Therapeutic Focus

THERAPEUTIC FOCUS

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HEMA Biologics

Putting the focus on specific rare bleeding disorders

Our two lead investigational product candidates are being developed for the potential treatment of hemophilia A or B with inhibitors and severe von Willebrand disease.

Hemophilia A with Inhibitors

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Hemophilia A with Inhibitors

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What are inhibitors?

Before talking about inhibitors, let's first look at the clotting cascade. The clotting cascade shows the many diferent steps that are needed to produce a clot.

The clotting cascade is activated by the platelet response to the leaky blood vessel. The platelet response is called Primary Hemostasis.

Primary Hemostasis: Platelet response to a bleed

There are two main pathways in the clotting cascade: the intrinsic pathway and the extrinsic pathway. People with hemophilia A are lacking factor VIII, which is a key factor in the intrinsic pathway. To form clots, they need to infuse replacement factor VIII, to supply what the body is missing.

Each step activates the enzyme necessary for the next step.

Sometimes, the body reacts to this replacement factor as an invader and forms an antibody against it. These antibodies are referred to as inhibitors to the infused clotting factor. These inhibitors act to eliminate the foreign body, making the replacement factor ineffective.

Learn More at NHF

Who develops an inhibitor?

Approximately 30% of people with severe hemophilia A will develop an inhibitor to infused factor VIII at some point in their lives. Even 5% to 8% of people with mild or moderate hemophilia A develop an inhibitor.

Learn More at NHF

How are bleeds treated in the presence of an inhibitor?

For patients with hemophilia A with low titer inhibitors, therapy with factor VIII can be effective for many bleeding episodes. Higher doses of factor or more frequent infusions may be required to overcome the inhibitor.

When patients have high titer inhibitors, high factor doses become ineffective. At this point, they must switch to an alternate clotting factor(s) to control their bleeding. These factors “bypass” the missing factor VIII to create a functioning clotting system—thus, they are called bypassing agents. FVIIa is normally found in the extrinsic pathway. When given as a treatment, factor VIIa works with platelets at the site of the injury to make clots form to stop bleeding. Other bypassing agents contain other clotting factors in addition to VIIa and also work to stop bleeding.

While there are currently two bypass treatments available, it has been over a decade since any new therapies for patients with an inhibitors have been available.

How do inhibitors affect the lives of patients?

Inhibitors make life more challenging for someone with hemophilia. New therapies may need to be added, which may cause an adjustment period. Bleeding episodes may take longer to control depending on the severity of the inhibitor. The longer bleeding continues, the higher the chance that blood may find its way into muscles and joints, causing additional damage.

What is HEMA Biologics doing to help?

We recognize that for people living with an inhibitor, every day brings challenges. Our primary goal is to advance the treatment and management of hemophilia A with inhibitors to positively affect patients’ lives.

Hemophilia B with Inhibitors

Hemophilia B with Inhibitors

What are inhibitors?

Before talking about inhibitors, let's first look at the clotting cascade. The clotting cascade shows the many diferent steps that are needed to produce a clot.

The clotting cascade is activated by the platelet response to the leaky blood vessel. The platelet response is called Primary Hemostasis.

Primary Hemostasis: Platelet response to a bleed

There are two main pathways in the clotting cascade: the intrinsic pathway and the extrinsic pathway. People with hemophilia B are lacking factor IX, which is a key factor in the intrinsic pathway. To form clots, they need to infuse replacement factor IX, to supply what the body is missing.

Each step activates the enzyme necessary for the next step.

Sometimes, the body reacts to this replacement factor as an invader by forming antibodies to the factor. These antibodies, also known as inhibitors, act to eliminate the foreign protein, making the replacement factor ineffective.

Learn More at NHF

Who develops an inhibitor?

Approximately 2% to 3% of people with hemophilia B will develop an inhibitor to infused factor IX. Although they are less common than in hemophilia A, inhibitors can be even more problematic in patients with hemophilia B due to the potential of developing a severe allergic reaction.

