 inhibition cannot address leaving patients with partial control of their PNH. Up to seventy-five percent of PNH patients treated with C5 inhibitors remain anemic during treatment, with up to one-third of those patients reporting the need for blood transfusions within the last year. Factor D is the critical, rate-limiting protein within the AP. By targeting Fac tor D, proximal AP inhibition may disable both downstream terminal complement activation (IVH) and upstream C3 fragment opsonization (EVH). Achillion is developing a potentially more complete approach to PNH with factor D inhibition to selectively block alternative pathway activity and protect against both destructive processes of RBCs in PNH with convenient oral therapies.
More information is available at http://www.achillion.com/patients-and-clinicians/.
About the Achillion Complement Factor D Portfolio
Achillion has leveraged its internal discovery capabilities and a novel complement-related platform to develop oral small molecule drug candidates that are inhibitors of complement factor D. Factor D is an essential serine protease involved in the alternative pathway (AP) of the complement system, a part of the innate immune system. Achillion's complement platform is focused on seeking to advance oral small molecules that inhibit the AP and can potentially be used in the treatment of immune-related diseases in which complement AP plays a critical role. Potential indications currently being evaluated for these compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G), and immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN).
About Achillion Pharmaceuticals
Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN) is a clinical-stage biopharmaceutical company focused on advancing its oral small molecule complement inhibitors into late-stage development and commercialization. Research has shown that an overactive complement system plays a critical role in multiple disease conditions including the therapeutic areas of nephrology, hematology, ophthalmology and neurology. Achillion is initially focusing its drug development activities on complement-mediated diseases where there are no approved therapies or where existing therapies are inadequate for patients. Potential indications being evaluated for its compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G), and immune complex membranoproliferative glomerulonephritis (IC-MPGN). Each of the product candidates in the Company's oral small molecule portfolio was discovered in its laboratories and is wholly owned. To achieve its goal of advancing its investigational pr oduct candidates into Phase 3 clinical trials and commercialization, the Company plans to work closely with key stakeholders including healthcare professionals, patients, regulators and payors.
crudeoil24
6 años hace
Achillion Reports Positive Interim Data for ACH-4471 Phase 2 Combination Trial with Eculizumab at The New Era of Aplastic Anemia and PNH Meeting
7:01 am ET May 17, 2019 (Globe Newswire) Print
- ACH-4471 increased hemoglobin and nearly eliminated the need for transfusions in patients with PNH being treated with C5 inhibitor, eculizumab -
Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN), a clinical-stage biopharmaceutical company dedicated to transforming the lives of patients and families affected by complement-mediated diseases, today reported interim data from a Phase 2 paroxysmal nocturnal hemoglobinuria (PNH) trial assessing the safety and effectiveness of its oral small molecule factor D inhibitor ACH-4471 in combination with intravenous eculizumab at The New Era of Aplastic Anemia and PNH Meeting in Naples, Italy.
"Anemia is a persistent problem in the majority of patients with PNH treated with standard and even high doses of eculizumab. These interim data are encouraging and demonstrate that ACH-4471, when used in combination with a C5 inhibitor, such as eculizumab, has the potential to improve anemia, decrease transfusions and lead to improvement in important clinical parameters of hemolysis as well as quality of life measurements for patients with this devastating condition," stated Joseph Truitt, Chief Executive Officer at Achillion.
ACH-4471 Interim Data
This proof-of-concept, 24-week trial is ongoing. Interim data in 11 enrolled patients were assessed between 4 to 24 weeks, depending on the patient's current treatment duration in the dose escalating trial. The oral presentation features the following data:
-- Increases in mean hemoglobin of approximately 2 g/dL at week 4 (n=11); for the 4 patients that have reached 24 weeks their mean rise in hemoglobin is 2.6 g/dL;
-- A reduction in blood transfusions from 34 transfusions totaling 58 units in the 24 weeks prior to screening to only 1 transfusion of 2 units during treatment with ACH-4471;
-- Meaningful improvement in Functional Assessment of Chronic Illness Therapy (FACIT) fatigue scores versus baseline, with a mean score increase of 11 at week 4;
-- Increase in the percentage of PNH RBC Type III clone size from 40% at baseline to 71% at week 12 (n=8);
-- Reduction in total bilirubin from a mean of 2.17 mg/dL to 1.21 mg/dL at week 16 (n=8);
-- Reduction in mean reticulocytes from 219 10^9/uL at baseline to 153 10^9/uL at week 16 (n=8);
-- Further reduction of LDH into the normal range;
-- Four patients are currently receiving the lowest study dose of 100 mg three times a day;
-- ACH-4471 was generally well tolerated when added to eculizumab in patients with PNH.
"In patients treated with C5 inhibitors alone, extravascular hemolysis (EVH) is an ongoing problem resulting in anemia with an impact on most of the clinical parameters of hemolysis. These interim findings from this important proof-of-concept trial show that if the alternative pathway is adequately inhibited, important parameters of EVH can be improved, including and most importantly hemoglobin, transfusion, bilirubin, reticulocyte count, and FACIT fatigue scores, when added to stable optimal dose eculizumab therapy. We look forward to completing the Phase 2 trial this summer and future discussions with the regulatory authorities. After completion of regulatory discussions, we hope to initiate a Phase 3 PNH combination trial of ACH-4471 with C5 inhibitors in the first half of 2020," stated Steven Zelenkofske, D.O. Chief Medical Officer at Achillion.
