Summary

• Page properties (1 modified, 0 added, 0 removed)

Details

Page properties

Content

...	...	@@ -2,12 +2,10 @@
2	2	{{toc/}}
3	3	{{/box}}
4	4
5		-= Paragraph 1 =
	5	+= Overview =
6	6
7		-==== Overview ====
	7	+Global Blood Therapeutics (GBT) is a clinical-stage biopharmaceutical company determined to discover, develop and deliver innovative treatments that provide hope to underserved patient communities. The company's lead product candidate is voxelotor (previously known as GBT440), an oral, once-daily therapy that modulates hemoglobin’s affinity for oxygen, which the company believe inhibits hemoglobin polymerization in sickle cell disease, or SCD.{{footnote}}https://fintel.io/doc/sec-gbt-global-blood-therapeutics-10k-2018-february-27-17968{{/footnote}}
8	8
9		-Global Blood Therapeutics is a clinical-stage biopharmaceutical company determined to discover, develop and deliver innovative treatments that provide hope to underserved patient communities. The company's lead product candidate is voxelotor (previously known as GBT440), an oral, once-daily therapy that modulates hemoglobin’s affinity for oxygen, which the company believe inhibits hemoglobin polymerization in sickle cell disease, or SCD.
10		-
11	11	Global Blood Therapeutics is currently evaluating voxelotor in SCD in a Phase 3 clinical trial in adult and adolescent patients with SCD. In addition, Global Blood Therapeutics is evaluating the safety and pharmacokinetics of single and multiple doses of voxelotor in a Phase 2a clinical trial of adolescent and pediatric patients with SCD, and in July 2017 the company announced that Global Blood Therapeutics has expanded this open-label trial to include a new single-dose cohort in children aged 6-11. In December 2015, the Food and Drug Administration, or FDA, granted Fast Track Designation and Orphan Drug Designation for voxelotor for the treatment of SCD and in November 2016 voxelotor was granted Orphan Drug Designation in Europe for the treatment of SCD. In June 2017, the European Medicines Agency, or EMA, granted PRIME designation for voxelotor for the treatment of SCD. The PRIME program is a new regulatory mechanism that provides for early and proactive EMA support to medicine developers to help patients benefit as early as possible from innovative new products that have demonstrated the potential to significantly address an unmet medical need. In September 2017, the FDA granted Rare Pediatric Disease designation to voxelotor for the treatment of SCD. In January 2018, the FDA granted Breakthrough Therapy Designation to voxelotor for the treatment of SCD.
12	12
13	13	SCD is marked by red blood cell, or RBC, destruction and occluded blood flow and hypoxia, leading to anemia, stroke, multi-organ failure, severe pain crises, and shortened patient life span. Voxelotor inhibits abnormal hemoglobin polymerization, the underlying mechanism that causes sickling of RBCs. In its clinical trials to date of voxelotor in SCD patients, the company observed reduced markers of red blood cell destruction, improvements in anemia, improvements in markers of tissue oxygenation, and reduced numbers of sickled RBCs.
...	...	@@ -42,7 +42,7 @@
42	42
43	43	To execute on the opportunities presented by its research and development portfolio, Global Blood Therapeutics has assembled a team of employees, management and directors rich in scientific experience and capabilities in drug discovery, development and commercialization. The company's management has a successful track record in developing and commercializing drug candidates. In aggregate, its management team has contributed to several drug approvals, including Avastin®, Herceptin®, Kaletra®, Kyprolis®, Ravicti® and Rituxan®. The company intend to leverage this expertise and experience to rapidly advance the development of voxelotor for SCD and advance other product candidates. Beyond evaluating voxelotor in SCD, Global Blood Therapeutics is also engaged in other research and development activities, all of which are currently in the pre-clinical phase. In addition, the company regularly evaluate opportunities to in-license, acquire or invest in new business, technology or assets or engage in related discussions with other business entities.
44	44
45		-==== Development Pipeline ====
	43	+= Development Pipeline =
46	46
47	47	The following table summarizes its development programs, potential indications, and their current stages of development:
48	48
...	...	@@ -140,7 +140,7 @@
140	140