Learn More at NHF

How are bleeds treated in the presence of an inhibitor?

For patients with hemophilia B with low titer inhibitors, therapy with factor IX can be effective for many bleeding episodes. Higher doses of factor or more frequent infusions may be required to overcome the inhibitor.

When patients have high titer inhibitors, high factor doses become ineffective. At this point, they must switch to an alternate clotting factor(s) to control their bleeding. These factors “bypass” the missing factor IX to create a functioning clotting system—thus, they are called bypassing agents. FVIIa is normally found in the extrinsic pathway. When given as a treatment, factor VIIa works with platelets at the site of the injury to make clots form to stop bleeding. Other bypassing agents contain other clotting factors in addition to VIIa and also work to stop bleeding.

While there are currently two bypass treatments available, it has been over a decade since any new therapies for patients with an inhibitors have been available.

How do inhibitors affect the lives of patients?

Inhibitors make life more challenging for someone with hemophilia. New therapies may need to be added, which may cause an adjustment period. Bleeding episodes may take longer to control depending on the severity of the inhibitor. The longer bleeding continues, the higher the chance that blood may find its way into muscles and joints, causing additional damage.

What is HEMA Biologics doing to help?

We recognize that for people living with an inhibitor, every day brings challenges. Our primary goal is to advance the treatment and management of hemophilia B with inhibitors to positively affect patients’ lives.

What are inhibitors?

Before talking about inhibitors, let's first look at the clotting cascade. The clotting cascade shows the many diferent steps that are needed to produce a clot.

The clotting cascade is activated by the platelet response to the leaky blood vessel. The platelet response is called Primary Hemostasis.

Primary Hemostasis: Platelet response to a bleed

There are two main pathways in the clotting cascade: the intrinsic pathway and the extrinsic pathway. People with hemophilia A are lacking factor VIII, which is a key factor in the intrinsic pathway. To form clots, they need to infuse replacement factor VIII, to supply what the body is missing.

Each step activates the enzyme necessary for the next step.

Sometimes, the body reacts to this replacement factor as an invader and forms an antibody against it. These antibodies are referred to as inhibitors to the infused clotting factor. These inhibitors act to eliminate the foreign body, making the replacement factor ineffective.

Learn More at NHF

Who develops an inhibitor?

Approximately 30% of people with severe hemophilia A will develop an inhibitor to infused factor VIII at some point in their lives. Even 5% to 8% of people with mild or moderate hemophilia A develop an inhibitor.

Learn More at NHF

How are bleeds treated in the presence of an inhibitor?

For patients with hemophilia A with low titer inhibitors, therapy with factor VIII can be effective for many bleeding episodes. Higher doses of factor or more frequent infusions may be required to overcome the inhibitor.

When patients have high titer inhibitors, high factor doses become ineffective. At this point, they must switch to an alternate clotting factor(s) to control their bleeding. These factors “bypass” the missing factor VIII to create a functioning clotting system—thus, they are called bypassing agents. FVIIa is normally found in the extrinsic pathway. When given as a treatment, factor VIIa works with platelets at the site of the injury to make clots form to stop bleeding. Other bypassing agents contain other clotting factors in addition to VIIa and also work to stop bleeding.

While there are currently two bypass treatments available, it has been over a decade since any new therapies for patients with an inhibitors have been available.

How do inhibitors affect the lives of patients?

Inhibitors make life more challenging for someone with hemophilia. New therapies may need to be added, which may cause an adjustment period. Bleeding episodes may take longer to control depending on the severity of the inhibitor. The longer bleeding continues, the higher the chance that blood may find its way into muscles and joints, causing additional damage.

What is HEMA Biologics doing to help?

We recognize that for people living with an inhibitor, every day brings challenges. Our primary goal is to advance the treatment and management of hemophilia A with inhibitors to positively affect patients’ lives.