Slides from the oral presentation will be available on the Company's website, today, May 17, at approximately 9:00 a.m. EST at http://ir.achillion.com/events-and-presentations.
ACH-4471 Phase 2 Trial in Combination with Eculizumab
ACH-4471 is being evaluated in combination with eculizumab, an intravenous C5 inhibitor that is currently approved as monotherapy for PNH. This is a Phase 2, open-label, multiple dose trial in adult patients on stable eculizumab treatment with blood transfusion dependent anemia, defined as receiving at least one transfusion in the 12 weeks prior to the study and a hemoglobin level below 10 g/dL. In addition to their usual dose of eculizumab, patients are administered ACH-4471 orally three times a day at a dose determined by patient clinical response. The primary outcome of the trial is the change in hemoglobin at 24 weeks compared to baseline. Secondary outcomes include the number of blood transfusions required, impact on selected clinical parameters, and safety. The trial will be followed by a long-term extension phase.
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH is thought to be caused by a mutation resulting in the absence of receptors normally present on red blood cells (RBCs) that interact with the complement system. The complement system typically functions normally in these patients but due to the lack of key receptors, known as CD55 and CD59, on the surface of PNH RBCs, the complement system treats these cells as foreign and destroys them via hemolysis in the circulatory system (intravascular) and in the liver or spleen (extravascular). The complement alternative pathway (AP) is a critical factor in the development of extravascular hemolysis. Complement factor D is a critical protein within the amplification loop of the AP and it is believed that inhibiting it could control the AP response. Furthermore, this mechanism of action represents a potentially distinct and unique therapeutic approach for controlling intravascular and extravascular hemolysis associated with PNH.
More information is available at http://www.achillion.com/patients-and-clinicians/.
About Achillion Pharmaceuticals
Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN) is a clinical-stage biopharmaceutical company focused on advancing its oral small molecule complement inhibitors into late-stage development and commercialization. Research has shown that an overactive complement system plays a critical role in multiple disease conditions including the therapeutic areas of nephrology, hematology, ophthalmology and neurology. Achillion is initially focusing its drug development activities on complement-mediated diseases where there are no approved therapies or where existing therapies are inadequate for patients. Potential indications being evaluated for its compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G), and immune complex membranoproliferative glomerulonephritis (IC-MPGN). Each of the product candidates in the Company's oral small molecule portfolio was discovered in its laboratories and is wholly owned. To advance its investigational product candidates into Phase 3 clinical trials and commercialization, the Company plans to work closely with key stakeholders including healthcare professionals, patients, regulators and payors.
More information is available at http://www.achillion.com.
Cautionary Note Regarding Forward-Looking Statements
This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other important factors that could cause actual results to differ materially from those indicated by such forward-looking statements. Achillion may use words such as "expect," "anticipate," "project," "target," "intend," "plan," "aim," "believe," "seek," "estimate," "can," "could" "focus," "will," "look forward," "continue," "goal," "strategy," "objective," "may," "potential," and similar expressions to identify such forward-looking statements. These forward-looking statements also include statements about: the potential benefits of factor D inhibition as a treatment for complement-mediated diseases, including ACH-4471 for PNH in combination with eculizumab; the potential benefits of, and indications for, Achillion's compounds that inhibit factor D, including ACH-4471, ACH-5228 and ACH-5548; Achillion's belief that its portfolio of compounds could expand factor D portfolio opportunities, provide strategic optionality or create significant value; the status of enrollment in Achillion's ongoing clinical trials; Achillion's expectations regarding the advancement of, and timeline for reporting results from, clinical trials of its product candidates as well as its ability to advance additional compounds; Achillion's expectations regarding the timing of regulatory interactions and filings; Achillion's anticipated cash expenditures for 2019 and the sufficiency of its existing cash resources; and other statements concerning Achillion's strategic goals, efforts, plans, and prospects. Among the important factors that could cause actual results to differ materially from those indicated by such forward-looking statements are risks relating to, among other things, Achillion's ability to: demonstrate in any current and future clinical trials the requisite safety, efficacy and combinability of its product candidates; advance the preclinical and clinical development of its complement factor D inhibitors under the timelines it projects in current and future preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials or preclinical studies will be indicative of the results of later clinical trials; enroll patients in its clinical trials on its projected timelines; obtain and maintain patent protection for its product candidates and the freedom to operate under third party intellectual property; obtain and maintain necessary regulatory approvals, and the granting of orphan designation does not alter the standard regulatory requirements and process for obtaining such approval; establish commercial manufacturing arrangements; identify, enter into and maintain collaboration and other commercial agreements with third-parties; compete successfully in the markets in which it seeks to develop and commercialize its product candidates and future products; manage expenses; manage litigation; raise the substantial additional capital needed to achieve its business objectives; and successfully execute on its business strategies. These and other risks are described in the reports filed by Achillion with the U.S. Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2019, and any other SEC filings that Achillion makes from time to time.
In addition, any forward-looking statement in this press release represents Achillion's views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Achillion disclaims any duty to update any forward-looking statement, except as required by applicable law.
Investors:
Brian Di Donato
Senior VP, Chief Financial Officer
Tel. 215-709-3032
bdidonato@achillion.com
Media:
Susanne Heinzinger
Senior VP, Corporate Communications
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