141	141	In November 2016, the European Commission, or EC, granted Orphan Drug Designation status in the European Union, or EU, for voxelotor for the treatment of SCD. In June 2017, the European Medicines Agency, or EMA, granted PRIME designation for voxelotor for the treatment of SCD. The PRIME program is a new regulatory mechanism that provides for early and proactive EMA support to medicine developers to help patients benefit as early as possible from innovative new products that have demonstrated the potential to significantly address an unmet medical need.
142	142
143		-==== Manufacturing ====
	141	+= Manufacturing =
144	144
145	145	The company do not own or operate, and currently have no plans to establish, any manufacturing facilities. The company currently depend on third-party contract manufacturing organizations, or CMOs, for all of its requirements of raw materials, drug substance and drug product for its nonclinical research and its ongoing clinical trials of its lead product candidate voxelotor. Global Blood Therapeutics has not entered into long-term commercial manufacturing agreements with its current CMOs. The company intend to continue to rely on CMOs for later-stage development and commercialization of voxelotor, as well as the development and commercialization of any other product candidates that the company may identify. Although the company rely on CMOs, Global Blood Therapeutics has personnel and third-party consultants with extensive manufacturing experience to oversee the relationships with its contract manufacturers.
146	146
...	...	@@ -148,7 +148,7 @@
148	148
149	149	Global Blood Therapeutics has completed development for a solid oral formulation of a tablet form of voxelotor as well as a pediatric dispersible tablet formulation of voxelotor.
150	150
151		-==== Intellectual Property ====
	149	+= Intellectual Property =
152	152
153	153	The company strive to protect the proprietary technology that the company believe is important to its business, including seeking and maintaining patents and patent applications intended to cover its product candidates and compositions, their methods of use and processes for their manufacture, and any other aspects of inventions that are commercially important to the development of its business. The company also rely on trade secrets to protect aspects of its business that are not amenable to, or that the company do not consider appropriate for, patent protection.
154	154
...	...	@@ -176,4 +176,7 @@
176	176
177	177	The base term of a U.S. patent is 20 years from the filing date of the earliest-filed non-provisional patent application from which the patent claims priority, assuming that all maintenance fees are paid. The term of a U.S. patent can be lengthened by patent term adjustment, which compensates the owner of the patent for administrative delays at the USPTO the extent of which is offset by delays by the patent owner before the USPTO in obtaining the patent. In some cases, the term of a U.S. patent is shortened by a terminal disclaimer that reduces its term to that of an earlier-expiring patent. The term of a U.S. patent may be eligible for patent term extension under the Drug Price Competition and Patent Term Restoration Act of 1984, referred to as the Hatch-Waxman Act, to account for at least some of the time the drug is under development and regulatory review after the patent is granted. With regard to a drug for which FDA approval is the first permitted marketing of the active ingredient, the Hatch-Waxman Act allows for extension of the term of one U.S. patent that includes at least one claim covering the composition of matter of an FDA-approved drug, an FDA-approved method of treatment using the drug and/or a method of manufacturing the FDA-approved drug. The extended patent term cannot exceed the shorter of five years beyond the non-extended expiration of the patent or 14 years from the date of the FDA approval of the drug. Some foreign jurisdictions, including Europe and Japan, have analogous patent term extension provisions, which allow for extension of the term of a patent that covers a drug approved by the applicable foreign regulatory agency. In the future, if its lead product candidate voxelotor or any other product candidates receive FDA approval, the company would expect to apply for patent term extension on patents, if issued, covering those products, their methods of use and/or methods of manufacture.
178	178
179		-
	177	+= References =
	178	+
	179	+
	180	+{{putFootnotes/}}