What are inhibitors?

Before talking about inhibitors, let's first look at the clotting cascade. The clotting cascade shows the many diferent steps that are needed to produce a clot.

The clotting cascade is activated by the platelet response to the leaky blood vessel. The platelet response is called Primary Hemostasis.

Primary Hemostasis: Platelet response to a bleed

There are two main pathways in the clotting cascade: the intrinsic pathway and the extrinsic pathway. People with hemophilia B are lacking factor IX, which is a key factor in the intrinsic pathway. To form clots, they need to infuse replacement factor IX, to supply what the body is missing.

Each step activates the enzyme necessary for the next step.

Sometimes, the body reacts to this replacement factor as an invader by forming antibodies to the factor. These antibodies, also known as inhibitors, act to eliminate the foreign protein, making the replacement factor ineffective.

Learn More at NHF

Who develops an inhibitor?

Approximately 2% to 3% of people with hemophilia B will develop an inhibitor to infused factor IX. Although they are less common than in hemophilia A, inhibitors can be even more problematic in patients with hemophilia B due to the potential of developing a severe allergic reaction.

Learn More at NHF

How are bleeds treated in the presence of an inhibitor?

For patients with hemophilia B with low titer inhibitors, therapy with factor IX can be effective for many bleeding episodes. Higher doses of factor or more frequent infusions may be required to overcome the inhibitor.

When patients have high titer inhibitors, high factor doses become ineffective. At this point, they must switch to an alternate clotting factor(s) to control their bleeding. These factors “bypass” the missing factor IX to create a functioning clotting system—thus, they are called bypassing agents. FVIIa is normally found in the extrinsic pathway. When given as a treatment, factor VIIa works with platelets at the site of the injury to make clots form to stop bleeding. Other bypassing agents contain other clotting factors in addition to VIIa and also work to stop bleeding.

While there are currently two bypass treatments available, it has been over a decade since any new therapies for patients with an inhibitors have been available.

How do inhibitors affect the lives of patients?

Inhibitors make life more challenging for someone with hemophilia. New therapies may need to be added, which may cause an adjustment period. Bleeding episodes may take longer to control depending on the severity of the inhibitor. The longer bleeding continues, the higher the chance that blood may find its way into muscles and joints, causing additional damage.

What is HEMA Biologics doing to help?

We recognize that for people living with an inhibitor, every day brings challenges. Our primary goal is to advance the treatment and management of hemophilia B with inhibitors to positively affect patients’ lives.


von Willebrand disease

What is von Willebrand disease?

von Willebrand factor (vWF) is an important component of the clotting cascade. vWF performs two functions leading to effective clotting:

  • It helps make platelets sticky
  • It is a carrier protein for factor VIII

People with von Willebrand disease (vWD) either have decreased levels of vWF or malfunctioning vWF activity, and therefore can’t form proper clots.

Learn More at NHF

Who gets vWD?

vWD is the most common bleeding disorder, with about 1% of the world’s population living with it. Both males and females are affected equally.

Learn More at NHF

What are the types of vWD?

There are three types of vWD:

  • Type 1—the mildest form of vWD. There is vWF present in the blood, but the levels are lower than normal. About 75% of people with vWD have the Type 1 subgroup of the disease.
  • Type 2—in this less common type, some vWF is present in the blood, but it does not work the way that it should.
  • Type 3—in this rare type, there is no vWF present in the blood. This is the most severe form of vWD.

How does severe vWD affect people’s lives?

Any type of vWD can be severe, where frequent, severe bleeding causes patients to make some adjustment in their lives for their disorder. Since any type of bleeding can be potentially life threatening, care must be taken to avoid injury. Women with vWD may experience heavy menstrual bleeding, which can lead to anemia or low blood iron.

What is HEMA Biologics doing to help?

Patients may feel their vWD is under-recognized, which can be frustrating. Our goal is to investigate new options to help people living with severe vWD.